10-Q

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549 

______________________________________

Form 10-Q 

______________________________________

(Mark One)

☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended September 30, 2023

Or

☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from                      to                      .

Commission File Number: 000-26727

______________________________________ 

BioMarin Pharmaceutical Inc.

(Exact name of registrant as specified in its charter)  

______________________________________

Delaware			68-0397820
(State or other jurisdiction of incorporation or organization)			(I.R.S. Employer Identification No.)
770 Lindaro Street	San Rafael	California	94901
(Address of principal executive offices)			(Zip Code)

(415) 506-6700

(Registrant’s telephone number including area code)

______________________________________

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, par value $0.001	BMRN	The Nasdaq Global Select Market

______________________________________

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.    Yes  ☒    No  ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).    Yes  ☒    No  ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act. 

Large Accelerated Filer	☒	Accelerated Filer	☐
Non-accelerated Filer	☐	Smaller Reporting Company	☐
Emerging Growth Company	☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.  ☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act.)    Yes  ☐    No  ☒

Applicable only to corporate issuers:

Indicate the number of shares outstanding of each of the issuer’s classes of common stock, as of the latest practicable date: 188,368,171 shares of common stock, par value $0.001, outstanding as of October 30, 2023.

Unless the context suggests otherwise, references in this Quarterly Report on Form 10-Q to “BioMarin,” the “Company,” “we,” “us,” and “our” refer to BioMarin Pharmaceutical Inc. and, where appropriate, its wholly owned subsidiaries.

BioMarin®, BRINEURA®, KUVAN®, NAGLAZYME®, PALYNZIQ®, VIMIZIM® and VOXZOGO® are our registered trademarks. ROCTAVIAN® is our registered trademark in the European Union. ROCTAVIANTM is our trademark in the United States (U.S.). ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this report are the property of their respective owners.

Forward-Looking Statements

This Quarterly Report on Form 10-Q contains “forward-looking statements” as defined under securities laws. Many of these statements can be identified by the use of terminology such as “believes,” “expects,” “intends,” “anticipates,” “plans,” “may,” “will,” “could,” would,” “projects,” “continues,” “estimates,” “potential,” “opportunity” or the negative versions of these terms and other similar expressions. Our actual results or experience could differ significantly from the forward-looking statements. Factors that could cause or contribute to these differences include those discussed in “Risk Factors,” in Part II, Item 1A of this Quarterly Report on Form 10-Q as well as information provided elsewhere in this Quarterly Report on Form 10-Q and our Annual Report on Form 10-K for the year ended December 31, 2022, which was filed with the Securities and Exchange Commission (the SEC) on February 27, 2023. You should carefully consider that information before you make an investment decision.

You should not place undue reliance on these types of forward-looking statements, which speak only as of the date that they were made. These forward-looking statements are based on the beliefs and assumptions of the Company’s management based on information currently available to management and should be considered in connection with any written or oral forward-looking statements that the Company may issue in the future as well as other cautionary statements the Company has made and may make. Except as required by law, the Company does not undertake any obligation to release publicly any revisions to these forward-looking statements after completion of the filing of this Quarterly Report on Form 10-Q to reflect later events or circumstances or the occurrence of unanticipated events.

The discussion of the Company’s financial condition and results of operations should be read in conjunction with the Company’s Condensed Consolidated Financial Statements and the related Notes thereto included in this Quarterly Report on Form 10-Q.

Risk Factors Summary

The following is a summary of the principal risks that could adversely affect our business, financial condition, operating results, cash flows or stock price. Discussion of the risks listed below, and other risks that we face, are discussed in the section titled “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q.

Business and Operational Risks

•If we fail to obtain and maintain an adequate level of coverage and reimbursement for our products by third-party payers, the sales of our products would be adversely affected or there may be no commercially viable markets for our products.

•Because the target patient populations for our products are relatively small, we must achieve significant market share and maintain high per-patient prices for our products to achieve and maintain profitability.

•If we fail to compete successfully with respect to product sales, we may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product and our revenues could be adversely affected.

•Changes in methods of treatment of disease could reduce demand for our products and adversely affect revenues.

•If we fail to develop new products and product candidates or compete successfully with respect to acquisitions, joint ventures, licenses or other collaboration opportunities, our ability to continue to expand our product pipeline and our growth and development would be impaired.

•The sale of generic versions of KUVAN by generic manufacturers has adversely affected and will continue to adversely affect our revenues and may cause a decline in KUVAN revenues faster than expected.

•If we do not achieve our projected development goals in the timeframes we announce or fail to achieve such goals, the commercialization of our product candidates may be delayed or never occur and the credibility of our management may be adversely affected and, as a result, our stock price may decline.

Regulatory Risks

•If we fail to obtain regulatory approval to commercially market and sell our product candidates, or if approval of our product candidates is delayed, we will be unable to generate revenues from the sale of these product candidates, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will increase.

•Any product for which we have obtained regulatory approval, or for which we obtain approval in the future, is subject to, or will be subject to, extensive ongoing regulatory requirements by the United States Food and Drug Administration (FDA), the European Commission (EC), the European Medicines Agency (EMA) and other comparable international regulatory authorities, and if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products, we may be subject to penalties, we will be unable to generate revenues from the sale of such products, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will be increased.

•To obtain regulatory approval to market our products, preclinical studies and costly and lengthy clinical trials are required and the results of the studies and trials are highly uncertain. Likewise, preliminary, initial or interim data from clinical trials should be considered carefully and with caution because the final data may be materially different from the preliminary, initial or interim data, particularly as more patient data become available.

•Government price controls or other changes in pricing regulation could restrict the amount that we are able to charge for our current and future products, which would adversely affect our revenues and results of operations.

•Government healthcare reform could increase our costs and adversely affect our revenues and results of operations.

Risks Related to Our Gene Therapy Programs

•Our gene therapy products and product candidates are based on a novel technology, which presents additional development, manufacturing, regulatory and treatment risks in relation to our other, more traditional drug development programs.

•As compared to our other, more traditional products, gene therapy products may present additional problems with respect to the pricing, coverage, and reimbursement and acceptance of the product.

Financial and Financing Risks

•If we incur operating losses or are unable to sustain positive cash flows for a period longer than anticipated, we may be unable to continue our operations at planned levels and may be forced to reduce our operations.

Manufacturing Risks

•If we fail to comply with manufacturing regulations, our financial results and financial condition will be adversely affected.

•If we are unable to successfully develop and maintain manufacturing processes for our product candidates to produce sufficient quantities at acceptable costs, we may be unable to support a clinical trial or be forced to terminate a program, or if we are unable to produce sufficient quantities of our products at acceptable costs, we may be unable to meet commercial demand, lose potential revenue, have reduced margins or be forced to terminate a program.

•Supply interruptions may disrupt our inventory levels and the availability of our products and product candidates and cause delays in obtaining regulatory approval for our product candidates, or harm our business by reducing our revenues.

Risks Related to International Operations

•We conduct a significant amount of our sales and operations outside of the U.S., which subjects us to additional business risks that could adversely affect our revenues and results of operations.

•A significant portion of our international sales are made based on special access programs, and changes to these programs could adversely affect our product sales and revenues in these countries.

•Our international operations pose currency risks, which may adversely affect our operating results and net income.

Intellectual Property Risks

•If we are unable to protect our intellectual property, we may not be able to compete effectively or preserve our market shares.

•Competitors and other third parties may have developed intellectual property that could limit our ability to market and commercialize our products and product candidates, if approved.

BIOMARIN PHARMACEUTICAL INC.

TABLE OF CONTENTS

		Page
PART I.	FINANCIAL INFORMATION	3
Item 1.	Financial Statements	3
	Condensed Consolidated Balance Sheets as of September 30, 2023 (Unaudited) and December 31, 2022	3
	Condensed Consolidated Statements of Comprehensive Income (Unaudited) for the three and nine months ended September 30, 2023 and 2022	4
	Condensed Consolidated Statement of Stockholders’ Equity (Unaudited) for the three and nine months ended September 30, 2023 and 2022	5
	Condensed Consolidated Statements of Cash Flows (Unaudited) for the nine months ended September 30, 2023 and 2022	6
	Notes to Condensed Consolidated Financial Statements (Unaudited)	7
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	20
Item 3.	Quantitative and Qualitative Disclosures about Market Risk	32
Item 4.	Controls and Procedures	32
PART II.	OTHER INFORMATION	33
Item 1.	Legal Proceedings	33
Item 1A.	Risk Factors	33
Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds	60
Item 3.	Defaults Upon Senior Securities	60
Item 4.	Mine Safety Disclosures	61
Item 5.	Other Information	61
Item 6.	Exhibits	62
SIGNATURES		64

2

PART I. FINANCIAL INFORMATION

Item 1.    Financial Statements

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

September 30, 2023 and December 31, 2022

(In thousands, except share amounts)

	September 30, 2023		December 31, 2022 ⁽¹⁾
ASSETS	(unaudited)
Current assets:
Cash and cash equivalents	$	785,414	$	724,531
Short-term investments	340,431		567,006
Accounts receivable, net	572,498		461,316
Inventory	1,032,159		894,083
Other current assets	224,806		104,521
Total current assets	2,955,308		2,751,457
Noncurrent assets:
Long-term investments	548,002		333,835
Property, plant and equipment, net	1,067,156		1,073,366
Intangible assets, net	302,476		338,569
Goodwill	196,199		196,199
Deferred tax assets	1,523,953		1,505,412
Other assets	165,069		176,236
Total assets	$	6,758,163	$	6,375,074
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable and accrued liabilities	$	669,825	$	572,959
Short-term convertible debt, net	493,398		—
Short-term contingent consideration	—		15,925
Total current liabilities	1,163,223		588,884
Noncurrent liabilities:
Long-term convertible debt, net	592,586		1,083,019
Other long-term liabilities	105,457		100,015
Total liabilities	1,861,266		1,771,918
Stockholders’ equity:
Common stock, $0.001 par value: 500,000,000 shares authorized; 188,339,482 and 186,250,719 shares issued and outstanding, respectively	188		186
Additional paid-in capital	5,542,774		5,404,895
Company common stock held by the Nonqualified Deferred Compensation Plan (the NQDC)	(10,393)		(8,859)
Accumulated other comprehensive income (loss)	6,257		(3,867)
Accumulated deficit	(641,929)		(789,199)
Total stockholders’ equity	4,896,897		4,603,156
Total liabilities and stockholders’ equity	$	6,758,163	$	6,375,074

    

(1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

The accompanying notes are an integral part of these Condensed Consolidated Financial Statements.

3

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME

Three and Nine Months Ended September 30, 2023 and 2022

(In thousands, except per share amounts)

(unaudited)

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
REVENUES:
Net product revenues	$	568,266	$	493,348	$	1,739,390	$	1,516,533
Royalty and other revenues	13,063		11,996		33,629		41,968
Total revenues	581,329		505,344		1,773,019		1,558,501
OPERATING EXPENSES:
Cost of sales	124,745		116,288		379,376		356,379
Research and development	191,314		157,829		540,523		476,855
Selling, general and administrative	223,928		216,816		662,267		608,270
Intangible asset amortization and contingent consideration	15,681		16,828		46,975		50,935
Gain on sale of nonfinancial assets, net	—		—		—		(108,000)
Total operating expenses	555,668		507,761		1,629,141		1,384,439
INCOME (LOSS) FROM OPERATIONS	25,661		(2,417)		143,878		174,062
Interest income	15,740		4,999		40,295		9,324
Interest expense	(3,779)		(4,679)		(11,237)		(12,344)
Other income (expense), net	4,047		193		(3,700)		(3,908)
INCOME (LOSS) BEFORE INCOME TAXES	41,669		(1,904)		169,236		167,134
Provision for income taxes	1,291		4,748		21,966		25,324
NET INCOME (LOSS)	$	40,378	$	(6,652)	$	147,270	$	141,810
EARNINGS (LOSS) PER SHARE, BASIC	$	0.21	$	(0.04)	$	0.78	$	0.77
EARNINGS (LOSS) PER SHARE, DILUTED	$	0.21	$	(0.04)	$	0.77	$	0.75
Weighted average common shares outstanding, basic	188,219		185,597		187,617		185,009
Weighted average common shares outstanding, diluted	191,173		185,597		195,042		192,252
COMPREHENSIVE INCOME	$	65,252	$	5,625	$	157,394	$	158,127

The accompanying notes are an integral part of these Condensed Consolidated Financial Statements.

4

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

Three and Nine Months Ended September 30, 2023 and 2022

(In thousands)

(unaudited)

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Shares of common stock, beginning balances (1)	188,152		185,453		186,251		183,913
Issuances under equity incentive plans	187		372		2,088		1,912
Shares of common stock, ending balances	188,339		185,825		188,339		185,825
Total stockholders' equity, beginning balances (1)	$	4,782,827	$	4,499,737	$	4,603,156	$	4,265,670
Common stock:
Beginning balances (1)	188		186		186		184
Issuances under equity incentive plans, net of tax	—		—		2		2
Ending balances	188		186		188		186
Additional paid-in capital:
Beginning balances (1)	5,493,956		5,272,666		5,404,895		5,191,502
Issuances under equity incentive plans, net of tax	(1,827)		6,847		(19,778)		(8,306)
Stock-based compensation	50,645		56,305		156,123		153,021
Common stock held by the NQDC	—		35		1,534		(364)
Ending balances	5,542,774		5,335,853		5,542,774		5,335,853
Company common stock held by the NQDC:
Beginning balances (1)	(10,393)		(9,290)		(8,859)		(9,689)
Common stock held by the NQDC	—		(35)		(1,534)		364
Ending balances	(10,393)		(9,325)		(10,393)		(9,325)
Accumulated other comprehensive income (loss):
Beginning balances (1)	(18,617)		18,472		(3,867)		14,432
Other comprehensive income	24,874		12,277		10,124		16,317
Ending balances	6,257		30,749		6,257		30,749
Accumulated Deficit:
Beginning balances (1)	(682,307)		(782,297)		(789,199)		(930,759)
Net income (loss)	40,378		(6,652)		147,270		141,810
Ending balances	(641,929)		(788,949)		(641,929)		(788,949)
Total stockholders' equity, ending balances	$	4,896,897	$	4,568,514	$	4,896,897	$	4,568,514

(1)The beginning balances for the nine-month periods were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 filed with the SEC on February 27, 2023.

The accompanying notes are an integral part of these Condensed Consolidated Financial Statements. 

5

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

Nine Months Ended September 30, 2023 and 2022

(In thousands)

(unaudited)

	Nine Months Ended September 30,
	2023		2022
CASH FLOWS FROM OPERATING ACTIVITIES:
Net income	$	147,270	$	141,810
Adjustments to reconcile net income to net cash used in operating activities:
Depreciation and amortization	77,525		77,416
Non-cash interest expense	3,198		3,089
Amortization of premium (accretion of discount) on investments	(6,781)		3,741
Stock-based compensation	152,244		149,574
Gain on sale of nonfinancial assets, net	—		(108,000)
Loss on equity investment	12,650		—
Deferred income taxes	(20,137)		(743)
Unrealized foreign exchange loss (gain)	5,454		(16,075)
Non-cash changes in the fair value of contingent consideration	—		2,243
Other	(224)		(700)
Changes in operating assets and liabilities:
Accounts receivable, net	(131,940)		(53,752)
Inventory	(97,948)		(27,419)
Other current assets	(59,389)		(8,558)
Other assets	(20,812)		12,140
Accounts payable and other short-term liabilities	56,333		(2,398)
Other long-term liabilities	14,333		(3,252)
Net cash provided by operating activities	131,776		169,116
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property, plant and equipment	(67,774)		(85,271)
Maturities and sales of investments	751,677		477,244
Purchases of investments	(727,043)		(457,382)
Proceeds from sale of nonfinancial assets	—		110,000
Purchase of intangible assets	(3,141)		(9,910)
Net cash provided by (used in) investing activities	(46,281)		34,681
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from exercises of awards under equity incentive plans	54,548		43,866
Taxes paid related to net share settlement of equity awards	(72,399)		(50,696)
Payments of contingent consideration	(9,475)		(21,054)
Principal repayments of financing leases	(2,241)		(1,635)
Net cash used in financing activities	(29,567)		(29,519)
Effect of exchange rate changes on cash	4,955		(39)
NET INCREASE IN CASH AND CASH EQUIVALENTS	60,883		174,239
Cash and cash equivalents:
Beginning of period	$	724,531	$	587,276
End of period	$	785,414	$	761,515
SUPPLEMENTAL CASH FLOW DISCLOSURES:
Cash paid for interest	$	6,640	$	6,575
Cash paid for income taxes	$	51,687	$	13,425
SUPPLEMENTAL CASH FLOW DISCLOSURES FOR NON-CASH INVESTING AND FINANCING ACTIVITIES:
Increase (decrease) in accounts payable and accrued liabilities related to fixed assets	$	7,977	$	(5,240)
Increase (decrease) in accounts payable and accrued liabilities related to intangible assets	$	6,449	$	(224)

The accompanying notes are an integral part of these Condensed Consolidated Financial Statements.

6

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

(1) BUSINESS OVERVIEW AND SIGNIFICANT ACCOUNTING POLICIES

Nature of Operations

Founded in 1997, BioMarin Pharmaceutical Inc. (the Company or BioMarin) is a global biotechnology company dedicated to transforming lives through genetic discovery. The Company develops and commercializes targeted therapies that address the root cause of genetic conditions. The Company's robust research and development (R&D) capabilities have resulted in multiple innovative commercial therapies for patients with rare genetic disorders. The Company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options.

Basis of Presentation

These Condensed Consolidated Financial Statements have been prepared pursuant to U.S. generally accepted accounting principles (U.S. GAAP) and the rules and regulations of the SEC for Quarterly Reports on Form 10-Q and do not include all of the information and note disclosures required by U.S. GAAP for complete financial statements, although management believes that the disclosures herein are adequate to ensure that the information presented is not misleading. The Condensed Consolidated Financial Statements should therefore be read in conjunction with the Consolidated Financial Statements and Notes thereto for the fiscal year ended December 31, 2022 included in the Company’s Annual Report on Form 10-K. The Condensed Consolidated Financial Statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany transactions have been eliminated. The results of operations for the three and nine months ended September 30, 2023 are not necessarily indicative of the results that may be expected for the fiscal year ending December 31, 2023 or any other period.

Use of Estimates

U.S. GAAP requires management to make estimates and assumptions that affect amounts reported in the Condensed Consolidated Financial Statements and accompanying disclosures. Although these estimates are based on management’s best knowledge of current events and actions that the Company may undertake in the future, actual results may be different from those estimates. The Condensed Consolidated Financial Statements reflect all adjustments of a normal, recurring nature that are, in the opinion of management, necessary for a fair presentation of results for these interim periods.

Management performed an evaluation of the Company’s activities through the date of filing of this Quarterly Report on Form 10-Q and has concluded that there were no subsequent events or transactions that occurred subsequent to the balance sheet date prior to filing this Quarterly Report on Form 10-Q that would require recognition or disclosure in the Condensed Consolidated Financial Statements.

Significant Accounting Policies

There have been no material changes to the Company’s significant accounting policies during the nine months ended September 30, 2023, as compared to the significant accounting policies disclosed in Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.

Recent Accounting Pronouncements

There have been no new accounting pronouncements adopted by the Company or new accounting pronouncements issued by the Financial Accounting Standards Board during the nine months ended September 30, 2023, as compared to the recent accounting pronouncements described in Note 1 to the Company’s Consolidated Financial Statements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, that the Company believes are of significance or potential significance to the Company.

(2) FINANCIAL INSTRUMENTS

All marketable securities were classified as available-for-sale as of September 30, 2023 and December 31, 2022.

The following tables show the Company’s cash, cash equivalents and available-for-sale securities by significant investment category for each period presented:

7

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS - (continued)

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

	September 30, 2023
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash	$	451,294	$	—	$	—	$	451,294	$	451,294	$	—	$	—
Level 2:
Money market instruments	291,425		—		—		291,425		291,425		—		—
Corporate debt securities	563,598		31		(6,135)		557,494		731		217,306		339,457
U.S. government agency securities	281,045		4		(3,023)		278,026		29,981		112,714		135,331
Commercial paper	21,864		—		(4)		21,860		11,983		9,877		—
Asset-backed securities	74,168		26		(446)		73,748		—		534		73,214
Subtotal	1,232,100		61		(9,608)		1,222,553		334,120		340,431		548,002
Total	$	1,683,394	$	61	$	(9,608)	$	1,673,847	$	785,414	$	340,431	$	548,002

	December 31, 2022
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash	$	463,248	$	—	$	—	$	463,248	$	463,248	$	—	$	—
Level 2:
Money market instruments	248,933		—		—		248,933		248,933		—		—
Corporate debt securities	504,984		34		(11,541)		493,477		1,881		299,153		192,443
U.S. government agency securities	312,720		45		(3,771)		308,994		—		229,846		79,148
Commercial paper	48,103		11		(22)		48,092		10,469		37,623		—
Asset-backed securities	63,151		69		(592)		62,628		—		384		62,244
Subtotal	1,177,891		159		(15,926)		1,162,124		261,283		567,006		333,835
Total	$	1,641,139	$	159	$	(15,926)	$	1,625,372	$	724,531	$	567,006	$	333,835

(1)    The Company’s short-term marketable securities mature in one year or less.

(2)    The Company’s long-term marketable securities mature between one and five years.

As of September 30, 2023, the Company had the ability and intent to hold all investments that were in an unrealized loss position until maturity. The Company considered its intent and ability to hold the securities until recovery of amortized cost basis, the extent to which fair value is less than amortized cost basis, conditions specifically related to the security’s industry and geography, payment structure and history and changes to the ratings (if any) in determining that the decline in fair value compared to carrying value is not related to a credit loss.

The Company has certain investments in non-marketable equity securities, measured using unobservable valuation inputs and remeasured on a nonrecurring basis, which are collectively considered strategic investments. As of September 30, 2023 and December 31, 2022, the fair value of the Company’s strategic investments was $11.2 million and $23.9 million, respectively. These investments were recorded to Other Assets in the Company’s Condensed Consolidated Balance Sheets. In the first quarter of 2023, based on new developments, the Company concluded that factors existed indicating it would no longer realize a $12.6 million equity investment in its non-marketable securities. The loss on the equity investment due to impairment was recorded to Other Income (Expense), Net on the Company’s Condensed Consolidated Statements of Comprehensive Income. See Note 1 - Business Overview and Significant Accounting Policies included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 for additional information related to the Company’s non-marketable securities policy.

8

BIOMARIN PHARMACEUTICAL INC.

NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

(3) SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION

Supplemental Balance Sheet Information

Inventory consisted of the following:

	September 30, 2023		December 31, 2022
Raw materials	$	142,133	$	131,071
Work-in-process	518,893		410,656
Finished goods	371,133		352,356
Total inventory	$	1,032,159	$	894,083

Property, Plant and Equipment, Net consisted of the following:

	September 30, 2023		December 31, 2022
Property, plant and equipment, gross	$	1,918,609	$	1,875,511
Accumulated depreciation	(851,453)		(802,145)
Total property, plant and equipment, net	$	1,067,156	$	1,073,366

Depreciation expense, net of amounts capitalized into inventory, for the three and nine months ended September 30, 2023 was $9.9 million and $29.6 million, respectively. Depreciation expense, net of amounts capitalized into inventory, for the three and nine months ended September 30, 2022 was $8.7 million and $29.9 million, respectively.

Intangible Assets, Net consisted of the following:

	September 30, 2023		December 31, 2022
Finite-lived intangible assets	$	701,793	$	690,871
Accumulated amortization	(399,317)		(352,302)
Net carrying value	$	302,476	$	338,569

    

Accounts Payable and Accrued Liabilities consisted of the following:

	September 30, 2023		December 31, 2022
Accounts payable and accrued operating expenses	$	301,369	$	231,238
Accrued compensation expense	175,183		207,573
Accrued rebates payable	103,379		72,654
Accrued litigation expense	39,000		—
Accrued royalties payable	12,293		13,306
Accrued income taxes	11,273		16,213
Foreign currency exchange forward contracts	10,584		12,601
Lease liability	9,324		10,375
Other	7,420		8,999
Total accounts payable and accrued liabilities	$	669,825	$	572,959

Reorganization Plan

On October 6, 2022, the Company announced a plan to simplify its organization that included a planned reduction in force of approximately 120 employees (representing approximately 4% of its global workforce), which was substantially completed as of December 31, 2022. The Company recorded costs of $23.0 million in 2022 and negligible adjustments in 2023 related to one-time

9

BIOMARIN PHARMACEUTICAL INC.

NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

severance and employee termination benefits within Selling, General and Administrative in the Company’s Condensed Consolidated Statements of Comprehensive Income. As of December 31, 2022, $11.1 million was included in Accounts Payable and Accrued Liabilities on the Company’s Consolidated Balance Sheet. As of September 30, 2023, substantially all costs have been paid.

(4) FAIR VALUE MEASUREMENTS

The Company measures certain financial assets and liabilities at fair value in accordance with the policy described in Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.

The following tables present the classification within the fair value hierarchy of financial assets and liabilities not disclosed elsewhere in these Condensed Consolidated Financial Statements that are remeasured on a recurring basis as of September 30, 2023 and December 31, 2022. Other than the Company’s fixed-rate convertible debt disclosed in Note 6 – Debt, there were no financial assets or liabilities that were remeasured using a quoted price in active markets for identical assets (Level 1) as of September 30, 2023 or December 31, 2022.

	Fair Value Measurements as of September 30, 2023
		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Other current assets:
NQDC Plan assets		$	1,836	$	—	$	1,836
Other assets:
NQDC Plan assets		25,541		—		25,541
Restricted investments (1)		2,361		—		2,361
Total other assets		27,902		—		27,902
Total assets		$	29,738	$	—	$	29,738
Liabilities:
Current liabilities:
NQDC Plan liability		$	1,836	$	—	$	1,836
Other long-term liabilities:
NQDC Plan liability		25,541		—		25,541
Total liabilities		$	27,377	$	—	$	27,377

10

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS - (continued)

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

	Fair Value Measurements as of December 31, 2022
	Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Other current assets:
NQDC Plan assets	$	2,654	$	—	$	2,654
Other assets:
NQDC Plan assets	19,867		—		19,867
Restricted investments (1)	2,429		—		2,429
Total other assets	22,296		—		22,296
Total assets	$	24,950	$	—	$	24,950
Liabilities:
Current liabilities:
NQDC Plan liability	$	2,654	$	—	$	2,654
Contingent consideration	—		15,925		15,925
Total current liabilities	2,654		15,925		18,579
Other long-term liabilities:
NQDC Plan liability	19,867		—		19,867
Total liabilities	$	22,521	$	15,925	$	38,446

(1)    The restricted investments as of September 30, 2023 and December 31, 2022 secure the Company's irrevocable standby letters of credit obtained in connection with certain commercial agreements.

There were no transfers between levels during the three and nine months ended September 30, 2023.

Liabilities measured at fair value using Level 3 inputs consisted of contingent consideration. The following table represents a roll-forward of contingent consideration.

Contingent consideration as of December 31, 2022	$	15,925
Milestone payments to Ares Trading S.A. (Merck Serono)	(16,255)
Realized foreign exchange loss on settlement of contingent consideration	330
Contingent consideration as of September 30, 2023	$	—

(5) DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES

The Company uses foreign currency exchange forward contracts (forward contracts) to protect against the impact of changes in the value of forecasted foreign currency cash flows resulting from revenues and operating expenses denominated in currencies other than the U.S. Dollar (USD), primarily the Euro. Certain of these forward contracts are designated as cash flow hedges and have maturities of up to two years. The Company also enters into forward contracts to manage foreign exchange risk related to asset or liability positions denominated in currencies other than USD. Such forward contracts are considered to be economic hedges, are not designated as hedging instruments and have maturities of up to six months. The Company does not use derivative instruments for speculative trading purposes. The Company is exposed to counterparty credit risk on its derivatives. The Company has established and maintains strict counterparty credit guidelines and enters into hedging agreements with financial institutions that are investment grade or better to minimize the Company’s exposure to potential defaults. The Company is not required to pledge collateral under these agreements.

11

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS - (continued)

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

The following table summarizes the aggregate notional amounts for the Company’s derivatives outstanding as of the periods presented.

Forward Contracts	September 30, 2023		December 31, 2022
Derivatives designated as hedging instruments:
Sell	$	988,286	$	808,635
Purchase	$	180,560	$	177,393
Derivatives not designated as hedging instruments:
Sell	$	301,825	$	218,903
Purchase	$	6,517	$	6,785

The fair value carrying amounts of the Company’s derivatives, which are classified as Level 2 within the fair value hierarchy, were as follows:

Balance Sheet Location	September 30, 2023		December 31, 2022
Derivatives designated as hedging instruments:
Asset Derivatives
Other current assets	$	21,267	$	19,464
Other assets	7,620		2,059
Subtotal	$	28,887	$	21,523
Liability Derivatives
Accounts payable and accrued liabilities	$	9,918	$	12,130
Other long-term liabilities	3,623		1,074
Subtotal	$	13,541	$	13,204
Derivatives not designated as hedging instruments:
Asset Derivatives
Other current assets	$	5,210	$	1,472
Liability Derivatives
Accounts payable and accrued liabilities	$	666	$	471
Total Derivatives Assets	$	34,097	$	22,995
Total Derivatives Liabilities	$	14,207	$	13,675

For additional discussion of fair value measurements, see Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.

12

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS - (continued)

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

The following tables summarize the impact of gains and losses from the Company's derivatives on its Condensed Consolidated Statements of Comprehensive Income for the periods presented.

	Three Months Ended September 30,
	2023			2022
Derivatives Designated as Cash Flow Hedging Instruments		Cash Flow Hedging Gains (Losses) Reclassified into Earnings		Cash Flow Hedging Gains (Losses) Reclassified into Earnings
Net product revenues		$	(4,636)	$	15,280
Operating expenses		$	908	$	(3,936)
Derivatives Not Designated as Hedging Instruments		Gains (Losses) Recognized in Earnings		Gains (Losses) Recognized in Earnings
Operating expenses		$	6,559	$	6,572

	Nine Months Ended September 30,
	2023			2022
Derivatives Designated as Cash Flow Hedging Instruments		Cash Flow Hedging Gains (Losses) Reclassified into Earnings		Cash Flow Hedging Gains (Losses) Reclassified into Earnings
Net product revenues		$	(489)	$	26,779
Operating expenses		$	426	$	(7,115)
Derivatives Not Designated as Hedging Instruments		Gains (Losses) Recognized in Earnings		Gains (Losses) Recognized in Earnings
Operating expenses		$	3,165	$	12,812

As of September 30, 2023, the Company expects to reclassify unrealized gains of $9.6 million from Accumulated Other Comprehensive Income (AOCI) to earnings as the forecasted revenues and operating expense transactions occur over the next twelve months. For additional discussion of balances in AOCI see Note 7 – Accumulated Other Comprehensive Income.

13

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS - (continued)

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

(6) DEBT

Convertible Notes

As of September 30, 2023, the Company had outstanding fixed-rate notes with varying maturities for an undiscounted aggregate principal amount of $1.1 billion (collectively, the Notes). The Notes are senior subordinated convertible obligations, and interest is payable in arrears, semi-annually. The following table summarizes information regarding the Company’s convertible debt:

	September 30, 2023		December 31, 2022
1.25% senior subordinated convertible notes due in May 2027 (the 2027 Notes)	$	600,000	$	600,000
Unamortized discount net of deferred offering costs	(7,414)		(8,941)
2027 Notes, net	592,586		591,059
0.599% senior subordinated convertible notes due in August 2024 (the 2024 Notes)	495,000		495,000
Unamortized discount net of deferred offering costs	(1,602)		(3,040)
2024 Notes, net (1)	493,398		491,960
Total convertible debt, net	$	1,085,984	$	1,083,019
Fair value of fixed-rate convertible debt (2):
2027 Notes	$	595,278	$	647,370
2024 Notes	480,417		526,230
Total fair value of fixed-rate convertible debt	$	1,075,695	$	1,173,600

(1)    As the 2024 Notes mature in August 2024, the outstanding principal of the 2024 Notes is classified as a current liability as of September 30, 2023.

(2)     The fair value of the Company’s fixed-rate convertible debt is based on open-market trades and is classified as Level 1 in the fair value hierarchy. For additional discussion of fair value measurements, see Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.

Interest expense on the Company’s convertible debt consisted of the following: 

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Coupon interest expense	$	2,616	$	2,616	$	7,849	$	7,849
Accretion of discount on convertible notes	841		838		2,519		2,511
Amortization of debt issuance costs	148		148		445		445
Total interest expense on convertible debt	$	3,605	$	3,602	$	10,813	$	10,805

See Note 10 - Debt to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 for additional information related to the Company’s convertible debt.

Revolving Credit Facility

In October 2018, the Company entered into an unsecured revolving credit facility of up to $200.0 million which included a letter of credit subfacility and a swingline loan subfacility. The credit facility was intended to finance ongoing working capital needs and for other general corporate purposes. In May 2021, the credit facility was amended to extend the original maturity date from October 19, 2021 to May 28, 2024. The credit facility was terminated on August 4, 2023 and there were no amounts outstanding under the credit facility as of September 30, 2023.

14

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS - (continued)

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

(7) ACCUMULATED OTHER COMPREHENSIVE INCOME

The following tables summarize changes in the accumulated balances for each component of AOCI, including current-period other comprehensive income and reclassifications out of AOCI, for the periods presented.

	Three Months Ended September 30, 2023
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of June 30, 2023	$	(10,035)	$	(8,582)	$	(18,617)
Other comprehensive income (loss) before      reclassifications	19,892		1,634		21,526
Less: gain (loss) reclassified from AOCI	(3,728)		—		(3,728)
Tax effect	—		(380)		(380)
Net current-period other comprehensive income (loss)	23,620		1,254		24,874
AOCI balance as of September 30, 2023	$	13,585	$	(7,328)	$	6,257

	Three Months Ended September 30, 2022
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of June 30, 2022	$	30,515	$	(12,043)	$	18,472
Other comprehensive income (loss) before      reclassifications	26,698		(4,011)		22,687
Less: gain (loss) reclassified from AOCI	11,344		—		11,344
Tax effect	—		934		934
Net current-period other comprehensive income (loss)	15,354		(3,077)		12,277
AOCI balance as of September 30, 2022	$	45,869	$	(15,120)	$	30,749

	Nine Months Ended September 30, 2023
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of December 31, 2022	$	8,226	$	(12,093)	$	(3,867)
Other comprehensive income (loss) before      reclassifications	5,296		6,220		11,516
Less: gain (loss) reclassified from AOCI	(63)		—		(63)
Tax effect	—		(1,455)		(1,455)
Net current-period other comprehensive income (loss)	5,359		4,765		10,124
AOCI balance as of September 30, 2023	$	13,585	$	(7,328)	$	6,257

	Nine Months Ended September 30, 2022
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of December 31, 2021	$	15,805	$	(1,373)	$	14,432
Other comprehensive income (loss) before      reclassifications	49,728		(17,905)		31,823
Less: gain (loss) reclassified from AOCI	19,664		—		19,664
Tax effect	—		4,158		4,158
Net current-period other comprehensive income (loss)	30,064		(13,747)		16,317
AOCI balance as of September 30, 2022	$	45,869	$	(15,120)	$	30,749

15

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS - (continued)

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

For additional discussion of reclassifications from AOCI see Note 5 – Derivative Instruments and Hedging Strategies.

(8) REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION

The Company operates in one business segment, which primarily focuses on the development and commercialization of innovative therapies for people with serious and life-threatening rare diseases and medical conditions.

The following table disaggregates total Net Product Revenues by product.

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Enzyme product revenues:
VIMIZIM	$	158,869	$	155,470	$	525,453	$	511,735
NAGLAZYME	108,916		99,506		322,040		343,320
PALYNZIQ	78,885		66,206		216,105		182,734
BRINEURA	41,042		37,793		118,244		111,691
ALDURAZYME	13,812		29,046		88,533		90,755
Total enzyme product revenues	401,524		388,021		1,270,375		1,240,235
VOXZOGO	122,973		48,289		324,146		102,321
KUVAN	42,947		57,038		144,047		173,977
ROCTAVIAN	822		—		822		—
Total net product revenues	568,266		493,348		1,739,390		1,516,533
Royalty and other revenues	13,063		11,996		33,629		41,968
Total revenues	$	581,329	$	505,344	$	1,773,019	$	1,558,501

The Company considers there to be revenue concentration risks for regions where Net Product Revenues exceed 10% of consolidated Net Product Revenues. The concentration of the Company’s Net Product Revenues within the regions below may have a material adverse effect on the Company’s revenues and results of operations if sales in the respective regions experience difficulties. The table below disaggregates total Net Product Revenues by geographic region, which is based on patient location for Company's commercial products sold directly by the Company, except for ALDURAZYME, which is marketed and sold exclusively by Sanofi worldwide.

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
United States	$	194,745	$	176,552	$	553,184	$	497,205
Europe	168,784		162,583		512,910		480,645
Latin America	81,524		60,791		224,716		213,246
Rest of world	109,401		64,376		360,047		234,682
Total net product revenues marketed by the Company	$	554,454	$	464,302	$	1,650,857	$	1,425,778
ALDURAZYME net product revenues marketed by Sanofi	13,812		29,046		88,533		90,755
Total net product revenues	$	568,266	$	493,348	$	1,739,390	$	1,516,533

16

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS - (continued)

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

The following table illustrates the percentage of the Company’s total Net Product Revenues attributed to the Company’s largest customers for the periods presented. 

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Customer A	15	%	15	%	15	%	16	%
Customer B	13	%	14	%	12	%	12	%
Customer C	10	%	10	%	10	%	9	%
Total	38	%	39	%	37	%	37	%

On a consolidated basis, two customers accounted for 16% and 11% of the Company’s September 30, 2023 accounts receivable balance, respectively, compared to December 31, 2022, when two customers accounted for 22% and 15% of the accounts receivable balance, respectively. As of September 30, 2023, and December 31, 2022, the accounts receivable balance for Sanofi included $54.8 million and $68.8 million, respectively, of unbilled accounts receivable, which becomes payable to the Company when the product is sold through by Sanofi. The Company does not require collateral from its customers, but does perform periodic credit evaluations of its customers’ financial condition and requires prepayments in certain circumstances.

The Company is mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, banking crises, natural disasters, the continuing effects of the COVID-19 pandemic, and supply chain disruptions, could affect the Company’s ability to achieve its goals. In addition, the Company sells its products in countries that face economic volatility and weakness. Although the Company has historically collected receivables from customers in certain countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to delay payment or be unable to pay for the Company’s products. The Company believes that the allowances for doubtful accounts related to these countries, if any, are adequate based on its analysis of the specific business circumstances and expectations of collection for each of the underlying accounts in these countries. The Company will continue to monitor these conditions and will attempt to adjust its business processes, as appropriate, to mitigate macroeconomic risks to its business.

(9) STOCK-BASED COMPENSATION

The Company has stockholder-approved equity incentive plans that provide for the granting of restricted stock units (RSUs) and stock options as well as other forms of equity compensation to its employees, officers and non-employee directors. The Company also has an Employee Stock Purchase Plan (ESPP). Compensation expense included in the Company’s Condensed Consolidated Statements of Comprehensive Income for all stock-based compensation arrangements was as follows: 

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Cost of sales	$	4,042	$	4,361	$	13,094	$	13,449
Research and development	14,490		17,055		49,373		47,878
Selling, general and administrative	29,855		33,247		89,777		88,247
Total stock-based compensation expense	$	48,387	$	54,663	$	152,244	$	149,574

(10) EARNINGS (LOSS) PER COMMON SHARE

Potentially issuable shares of common stock include shares issuable upon the exercise of outstanding employee stock option awards, common stock issuable under the ESPP, unvested RSUs and contingent issuances of common stock related to the Company’s convertible debt.

17

BIOMARIN PHARMACEUTICAL INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS - (continued)

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

The following table sets forth the computation of basic and diluted earnings (loss) per common share (common shares in thousands):

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Numerator:
Net Income (loss), basic	$	40,378	$	(6,652)	$	147,270	$	141,810
Add: Interest expense, net of tax, on the Company's convertible debt	—		—		2,810		2,806
Net Income (loss), diluted	$	40,378	$	(6,652)	$	150,080	$	144,616
Denominator:
Weighted-average common shares outstanding, basic	188,219		185,597		187,617		185,009
Effect of dilutive securities:
Issuances under equity incentive plans	2,954		—		3,455		3,273
Common stock issuable under the Company’s convertible debt(1)	—		—		3,970		3,970
Weighted-average common shares outstanding, diluted	191,173		185,597		195,042		192,252
Earnings (loss) per common share, basic	$	0.21	$	(0.04)	$	0.78	$	0.77
Earnings (loss) per common share, diluted	$	0.21	$	(0.04)	$	0.77	$	0.75

In addition to the equity instruments included in the table above, the table below presents potential shares of common stock that were excluded from the computation of diluted earnings per common share as they were anti-dilutive (in thousands):

	Three Months Ended September 30,		Nine Months Ended September 30,
	2023	2022	2023	2022
Issuances under equity incentive plans	8,547	12,221	8,045	8,948
Common stock issuable under the Company’s convertible debt (1)	8,335	8,335	4,365	4,365
Total number of potentially issuable shares	16,882	20,556	12,410	13,313

(1)    If converted, the Company would issue 4.0 million shares under the 2024 Notes and 4.4 million shares under the 2027 Notes. For additional discussion of our convertible debt, see Note 6 - Debt.

(11) COMMITMENTS AND CONTINGENCIES

Contingencies

From time to time the Company is involved in legal actions arising in the normal course of its business. The process of resolving matters through litigation or other means is inherently uncertain and it is possible that an unfavorable resolution of these matters could adversely affect the Company, its results of operations, financial condition or cash flows. The Company’s general practice is to expense legal fees as services are rendered in connection with legal matters, and to accrue for liabilities when losses are probable and reasonably estimable based on existing information. The Company accrues for the best estimate of a loss within a range; however, if no estimate in the range is better than any other, then the minimum amount in the range is accrued. Liabilities are evaluated and refined each reporting period as additional information is known. Any receivables for insurance recoveries for these liability claims are recorded as assets when it is probable that a recovery will be realized.

18

BIOMARIN PHARMACEUTICAL INC.

NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

(In thousands of U.S. Dollars, except per share amounts or as otherwise disclosed)

The Company is involved in a purported shareholder class action lawsuit filed against the Company and certain officers and directors alleging violations under Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 as amended (the Exchange Act) for making materially false or misleading statements regarding the clinical trials and Biologics License Application (BLA) for ROCTAVIAN (formerly known as valoctocogene roxaparvovec) by purportedly failing to disclose that differences between the Company’s Phase 1/2 and Phase 3 clinical studies limited the ability of the Phase 1/2 study to support ROCTAVIAN’s durability of effect and, as a result, that it was foreseeable that the FDA would not approve the BLA without additional data. On March 21, 2023, the Court entered an order staying all proceedings and vacating all deadlines because the parties agreed to settle the case through a binding term sheet. The Court preliminarily approved the settlement on June 8, 2023. On October 4, 2023, plaintiff filed a motion for final approval of settlement. A final approval hearing is scheduled for November 8, 2023. The Company maintains directors and officers liability insurance that covers exposure related to this class action lawsuit. As of September 30, 2023, based on these terms the Company has recorded an estimated loss contingency of $39.0 million to Accounts Payable and Accrued Liabilities and the same amount recorded for expected insurance recoveries in Other Current Assets on the Company’s Condensed Consolidated Balance Sheets. As such, there was a net zero impact on the Company’s Condensed Consolidated Statement of Comprehensive Income in the three and nine months ended September 30, 2023.

Contingent Payments

As of September 30, 2023, the Company was subject to contingent payments considered reasonably possible of $788.3 million, of which $591.5 million related to early-stage development programs licensed from two third parties.

Other Commitments

The Company uses experts and laboratories at universities and other institutions to perform certain R&D activities. These amounts are recorded as R&D expense as services are provided. In the normal course of business, the Company enters into various firm purchase commitments primarily to procure active pharmaceutical ingredients, certain inventory-related items and certain third-party R&D services, production services and facility construction services. As of September 30, 2023, such commitments were estimated at $246.0 million, of which $134.4 million is expected to be paid in 2023 as underlying goods and services are received. The Company has also licensed technology from third parties, for which it is required to pay royalties upon future sales, subject to certain annual minimums.

19

Item 2.    Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion of our financial condition and results of operations should be read in conjunction with our Condensed Consolidated Financial Statements and the related Notes thereto included in this Quarterly Report on Form 10-Q. This discussion contains forward-looking statements that involve risks and uncertainties. When reviewing the discussion below, you should keep in mind the substantial risks and uncertainties that could impact our business. In particular, we encourage you to review the risks and uncertainties described in “Risk Factors” in Part II, Item 1A in this Quarterly Report on Form 10-Q. These risks and uncertainties could cause actual results to differ significantly from those projected in forward-looking statements contained in this report or implied by past results and trends. Forward-looking statements are statements that attempt to forecast or anticipate future developments in our business, financial condition or results of operations. See the section titled “Forward-Looking Statements” that appears at the beginning of this Quarterly Report on Form 10-Q. These statements, like all statements in this report, speak only as of the date of this Quarterly Report on Form 10-Q (unless another date is indicated), and, except as required by law, we undertake no obligation to update or revise these statements in light of future developments. Our Condensed Consolidated Financial Statements have been prepared in accordance with United States (U.S.) generally accepted accounting principles (U.S. GAAP) and are presented in U.S. Dollars (USD).

20

Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions, except as otherwise disclosed)

Overview

Founded in 1997, we are a global biotechnology company dedicated to transforming lives through genetic discovery. We develop and commercialize targeted therapies that address the root cause of genetic conditions. Our robust research and development capabilities have resulted in multiple innovative commercial therapies for patients with rare genetic disorders. Our distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. A summary of our commercial products, as of September 30, 2023, is provided below:

Commercial Products	Indication
Enzyme products:
VIMIZIM (elosulfase alpha)	MPS (1) IVA
NAGLAZYME (galsulfase)	MPS VI (2)
PALYNZIQ (pegvaliase-pqpz)	PKU (3)
BRINEURA (cerliponase alfa)	CLN2 (4)
ALDURAZYME (laronidase)	MPS I (5)
Other products:
VOXZOGO (vosoritide) (6)	Achondroplasia
KUVAN (sapropterin dihydrochloride)	PKU
ROCTAVIAN (valoctocogene roxaparvovec) (7)	Severe Hemophilia A

(1)For the treatment of Mucopolysaccharidosis IV Type A

(2)For the treatment of MPS VI

(3)For the treatment of adult patients with phenylketonuria (PKU)

(4)For the treatment of neuronal ceroid lipofuscinosis type 2

(5)For the treatment of MPS I. ALDURAZYME is marketed worldwide by Sanofi

(6)For the treatment of achondroplasia in children of all ages for the U.S., aged four months and older for the European Union (EU) and for various age ranges for other markets.

(7)For the treatment of adults with severe hemophilia A. ROCTAVIAN was conditionally approved by the European Commission (EC) in August 2022 and approved by the Food and Drug Administration (FDA) in the U.S. in June 2023.

A summary of our on-going clinical development programs, as of September 30, 2023, is provided below:

Clinical Development Programs	Target Indication	Stage
BMN 255	Hyperoxaluria	Clinical Phase 1/2
BMN 331	Hereditary Angioedema (HAE)	Clinical Phase 1/2

21

Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions, except as otherwise disclosed)

Financial Highlights

Key components of our results of operations include the following:

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Total revenues	$	581.3	$	505.3	$	1,773.0	$	1,558.5
Cost of sales	$	124.7	$	116.3	$	379.4	$	356.4
Research and development (R&D) expense	$	191.3	$	157.8	$	540.5	$	476.9
Selling, general and administrative (SG&A) expense	$	223.9	$	216.8	$	662.3	$	608.3
Gain on sale of nonfinancial assets, net	$	—	$	—	$	—	$	(108.0)
Provision for income taxes	$	1.3	$	4.7	$	22.0	$	25.3
Net income (loss)	$	40.4	$	(6.7)	$	147.3	$	141.8

See “Results of Operations” below for discussion of our results for the periods presented.

Uncertainty Relating to Macroeconomic Environment

Conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, banking crises, natural disasters, the continuing effects of the COVID-19 pandemic, and supply chain disruptions, could impact our global revenue sources and our overall business operations. The extent and duration of such effects remain uncertain and difficult to predict. We are actively monitoring and managing our response and assessing actual and potential impacts to our operating results and financial condition, as well as developments in our business, which could further impact the developments, trends and expectations described below. See the risk factor, “Our business is affected by macroeconomic conditions.” described in “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q.

Business Developments

We continued to grow our commercial business and advance our product candidate pipeline during 2023. We believe that the combination of our internal research programs, and any acquisitions and partnerships, will allow us to continue to develop and commercialize innovative therapies for people with serious and life-threatening rare diseases and medical conditions. Below is a summary of key business developments:

Regulatory Approvals and Continued Emphasis on Research and Development

Product Launches, Approvals and Product Life Cycle Expansion Opportunities

•ROCTAVIAN: In June 2023, the FDA approved our gene therapy for the treatment of adults with severe hemophilia A. In the third quarter of 2023, we executed a number of critical steps to raise awareness among patients, physicians and patient advocate groups, as well as prepare Hemophilia Treatment Center (HTC) sites to treat patients. ROCTAVIAN was first conditionally approved by the EC in August 2022. We treated the first commercial patient with ROCTAVIAN in Germany in the third quarter of 2023, followed by a second commercial patient in November 2023. We continue to make progress on the pricing and reimbursement process in Germany and Italy to facilitate access.

We continue to explore ROCTAVIAN product expansion opportunities. These potential opportunities include a clinical study investigating ROCTAVIAN treatment in those with active or prior inhibitors and continued exploration of methods of administering ROCTAVIAN in people with pre-existing antibodies against AAV5.

•VOXZOGO: In October 2023, the FDA approved VOXZOGO for the treatment of achondroplasia in children of all ages with open growth plates in the U.S. and the EC adopted the decision to expand the indication for VOXZOGO to treat children with achondroplasia aged four-months and older with open growth plates.

In the near future, we plan to begin enrollment in the pivotal program with VOXZOGO for the treatment of children with hypochondroplasia, a genetic statural condition caused by a mutation (gene change) in the fibroblast growth factor receptor-3 (FGFR3) gene that is characterized by impaired bone growth. The six-month observation arm of the study will be followed by the 52-week randomized, double-blind, placebo-controlled phase of the 80-participant clinical trial. If successful, we believe this study will be able to support regulatory approval in this large indication.

We are also preparing to initiate two additional clinical programs in 2024 with VOXZOGO, one in idiopathic short

22

Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions, except as otherwise disclosed)

stature and one in genetic short stature conditions.

Select Earlier-stage Development Portfolio

•BMN 255 for hyperoxaluria in chronic liver disease: We believe the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in a patient population with significant unmet need. We expect to have a determination of clinical proof of concept in 2024.

•BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): Currently this product candidate is in the Phase 1/2 HAERMONY study to evaluate this investigational AAV5-mediated gene therapy for people living with HAE. We expect to dose additional patients with an optimized corticosteroid regimen with an anticipated determination of clinical proof of concept by 2025.

Critical Accounting Estimates

In preparing our Condensed Consolidated Financial Statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the Securities and Exchange Commission (the SEC), we make assumptions, judgments and estimates that can have a significant impact on our net income/loss and affect the reported amounts of certain assets, liabilities, revenues and expenses, and related disclosures. On an ongoing basis, we evaluate our estimates and discuss our critical accounting policies and estimates with the Audit Committee of our Board of Directors. We base our estimates on historical experience and various other assumptions that we believe to be reasonable under the circumstances. Actual results could differ materially from these estimates under different assumptions or conditions. Historically, our assumptions, judgments and estimates relative to our critical accounting estimates have not differed materially from actual results.

There have been no significant changes to our critical accounting estimates during the nine months ended September 30, 2023, compared to those disclosed in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

Recent Accounting Pronouncements

See Note 1 to our accompanying Condensed Consolidated Financial Statements for a description of recent accounting pronouncements, if any, and our expectation of their impact on our results of operations and financial condition.

Results of Operations

Net Product Revenues

Net Product Revenues consisted of the following:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Enzyme product revenues:
VIMIZIM	$	158.9	$	155.5	$	3.4	$	525.6	$	511.7	$	13.9
NAGLAZYME	108.9		99.5		9.4		322.0		343.3		(21.3)
PALYNZIQ	78.9		66.2		12.7		216.1		182.7		33.4
BRINEURA	41.0		37.8		3.2		118.2		111.7		6.5
ALDURAZYME	13.8		29.0		(15.2)		88.5		90.8		(2.3)
Total enzyme product revenues	$	401.5	$	388.0	$	13.5	$	1,270.4	$	1,240.2	$	30.2
VOXZOGO	123.1		48.3		74.8		324.2		102.3		221.9
KUVAN	42.9		57.0		(14.1)		144.0		174.0		(30.0)
ROCTAVIAN	0.8		—		0.8		0.8		—		0.8
Total net product revenues	$	568.3	$	493.3	$	75.0	$	1,739.4	$	1,516.5	$	222.9

    

Net Product Revenues include revenues generated from our commercial products. In the U.S., our commercial products, except for PALYNZIQ and ALDURAZYME, are generally sold to specialty pharmacies or end-users, such as hospitals, which act as

23

Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions of U.S. dollars, except as otherwise disclosed)

retailers. PALYNZIQ is distributed in the U.S. through certain certified specialty pharmacies under the PALYNZIQ Risk Evaluation and Mitigation Strategy (REMS) program, and ALDURAZYME is marketed worldwide by Sanofi. Outside the U.S., our commercial products are sold to authorized distributors or directly to government purchasers or hospitals, which act as the end users.

The increase in Net Product Revenues for the three months ended September 30, 2023 as compared to the three months ended September 30, 2022 was primarily attributed to the following:

•VOXZOGO: higher sales volume from new patients initiating therapy across all regions;

•PALYNZIQ: higher sales volume from new patients initiating therapy, particularly in the U.S.; partially offset by

•ALDURAZYME: lower sales volume due to timing of order fulfillment to Sanofi; and

•KUVAN: lower product revenues attributed to increasing generic competition as a result of the loss of exclusivity in the U.S.

The increase in Net Product Revenues for the nine months ended September 30, 2023 as compared to the nine months ended September 30, 2022 was primarily attributed to the following:

•VOXZOGO: higher sales volume from continued global market expansion and new patients initiating therapy across all regions;

•PALYNZIQ: higher sales volume from new patients initiating therapy, particularly in the U.S.; partially offset by

•KUVAN: lower product revenues primarily attributed to increasing generic competition as a result of the loss of exclusivity in the U.S.; and

•NAGLAZYME: lower sales volume primarily due to timing of orders in countries that place large government orders, particularly in Latin America, and the impact of foreign currency exchange rates.

In certain countries, governments place large periodic orders for our products. We expect that the timing of these large government orders will continue to be inconsistent, which may create significant period to period variation in our revenues.

Strong demand for VOXZOGO in certain markets has outpaced our projections in recent quarters, and we expect to face challenges meeting VOXZOGO demand during the remainder of 2023 and through the first half of 2024. These demand challenges will result in modest reduction of our revenue growth for VOXZOGO during the supply-constrained period. The projected temporary supply constraint could result in postponement of planned entry into additional markets or delayed clinical development activities until VOXZOGO inventory levels increase. We are working to increase fill-finish capacity to meet this increased demand while also implementing actions to manage growth and minimize patient impact. For example, we recently secured increased supply commitments beginning in mid-2024. We do not expect a material impact on our revenues if we successfully execute our manufacturing plans. See "Risk Factors" in Part II, Item 1A of this Quarterly Report for additional information on risk factors that could impact our business and operations.

See also the risk factor “The sale of generic versions of KUVAN by generic manufacturers has adversely affected and will continue to adversely affect our revenues and may cause a decline in KUVAN revenues faster than expected” included in Part II, Item 1A of this Quarterly Report for additional information on risks we face.

24

Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions of U.S. dollars, except as otherwise disclosed)

We face exposure to movements in foreign currency exchange rates, primarily the Euro, which we expect to continue in future periods. We use foreign currency exchange forward contracts to hedge a percentage of our foreign currency exposure. The following table shows our Net Product Revenues denominated in USD and foreign currencies:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Sales denominated in USD	$	257.9	$	239.2	$	18.7	$	830.6	$	739.8	$	90.8
Sales denominated in foreign currencies	310.4		254.1		56.3		908.8		776.7		132.1
Total net product revenues	$	568.3	$	493.3	$	75.0	$	1,739.4	$	1,516.5	$	222.9

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Unfavorable impact of foreign currency exchange rates on product sales denominated in currencies other than USD	$	(21.8)	$	(18.1)	$	(3.7)	$	(71.9)	$	(40.6)	$	(31.3)

The unfavorable impact for the three and nine months ended September 30, 2023 as compared to the three and nine months ended September 30, 2022 was primarily driven by weakening of Argentine Peso, Euro, Russian Ruble and Japanese Yen with the nine month comparative period also impacted by weakening of the Colombian Peso.

Royalty and Other Revenues

Royalty and Other Revenues include royalties earned on net sales of products sold by third parties, up-front licensing fees, milestones achieved by licensees or sublicensees and rental income associated with the tenants in our facilities.

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Royalty and other revenues	$	13.1	$	12.0	$	1.1	$	33.6	$	42.0	$	(8.4)

The increase in Royalty and Other Revenues for the three months ended September 30, 2023 as compared to the three months ended September 30, 2022 was primarily due to higher royalty revenues earned from third parties. The decrease in Royalty and Other Revenues for the nine months ended September 30, 2023 as compared to the nine months ended September 30, 2022 was primarily due to lower royalty revenues earned from third parties.

We expect to continue to earn royalties from third parties in the future.

Cost of Sales and Gross Margin

Cost of Sales includes raw materials, personnel and facility and other costs associated with manufacturing our commercial products. These costs include production materials, production costs at our manufacturing facilities, third-party manufacturing

25

Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions of U.S. dollars, except as otherwise disclosed)

costs, amortization of technology transfer intangible assets and internal and external final formulation and packaging costs. Cost of Sales also includes royalties payable to third parties based on sales of our products and charges for inventory valuation reserves.

The following table summarizes our Cost of Sales and gross margin:

	Three Months Ended September 30,									Nine Months Ended September 30,
	2023			2022			Change			2023			2022			Change
Total revenues	$	581.3		$	505.3		$	76.0		$	1,773.0		$	1,558.5		$	214.5
Cost of sales	$	124.7		$	116.3		$	8.4		$	379.4		$	356.4		$	23.0
Gross margin	78.5		%	77.0		%	1.5		%	78.6		%	77.1		%	1.5		%

Cost of Sales increased for the three and nine months ended September 30, 2023 as compared to the three and nine months ended September 30, 2022, primarily due to increased sales volumes as noted above. Gross margin increased for the three months ended September 30, 2023 as compared to the three months ended September 30, 2022 primarily due to the increased sales volumes for products with higher margin and lower per unit manufacturing costs for our enzyme products, partially offset by higher inventory reserves. Gross margin increased for the nine months ended September 30, 2023 compared to the same period in 2022 primarily due to the increased sales volumes for products with higher margin.

We expect gross margin to range between approximately 77.8% and 79.0% through the remainder of 2023.

Research and Development

R&D expense includes costs associated with the research and development of product candidates and post-marketing research commitments related to our commercial products. R&D expense primarily includes preclinical and clinical studies, personnel and raw materials costs associated with manufacturing clinical product, quality control and assurance, other R&D activities, R&D facilities and regulatory costs.

We manage our R&D expense by identifying the R&D activities we anticipate will be performed during a given period and then prioritizing efforts based on scientific data, probability of successful development, market potential, available human and capital resources and other similar considerations. We continually review our product pipeline and the development status of product candidates and, as necessary, reallocate resources among the research and development portfolio that we believe will best support the future growth of our business.

R&D expense consisted of the following:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Research and early development	$	84.1	$	62.6	$	21.5	$	223.3	$	188.3	$	35.0
Other commercial products	34.3		29.4		4.9		95.1		86.4		8.7
ROCTAVIAN	32.4		29.0		3.4		95.0		90.7		4.3
VOXZOGO	20.6		22.5		(1.9)		70.0		76.9		(6.9)
BMN 331	10.1		8.4		1.7		28.7		21.9		6.8
BMN 255	5.6		2.3		3.3		12.3		6.8		5.5
Other	4.2		3.6		0.6		16.1		5.9		10.2
Total R&D expense	$	191.3	$	157.8	$	33.5	$	540.5	$	476.9	$	63.6

The increase in R&D expense for the three and nine months ended September 30, 2023 as compared to the three and nine months ended September 30, 2022 was primarily due to higher spend in research and early development programs due to increased pre-clinical activities, including studies for planned Investigational New Drug (IND) application submissions, higher spend on activities related to our commercial and clinical products and an increase in other R&D costs primarily related to incremental compensation expense due to the increase in fair value of the plan assets held in our nonqualified deferred compensation plan. Increases were partially offset by a reduction in clinical activities related to VOXZOGO’s commercial indications.

26

Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions of U.S. dollars, except as otherwise disclosed)

We expect R&D expense to increase in future periods, primarily due to increased activities for our research and early development programs and clinical programs.

Selling, General and Administrative

Sales and marketing (S&M) expense primarily consisted of employee-related expenses for our sales group, brand marketing, patient support groups and pre-commercialization expenses related to our product candidates. General and administrative (G&A) expense primarily consisted of corporate support and other administrative expenses, including employee-related expenses.

SG&A expense consisted of the following:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
S&M expense	$	121.9	$	111.3	$	10.6	$	355.2	$	325.7	$	29.5
G&A expense	102.1		105.5		(3.4)		307.1		282.6		24.5
Total SG&A expense	$	224.0	$	216.8	$	7.2	$	662.3	$	608.3	$	54.0

S&M expense consisted of the following:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Enzyme Products	54.9		54.1		0.8		$	167.6	$	165.3	$	2.3
VOXZOGO	27.4		26.5		0.9		78.4		72.5		5.9
ROCTAVIAN	27.2		18.4		8.8		74.6		52.6		22.0
Other	12.4		12.3		0.1		34.6		35.3		(0.7)
Total S&M expense	$	121.9	$	111.3	$	10.6	$	355.2	$	325.7	$	29.5

The increase in S&M expense for the three and nine months ended September 30, 2023 as compared to the three and nine months ended September 30, 2022 was primarily a result of increased activities in support of the European and U.S. launch of ROCTAVIAN and, to a lesser extent, activities in support of the continued VOXZOGO global expansion.    

The decrease in G&A expense for the three months ended September 30, 2023 as compared to the three months ended September 30, 2022 was primarily due to lower foreign currency losses, partially offset by increased costs related to strategic initiatives. The increase in G&A expense for the nine months ended September 30, 2023 as compared to the nine months ended September 30, 2022 was primarily due to increased costs related to strategic initiatives and unfavorable fluctuations of unhedged currencies.

We expect SG&A expense to increase in future periods as we prepare to launch new products and support the expansion of our global brands.

27

Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions of U.S. dollars, except as otherwise disclosed)

Intangible Asset Amortization and Contingent Consideration and Gain on Sale of Nonfinancial Assets, Net

Changes during the periods presented for Intangible Asset Amortization and Contingent Consideration and Gain on Sale of Nonfinancial Assets, Net were as follows:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Amortization of intangible assets	$	15.7	$	15.9	$	(0.2)	$	47.0	$	47.1	$	(0.1)
Changes in the fair value of contingent consideration	—		0.9		(0.9)		—		3.8		(3.8)
Total intangible asset amortization and contingent consideration	$	15.7	$	16.8	$	(1.1)	$	47.0	$	50.9	$	(3.9)
Gain on sale of nonfinancial assets, net	$	—	$	—	$	—	$	—	$	108.0	$	(108.0)

Amortization of intangible assets – the expense for the three and nine months ended September 30, 2023 as compared to the three and nine months ended September 30, 2022 was relatively flat.

Fair value of contingent consideration – the decrease in the fair value of contingent consideration for the three and nine months ended September 30, 2023 as compared to the three and nine months ended September 30, 2022 was attributable to attainment of the final commercial milestone in the fourth quarter of 2022.

Gain on Sale of Nonfinancial Assets, Net – the decrease in the nine months ended September 30, 2023 as compared to the nine months ended September 30, 2022 was due to the sale in the first quarter of 2022 of a Priority Review Voucher (PRV) with no similar transaction in 2023.

Interest Income

We invest our cash equivalents and investments in U.S. government securities and other high credit quality debt securities in order to limit default and market risk.

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Interest income	$	15.7	$	5.0	$	10.7	$	40.3	$	9.3	$	31.0

The increase in Interest Income for the three and nine months ended September 30, 2023 compared to the three and nine months ended September 30, 2022 was primarily due to higher yields on our investment portfolio. We do not expect Interest Income to fluctuate significantly over the next 12 months due to anticipated interest rates and yields on our cash equivalents and investments.

Interest Expense

We incur interest expense primarily on our convertible debt. Interest Expense for the periods presented was as follows:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Interest expense	$	3.8	$	4.7	$	(0.9)	$	11.2	$	12.3	$	(1.1)

Interest Expense for the three and nine months ended September 30, 2023 as compared to the three and nine months ended September 30, 2022 was relatively flat. We do not expect Interest Expense to fluctuate significantly over the next 12 months as the rates on our convertible debt are fixed. See Note 6 to our accompanying Condensed Consolidated Financial Statements for additional information regarding our debt.

28

Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions of U.S. dollars, except as otherwise disclosed)

Other Income (Expense), Net

Other Income (Expense), Net for the periods presented was as follows:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Other income (expense), net	$	4.0	$	0.2	$	3.8	$	(3.7)	$	(3.9)	$	0.2

The increase in Other Income (Expense), Net for the three months ended September 30, 2023 compared to the three months ended September 30, 2022 was primarily due to a gain related to a fiscal 2022 refundable tax credit. The change in Other Income (Expense), Net for the nine months ended September 30, 2023 compared to the nine months ended September 30, 2022 was relatively flat.

Provision for Income Taxes

The Provision for Income Taxes for the periods presented was as follows:

	Three Months Ended September 30,						Nine Months Ended September 30,
	2023		2022		Change		2023		2022		Change
Provision for income taxes	$	1.3	$	4.7	$	(3.4)	$	22.0	$	25.3	$	(3.3)

The decrease in Provision for Income Taxes for the three months ended September 30, 2023 as compared to the three months ended September 30, 2022 was primarily due to a discrete benefit for a valuation allowance release partially offset by higher pre-tax income in the third quarter of 2023 compared to the third quarter of 2022. In the third quarter of 2023, we determined that it is more likely than not that the deferred tax assets related to a future royalty stream will be realized. In making this determination, we analyzed both the consistent historical royalty earnings and the forecast of future royalty earnings and reached the conclusion that it was appropriate to release the valuation allowance reserve. The decrease in the nine months ended September 30, 2023 compared to the nine months ended September 30, 2022 was primarily related to a discrete benefit for the valuation allowance release in the third quarter of 2023, partially offset by an increase in the effective tax rate.

Financial Condition, Liquidity and Capital Resources

Our cash, cash equivalents, and investments as of September 30, 2023 and December 31, 2022 were as follows:

	September 30, 2023		December 31, 2022		Change
Cash and cash equivalents	$	785.4	$	724.5	$	60.9
Short-term investments	340.4		567.0		(226.6)
Long-term investments	548.0		333.9		214.1
Cash, cash equivalents and investments	$	1,673.8	$	1,625.4	$	48.4

We believe our cash generated from sales of our commercial products, in addition to our cash, cash equivalents and short-term investments will be sufficient to satisfy our liquidity requirements for at least the next 12 months. We believe we will meet longer-term expected future cash requirements and obligations through a combination of cash flows from operating activities and available cash and long-term investment balances. We will need to raise additional funds by issuing equity, debt or convertible securities, taking loans or entering into collaborative or other agreements if we are unable to satisfy our liquidity requirements. For example, we may require additional financing to fund the repayment of our convertible debt, future milestone payments and our future operations, including the commercialization of our products and product candidates currently under development, preclinical studies and clinical trials, and potential licenses and acquisitions. The timing and mix of our funding alternatives could change depending on many factors, including how much we elect to spend on our development programs, potential licenses and acquisitions of complementary technologies, products and companies or if we settle our convertible debt in cash.

We are mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, banking crises, natural disasters, the continuing effects of the COVID-19 pandemic, and supply

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Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions of U.S. dollars, except as otherwise disclosed)

chain disruptions could affect our ability to achieve our goals. In addition, we sell our products in certain countries that face economic volatility and weakness. Although we have historically collected receivables from customers in such countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to be unable to pay for our products. We will continue to monitor these conditions and will attempt to adjust our business processes, as appropriate, to mitigate macroeconomic risks to our business.

Our cash flows are summarized as follows:

	Nine Months Ended September 30,
	2023		2022		Change
Net cash provided by operating activities	$	131.8	$	169.1	$	(37.3)
Net cash provided by (used in) investing activities	$	(46.3)	$	34.7	$	(81.0)
Net cash used in financing activities	$	(29.6)	$	(29.5)	$	(0.1)

The decrease in net cash provided by operating activities in the nine months ended September 30, 2023 compared to September 30, 2022 was primarily attributed to the timing of cash receipts from our customers and increased payments for inventory purchases, partially offset by the timing of payments to vendors and payments of income taxes.

The increase in net cash used in investing activities in the nine months ended September 30, 2023 compared to September 30, 2022 was primarily attributable to the absence of $110.0 million proceeds from the sale of the PRV in the first quarter of 2022, partially offset by a decrease in purchases of fixed assets, lower payments related to purchases of intangible assets and higher maturities of available-for-sale debt securities net of purchases.

The change in net cash used in financing activities in the nine months ended September 30, 2023 compared to September 30, 2022 was flat but was comprised of higher taxes paid for net settlement of shares under our equity incentive plans offset by a decrease in milestone payments to a third party related to PKU sales milestones achieved in 2022 and increased proceeds from stock option exercises.

Financing and Credit Facilities

Our $1.1 billion (undiscounted) of total convertible debt as of September 30, 2023 will impact our liquidity due to the semi-annual cash interest payments as well as the repayment of the principal amount, if not converted. As of September 30, 2023, our indebtedness consisted of our 0.599% senior subordinated convertible notes due in 2024 and our 1.25% senior subordinated convertible notes due in 2027, which, if not converted, will be required to be repaid in cash at maturity in August 2024 and May 2027, respectively. For additional information related to our convertible debt, see Note 6 to our accompanying Condensed Consolidated Financial Statements and Note 10 - Debt to the Consolidated Financial Statements accompanying our Annual Report on Form 10-K for the year ended December 31, 2022. We have reclassified all of the outstanding principal of the 2024 Notes as a current liability as there are less than twelve months remaining until maturity.

In October 2018, we entered into an unsecured revolving credit facility of up to $200.0 million which included a letter of credit subfacility and a swingline loan subfacility. The credit facility was intended to finance ongoing working capital needs and for other general corporate purposes. In May 2021, the credit facility was amended to extend the original maturity date from October

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Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

(In millions of U.S. dollars, except as otherwise disclosed)

19, 2021 to May 28, 2024. The credit facility was terminated on August 4, 2023 and there were no amounts outstanding under the terminated credit facility as of September 30, 2023.

Material Cash Requirements

Funding Commitments

Our investment in our research and early development of product candidates and continued development of our existing commercial products has a major impact on our operating performance. R&D expenses for our commercial products and certain product candidates for the period since inception as of September 30, 2023 were as follows:

	Since Program Inception
ROCTAVIAN	$	1,039.1
VOXZOGO	$	871.0
BMN 331	$	138.6
BMN 255	$	47.2
Other commercial products	$	2,584.1

We cannot estimate with certainty the cost to complete any of our product development programs. We may need or elect to increase our spending above our current long-term plans to be able to achieve our long-term goals. This may increase our capital requirements, including: costs associated with the commercialization of our products; additional clinical trials; investments in the manufacturing of our commercial products; preclinical studies and clinical trials for our product candidates; potential licenses and other acquisitions of complementary technologies, products and companies; and general corporate purposes. Additionally, we cannot precisely estimate the time to complete any of our product development programs or when we expect to receive net cash inflows from any of our product development programs. Please see “Risk Factors” included in Part II, Item 1A of this Quarterly Report on Form 10-Q, for a discussion of the reasons we are unable to estimate such information.

Purchase Obligations

As of September 30, 2023, we had obligations of approximately $246.0 million, which primarily related to firm purchase commitments entered into in the normal course of business to procure active pharmaceutical ingredients, certain inventory-related items, certain third-party R&D services, production services and facility construction services. Of this amount, $134.4 million is expected to be paid in 2023. The remainder of the estimated obligation is expected to be paid in the following year as underlying goods and services are received.

Other Obligations

As of September 30, 2023, we expect to pay $39.0 million related to a litigation-related loss contingency, which was included in Accounts Payable and Accrued Liabilities and the same amount for expected insurance recoveries was included in Other Current Assets on our Condensed Consolidated Balance Sheets. We do not expect these anticipated payments and receipts to affect our liquidity.

Our lease, contingent obligations and unrecognized tax benefits as of September 30, 2023 have not materially changed from those discussed in “Financial Condition, Liquidity and Capital Resources” in Part II, Item 7 of our Annual Report on Form 10-K for the year ended December 31, 2022.

See Note 11 to our accompanying Condensed Consolidated Financial Statements for additional information on our commitments.

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Item 3.    Quantitative and Qualitative Disclosures About Market Risk

Our market risks during the nine months ended September 30, 2023 have not materially changed from those discussed in Part II, Item 7A of our Annual Report on Form 10-K for the year ended December 31, 2022.

Item 4.    Controls and Procedures

(a) Controls and Procedures

An evaluation was carried out, under the supervision of and with the participation of our management, including our Chief Executive Officer and our Chief Financial Officer, of the effectiveness of our disclosure controls and procedures (as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the Exchange Act)), as of the end of the period covered by this report.

Based on the evaluation, our Chief Executive Officer and our Chief Financial Officer have concluded that our disclosure controls and procedures were effective, at the reasonable assurance level, as of September 30, 2023.

In designing and evaluating our disclosure controls and procedures, our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objectives, and our management must apply its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Accordingly, our disclosure controls and procedures are designed to provide reasonable, not absolute, assurance that the objectives of our disclosure controls system are met.

(b) Change in Internal Control over Financial Reporting

There were no changes in our internal control over financial reporting, as such term is defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act, during our most recently completed quarter that have materially affected or are reasonably likely to materially affect our internal control over financial reporting. We continue to utilize the Committee of Sponsoring Organizations of the Treadway Commission (COSO) 2013 Framework on internal control.

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PART II. OTHER INFORMATION

Item 1.    Legal Proceedings

On September 25, 2020, a purported shareholder class action lawsuit was filed against us, our Chief Executive Officer, our President of Worldwide Research and Development and our Chief Financial Officer in the United States District Court in the Northern District of California, alleging violations under Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 as amended (the Exchange Act). The complaint alleges that we made materially false or misleading statements regarding the clinical trials and Biologics License Application (BLA) for ROCTAVIAN (formerly known as valoctocogene roxaparvovec) by purportedly failing to disclose that differences between the Company’s Phase 1/2 and Phase 3 clinical studies limited the ability of the Phase 1/2 study to support ROCTAVIAN’s durability of effect and, as a result, that it was foreseeable that the Food and Drug Administration (FDA) would not approve the BLA without additional data. The complaint seeks an unspecified amount of damages, prejudgment and post-judgment interest, attorneys’ fees, expert fees, and other costs. The lead plaintiff filed an amended complaint in February 2021, dropping our Chief Financial Officer as a defendant, and asserting that the Company misled investors about the progress of the FDA's review of our BLA for ROCTAVIAN. On April 22, 2021, we moved to dismiss the amended complaint. On January 6, 2022, the court denied our motion to dismiss. We answered the amended complaint on February 15, 2022. Plaintiff filed a motion for class certification on October 17, 2022. We filed an opposition to plaintiff’s motion for class certification on January 27, 2023. On March 21, 2023, the Court entered an order staying all proceedings and vacating all deadlines because the parties agreed to settle the case through a binding term sheet. The Court preliminarily approved the settlement on June 8, 2023. On October 4, 2023, plaintiff filed a motion for final approval of settlement. A final approval hearing is scheduled for November 8, 2023.

On October 22, 2021, a purported securities class action lawsuit was filed against us, our Chief Executive Officer, our current and prior Chief Financial Officers, and our President of Worldwide Research & Development in the United States District Court for the Northern District of California, alleging violations under Sections 10(b) and 20(a) of the Exchange Act. The complaint alleges that we made materially false or misleading statements regarding BMN 307 by purportedly failing to disclose information about BMN 307’s safety profile, and by purportedly overstating BMN 307’s clinical and commercial prospects. The complaint seeks an unspecified amount of damages, pre-judgment and post-judgment interest, attorneys’ fees, expert fees, and other costs. The Court appointed lead plaintiffs and lead counsel on January 10, 2022. Lead plaintiffs filed an amended complaint on March 25, 2022. We filed a motion to dismiss the amended complaint on May 25, 2022. On January 19, 2023, the Court granted our motion to dismiss the complaint without prejudice. On February 21, 2023, the court dismissed the complaint with prejudice at plaintiffs’ request. Plaintiffs have appealed the court’s January 19, 2023 order to the United State Court of Appeals for the Ninth Circuit and filed their opening brief on June 23, 2023. Defendants filed their answering brief on August 23, 2023. Plaintiffs filed their reply brief on October 13, 2023. We believe that the claims have no merit and we intend to vigorously defend this action.

On January 19, 2023 and May 30, 2023, certain of our officers and directors were named as defendants in two shareholder derivative actions filed in the Delaware Court of Chancery. The complaints assert, inter alia, breach of fiduciary duty claims arising from the facts underlying the securities class action related to ROCTAVIAN. The complaints seek unspecified monetary damages, internal governance reforms by the Company, attorneys fees and costs, and any other relief the court may deem just and proper. We believe that the claims in both cases have no merit and we intend to vigorously defend these actions.

Item 1A.     Risk Factors

An investment in our securities involves a high degree of risk. We operate in a dynamic and rapidly changing industry that involves numerous risks and uncertainties. The risks and uncertainties described below are not the only ones we face. Other risks and uncertainties, including those that we do not currently consider material, may impair our business. If any of the risks discussed below actually occur, our business, financial condition, operating results or cash flows could be materially adversely affected. This could cause the value of our securities to decline, and you may lose all or part of your investment.

We have marked with an asterisk (*) those risk factors below that include a substantive change from or update to the risk factors included in our Annual Report on Form 10-K for the year ended December 31, 2022, which was filed with the SEC on February 27, 2023.

Business and Operational Risks

If we fail to obtain and maintain an adequate level of coverage and reimbursement for our products by third-party payers, the sales of our products would be adversely affected or there may be no commercially viable markets for our products.

The course of treatment for patients using our products is expensive. For all our products except ROCTAVIAN, we expect patients to need treatment for extended periods, and for some products throughout the lifetimes of the patients. We expect that most families of patients will not be capable of paying for this treatment themselves. There will be no commercially viable market for

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our products without coverage and reimbursement from third-party payers. Additionally, even if there is a commercially viable market, if the level of reimbursement is below our expectations, our revenues and gross margin will be adversely affected.

Third-party payers, such as government or private healthcare insurers, carefully review and increasingly challenge the prices charged for drugs. Reimbursement rates from private companies vary depending on the third-party payer, the insurance plan and other factors. Obtaining coverage and adequate reimbursement for our products may be particularly difficult because of the higher prices often associated with drugs administered under the supervision of a physician. Reimbursement systems in international markets vary significantly by country and by region, and reimbursement approvals must be obtained on a country-by-country basis.

Government authorities and other third-party payers are developing increasingly sophisticated methods of controlling healthcare costs, such as by limiting coverage and the amount of reimbursement for particular medications. Increasingly, third-party payers are requiring that drug companies provide them with predetermined discounts from list prices as a condition of coverage, are using restrictive formularies and preferred drug lists to leverage greater discounts in competitive classes, and are challenging the prices charged for medical products. Further, no uniform policy requirement for coverage and reimbursement for drug products exists among third-party payers in the U.S. Therefore, coverage and reimbursement for drug products can differ significantly from payer to payer. As a result, the coverage determination process is often a time-consuming and costly process that will require us to provide scientific and clinical support for the use of our products to each payer separately, with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance.

We cannot be sure that coverage and reimbursement will be available for any product that we commercialize or will continue to be available for any product that we have commercialized and, if reimbursement is available, what the level of reimbursement will be. Even if favorable coverage and reimbursement status is attained for one or more products for which we receive regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future. Coverage and reimbursement may impact the demand for, or the price of, any product candidate for which we obtain marketing approval. If coverage and reimbursement are not available or reimbursement is available only to limited levels, we may not successfully commercialize any product candidate for which we obtain marketing approval or continue to market any product that has already been commercialized.

Reimbursement in the European Union (EU) and many other territories must be negotiated on a country-by-country basis and in many countries the product cannot be commercially launched until pricing and/or reimbursement is approved. The timing to complete the negotiation process in each country is highly uncertain, and in some countries, we expect that it will exceed 12 months. Even after a price is negotiated, countries frequently request or require reductions to the price and other concessions over time.

For our future products, we will not know what the reimbursement rates will be until we are ready to market the product and we actually negotiate the rates. If we are unable to obtain sufficiently high reimbursement rates for our products, they may not be commercially viable or our future revenues and gross margin may be adversely affected.

Because the target patient populations for our products are relatively small, we must achieve significant market share and maintain high per-patient prices for our products to achieve and maintain profitability.

All of our products target diseases with relatively small patient populations. Our two newest products, VOXZOGO and ROCTAVIAN, address potentially larger patient populations than most of our other products; however, their market sizes are considerably smaller than many drugs marketed by other pharmaceutical and biotechnology companies. As a result, our per-patient prices must be relatively high in order to recover our development and manufacturing costs and achieve and maintain profitability. For BRINEURA, NAGLAZYME and VIMIZIM in particular, we must market worldwide to achieve significant market penetration of the product. In addition, because the number of potential patients in each disease population is small, it is not only important to find patients who begin therapy to achieve significant market penetration of the product, but we also need to be able to maintain these patients on therapy for an extended period of time. Due to the expected costs of treatment for our products, we may be unable to maintain or obtain sufficient market share at a price high enough to justify our product development efforts and manufacturing expenses.

*If we fail to compete successfully with respect to product sales, we may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product and our revenues could be adversely affected.

Our competitors may develop, manufacture and market products that are more effective or less expensive than ours. They may also obtain regulatory approvals for their products faster than we can obtain them (including those products with orphan drug designation, which may prevent us from marketing our product entirely) or commercialize their products before we do. With respect to ROCTAVIAN, we face a highly developed and competitive market for hemophilia A treatments. As we commercialize ROCTAVIAN, we may face intense competition from large pharmaceutical companies with extensive resources and established relationships in the hemophilia A community. If we do not compete successfully, our revenues would be adversely affected, and we

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may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product.

Changes in methods of treatment of disease could reduce demand for our products and adversely affect revenues.

Even if our product candidates are approved, if doctors elect a course of treatment which does not include our products, this decision would reduce demand for our products and adversely affect revenues. For example, if gene therapy becomes widely used as a treatment of genetic diseases, the use of enzyme replacement therapy, such as ALDURAZYME, NAGLAZYME, and VIMIZIM in MPS diseases, could be greatly reduced. Changes in treatment method can be caused by the introduction of other companies’ products or the development of new technologies or surgical procedures which may not directly compete with ours, but which have the effect of changing how doctors decide to treat a disease.

*If we fail to develop new products and product candidates or compete successfully with respect to acquisitions, joint ventures, licenses or other collaboration opportunities, our ability to continue to expand our product pipeline and our growth and development would be impaired.

Our future growth and development depend in part on our ability to successfully develop new products from our research and development activities. The development of biopharmaceutical products is very expensive and time intensive and involves a great degree of risk. The outcomes of research and development programs, especially for innovative biopharmaceuticals, are inherently uncertain and may not result in the commercialization of any products.

Our competitors compete with us to attract organizations for acquisitions, joint ventures, licensing arrangements or other collaborations. To date, several of our former and current product programs have been acquired through acquisitions and several of our former and current product programs have been developed through licensing or collaborative arrangements, such as ALDURAZYME, KUVAN and NAGLAZYME. These collaborations include licensing proprietary technology from, and other relationships with, academic research institutions. Our future success will depend, in part, on our ability to identify additional opportunities and to successfully enter into partnering or acquisition agreements for those opportunities. If our competitors successfully enter into partnering arrangements or license agreements with academic research institutions, we will then be precluded from pursuing those specific opportunities. Because each of these opportunities is unique, we may not be able to find a substitute. Several pharmaceutical and biotechnology companies have already established themselves in the field of genetic diseases. These companies have already begun many drug development programs, some of which target diseases that we are also targeting or may target in the future, and have already entered into partnering and licensing arrangements with academic research institutions, reducing the pool of available opportunities.

Universities and public and private research institutions also compete with us. While these organizations primarily have educational or basic research objectives, they may develop proprietary technology and acquire patents that we may need for the development of our product candidates. We will attempt to license this proprietary technology, if available. These licenses may not be available to us on acceptable terms, if at all. If we are unable to compete successfully with respect to acquisitions, joint venture and other collaboration opportunities, we may be limited in our ability to develop new products and to continue to expand our product pipeline.

*The sale of generic versions of KUVAN by generic manufacturers has adversely affected and will continue to adversely affect our revenues and may cause a decline in KUVAN revenues faster than expected.

Generic versions of KUVAN are available in several countries around the world, including multiple generic versions in the U.S. This generic competition has adversely affected and will continue to adversely affect our revenues from KUVAN, and we cannot accurately predict the rate of decline of KUVAN revenues in these countries. We are also aware that manufacturers are challenging our patent portfolio related to KUVAN in several jurisdictions, and several generic versions of KUVAN have been approved either centrally by the European Commission (EC) or on a country-by-country basis throughout the EU. If these patent challenges are successful, or if a manufacturer chooses to offer a generic version of KUVAN, notwithstanding our existing patents, our revenues from KUVAN may decline faster than expected.

If we do not achieve our projected development goals in the timeframes we announce or fail to achieve such goals, the commercialization of our product candidates may be delayed or never occur and the credibility of our management may be adversely affected and, as a result, our stock price may decline.

For planning purposes, we estimate the timing of the accomplishment of various scientific, clinical, regulatory and other product development goals, which we sometimes refer to as milestones. These milestones may include the commencement or completion of scientific studies and clinical trials and the submission of regulatory filings. From time to time, we publicly announce the expected timing of some of these milestones. All of these milestones are based on a variety of assumptions. The actual timing of these milestones can vary dramatically compared to our estimates or the milestones may never be achieved, in many cases for reasons beyond our control. For example, in 2021 and early 2022, we announced that we planned to resubmit our Biologics

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License Application (BLA) for ROCTAVIAN to the Food and Drug Administration (FDA) in the first half of 2022; however, we did not file the BLA until the third quarter of 2022 due to the additional time we needed to include supplemental information and analyses of data requested by the FDA. If we do not meet development milestones as publicly announced, the commercialization of our products may be delayed or never occur and the credibility of our management may be adversely affected and, as a result, our stock price may decline.

We have in the past and may in the future enter into licensing arrangements, and we may not realize the benefits of such licensing arrangements.

We have in the past and may in the future enter into licensing arrangements with third parties. It is possible that we may not achieve financial or strategic benefits that justify a specific license, or we may otherwise not realize the benefits of such licensing arrangement. Further, licensing arrangements impose various diligence, milestone and royalty payment and other obligations on us. If we fail to comply with our obligations under any current or future licenses, our licensors may have the right to terminate these license agreements, which could harm our business prospects, financial condition and results of operations. Additionally, counterparties to our license agreements have in the past alleged and may in the future allege that we have breached a license agreement, which can result in litigation or other disputes that can divert management’s attention away from our business and require us to expend resources, as well as potentially having to negotiate new or reinstated licenses with less favorable terms. Any such situation could adversely affect our business, financial condition, and results of operations.

Regulatory Risks

*If we fail to obtain regulatory approval to commercially market and sell our product candidates, or if approval of our product candidates is delayed, we will be unable to generate revenues from the sale of these product candidates, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will increase.

We must obtain regulatory approval to market and sell our product candidates. For example, in the U.S., we must obtain FDA approval for each product candidate that we intend to commercialize, and in the EU, we must obtain approval from the EC, based on the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The FDA and EC approval processes are typically lengthy and expensive, and approval is never certain. To obtain regulatory approval, we must first show that our product candidates are safe and effective for target indications through preclinical studies and clinical trials. Preclinical studies and clinical development are long, expensive and uncertain processes. Completion of clinical trials may take several years, and failure may occur at any stage of development. The length of time required varies substantially according to the type, complexity, novelty and intended use of a product candidate. Interim results of a preclinical test or clinical trial do not necessarily predict final results, and acceptable results in early clinical trials may not be repeated in later clinical trials. Accordingly, there are no assurances that we will obtain regulatory approval for any of our product candidates. Furthermore, there can be no assurance that approval of one of our product candidates by one regulatory authority will mean that other authorities will also approve the same product candidate. Similarly, in the EU, a positive CHMP opinion for approval of a product candidate does not guarantee that the EC will approve the product candidate. Moreover, regulatory authorities may approve a product candidate for fewer or more limited indications than requested. In addition, regulatory authorities may not approve the labeling claims that are necessary or desirable for the successful commercialization of our product candidates.

We have had fewer interactions with regulatory authorities outside the U.S. and the EU as compared to our interactions with the FDA, the EC and the EMA. The approval procedures vary among countries and can involve additional clinical testing, and the time required to obtain approval may differ from that required to obtain FDA or EC approval. Moreover, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries. Approval by the FDA or EC does not ensure approval by regulatory authorities in other countries, and approval by one or more non-U.S. regulatory authorities does not ensure approval by regulatory authorities in other non-U.S. countries or by the FDA or EC. However, a failure or delay in obtaining regulatory approval in one country may have a negative effect on the regulatory process in others. The non-U.S. regulatory approval process may include all of the risks associated with obtaining FDA or EC approval. We may not obtain non-U.S. regulatory approvals on a timely basis, if at all. We may not be able to file for regulatory approvals and even if we file, we may not receive necessary approvals to commercialize our product candidates in any market.

We also rely on independent third-party Contract Research Organizations (CROs) to file some of our non-U.S. marketing applications, and while we keep a close oversight on the activities we delegate to CROs, important aspects of the services performed for us by the CROs are out of our direct control. If we fail to adequately manage our CROs, if the CRO elects to prioritize work on our projects below other projects or if there is any dispute or disruption in our relationship with our CROs, the filing of our applications may be delayed.

Although the FDA, the EC and the EMA have programs to facilitate expedited development and accelerated approval processes, the timelines agreed under legislative goals or mandated by regulations are subject to the possibility of substantial delays. Accordingly, even if any of our applications receives a designation to facilitate expedited development and accelerated

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approval processes, these designations may not result in faster review or approval for our product candidates compared to product candidates considered for approval under conventional procedures and, in any event, do not assure ultimate approval of our product candidates by regulatory authorities. In addition, the FDA, the EC, the EMA and other comparable international regulatory authorities have substantial discretion over the approval process for pharmaceutical products. These regulatory authorities may not agree that we have demonstrated the requisite level of product safety and efficacy to warrant approval and may require, and in the past have required, additional data. If we fail to obtain regulatory approval for our product candidates, we will be unable to market and sell those product candidates, which would have a negative effect on our business and financial condition.

Regulatory authorities and the new requirements and guidelines they promulgate may lengthen the regulatory review process, require us to perform additional or larger studies, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of our treatment candidate or lead to significant post-approval studies, limitations or restrictions. For example, on August 18, 2020, the FDA issued a Complete Response Letter (CRL) to our BLA for ROCTAVIAN for the treatment of adults with severe hemophilia A. In the CRL, the FDA introduced a new request for two-year follow-up safety and efficacy data on all study participants from our ongoing Phase 3 study of ROCTAVIAN. In January 2022, we announced results from the requested two-year data analysis from our Phase 3 study. In the third quarter of 2022, we resubmitted our BLA, and the FDA subsequently accepted our submission with an original Prescription Drug User Fee Act (PDUFA) target action date of March 31, 2023. In early 2023, we supplemented our BLA by submitting our three-year analysis of the global Phase 3 study of ROCTAVIAN, which the FDA deemed to be a Major Amendment to our BLA due to the substantial amount of additional data, and extended the PDUFA target action date by three months. The FDA approved ROCTAVIAN for the treatment of adults with severe hemophilia A on June 29, 2023.

In addition, some of our product candidates are intended to be used in combination with a medical device, such as an injector or other delivery system or companion diagnostic. For example, ROCTAVIAN is approved with a companion diagnostic test intended to detect pre-existing anti-AAV5 antibodies, which may render the gene therapy less effective or ineffective. Some of these products intended to be used with a medical device may be regulated as “combination products” in the U.S. and the EU, which are generally defined as products consisting of components from two or more regulatory categories (e.g., drug/device, device/biologic, drug/biologic). In the U.S., each component of a combination product is subject to the requirements established by the FDA for that type of component, whether a new drug, biologic or device. In order to facilitate pre-market review of combination products, the FDA designates one of its centers to have primary jurisdiction for the pre-market review and regulation of the overall product based upon a determination by the FDA of the primary mode of action of the combination product. The determination whether a product is a combination product or two separately regulated products is made by the FDA on a case-by-case basis. In the EU, medical devices and medicinal products are regulated separately, through different legislative instruments. The related applicable requirements will vary depending on the type of drug-device combination product. If, for example, a device intended to administer a medicinal product is sold together with such medicinal product in such a way that they form a single integral product which is intended exclusively for use in the given combination and which is not reusable, that single integral product is regulated as a medicinal product. In addition, the relevant general safety and performance requirements (GSPRs) established for medical devices by EU medical devices legislation apply to the device component of such combination products. In the EU, companion diagnostics are deemed to be in vitro diagnostic medical devices and must conform with the applicable GSPRs. To demonstrate compliance with the GSPRs, companion diagnostics must undergo a conformity assessment by a Notified Body. If the related medicinal product has been, or is in the process of being, authorized through the centralized procedure for the authorization of medicinal products, the Notified Body will, before it can issue a CE Certificate of Conformity, be required to seek a scientific opinion from the EMA on the suitability of the companion diagnostic for use in relation to the medicinal product concerned. For medicinal products that have been or are in the process of authorization through any other route provided in EU legislation, the Notified Body must seek the opinion of the national competent authority of an EU Member State. Our product candidates intended for use with separately regulated devices, or expanded indications that we may seek for our products used with such devices, may not be approved or may be substantially delayed in receiving approval if the devices do not gain and/or maintain their own regulatory approvals, clearances, or certifications. Where approval of the drug or biologic product and device is sought under a single application, the increased complexity of the review process may delay approval. The FDA and EU review processes and related criteria are complex, which could also lead to delays in the approval process. In addition, because these devices are provided by unaffiliated third-party companies, we are dependent on the sustained cooperation and effort of those third-party companies both to obtain regulatory approval and to maintain their own regulatory compliance. Failure of third-party companies to assist in the approval process or to maintain their own regulatory compliance could delay or prevent approval of our product candidates, or limit our ability to sell a product once it is approved.

From time to time during the development and regulatory approval process for our products and product candidates, we engage in discussions with the FDA, the EC, the EMA and other comparable international regulatory authorities regarding our development programs, including discussions about the regulatory requirements for approval. As part of these discussions, we sometimes seek advice in the design of our clinical programs from various regulatory authorities globally, but we do not always follow such guidance. This increases the chance of adverse regulatory actions, but we try to always provide appropriate scientific evidence to support approval. Moreover, sometimes different regulatory authorities provide different or conflicting advice. While we attempt to harmonize the advice we receive from multiple regulatory authorities, it is not always practical to do so. Also, we may choose not to harmonize conflicting advice when harmonization would significantly delay clinical trial data or is otherwise inappropriate. If we are unable to effectively and efficiently resolve and comply with the inquiries and requests of the FDA, the EC,

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the EMA and other comparable international regulatory authorities, the approval of our product candidates may be delayed and their value may be reduced.

*Any product for which we have obtained regulatory approval, or for which we obtain approval in the future, is subject to, or will be subject to, extensive ongoing regulatory requirements by the FDA, the EC, the EMA and other comparable international regulatory authorities, and if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products, we may be subject to penalties, we will be unable to generate revenues from the sale of such products, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will be increased.

ALDURAZYME, BRINEURA, KUVAN, NAGLAZYME and VIMIZIM have received regulatory approval to be commercially marketed and sold in the U.S., the EU and certain other countries, PALYNZIQ has received regulatory approval to be commercially marketed in the U.S., the EU, and Australia. VOXZOGO has received regulatory approval to be commercially marketed in the U.S., the EU, and Brazil. ROCTAVIAN has received regulatory approval to be commercially marketed in the U.S. and conditional approval to be commercially marketed in the EU. Any product for which we have obtained regulatory approval, or for which we obtain regulatory approval in the future, along with the manufacturing processes and practices, post-approval clinical research, product labeling, advertising and promotional activities for such product, are subject to continual requirements of, and review by, the FDA, the EC, the EMA and/or other comparable international and national regulatory authorities. These requirements include submissions of safety and other post-marketing information and reports, registration and listing requirements, current good manufacturing practices (cGMP) requirements relating to manufacturing, quality control, quality assurance and corresponding maintenance of records and documents, import and export requirements and record keeping.

An example of the ongoing regulatory requirements our products are subject to is the PALYNZIQ Risk Evaluation and Mitigation Strategy (REMS) program. In the U.S., PALYNZIQ is only available through the REMS program, which is required by the FDA to mitigate the risk of anaphylaxis while using the product. Notable requirements of our REMS program include the following:

•prescribers must be certified by enrolling in the REMS program and completing training;

•prescribers must prescribe auto-injectable epinephrine with PALYNZIQ;

•pharmacies must be certified with the REMS program and must dispense PALYNZIQ only to patients who are authorized to receive it;

•patients must enroll in the REMS program and be educated about the risk of anaphylaxis by a certified prescriber to ensure they understand the risks and benefits of treatment with PALYNZIQ; and

•patients must have auto-injectable epinephrine available at all times while taking PALYNZIQ.

Failure of prescribers, pharmacies or patients to enroll in our REMS program or to successfully complete and comply with its requirements may result in regulatory action from the FDA or decreased sales of PALYNZIQ. The restrictions and requirements under our REMS program, as well as potential changes to these restrictions and requirements in the future, subject us to increased risks and uncertainties, any of which could harm our business. The requirement for a REMS program can materially affect the potential market for and profitability of a drug. We cannot predict whether the FDA will request, seek to require or ultimately require modifications to, or impose additional requirements on, the PALYNZIQ REMS program, or whether the FDA will permit modifications to the PALYNZIQ REMS program that we consider warranted. Any modifications required or rejected by the FDA could make it more difficult or expensive for us to distribute PALYNZIQ in the U.S., impair the safety profile of PALYNZIQ, disrupt continuity of care for PALYNZIQ patients and/or negatively affect sales of PALYNZIQ.

Moreover, promotional communications with respect to prescription drugs, including biologics, are subject to a variety of legal and regulatory restrictions and must be consistent with the information in the product’s approved labeling. In particular, a product may not be promoted for uses that are not approved by the FDA or the EC as reflected in the product’s approved labeling. Although the FDA and other comparable international and national regulatory authorities do not regulate a physician’s choice of drug treatment made in the physician’s independent medical judgment, they do restrict promotional communications from companies or their sales force with respect to off-label uses of products for which marketing clearance has not been issued. The FDA and other national competent authorities or international regulatory authorities actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to significant civil, criminal and administrative penalties. Thus, we are not able to promote any products we develop for indications or uses for which they are not approved. Additionally, in the EU, it is prohibited to promote prescription drugs to the general public and we are therefore limited to promote our products exclusively to healthcare professionals. Public prosecutors, industry associations, healthcare professionals and other members of the public closely scrutinize advertising and promotion of any product in the EU.

Moreover, if original FDA approval for one of our product candidates is granted via the accelerated approval pathway, we will be required to conduct a post-marketing confirmatory trial to verify and describe the clinical benefit in support of full approval. An unsuccessful post-marketing study or failure to complete such a study with due diligence could result in the withdrawal of the

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FDA’s marketing approval for a product candidate. For example, VOXZOGO is approved in the U.S. under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory studies. To fulfill this post-marketing requirement, we intend to use our ongoing open-label extension studies compared to available natural history. In addition, the FDA and the EC often require post-marketing testing and surveillance to monitor the effects of products. The FDA, the EC and other comparable international regulatory authorities may condition approval of our product candidates on the completion of such post-marketing clinical studies. These post-marketing studies may suggest that a product causes undesirable side effects or may present a risk to the patient.

Discovery after approval of previously unknown problems with any of our products, manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in actions such as:

•the issuance of safety alerts, press releases or other communications containing warnings about related products;

•modifications to promotional materials or corrective information to healthcare professionals;

•restrictions on our ability to conduct clinical trials, including full or partial clinical holds on ongoing or planned trials;

•suspensions or restrictions on our operations, including product manufacturing processes;

•restrictions on the marketing of a product;

•restrictions on product distribution;

•requirements to conduct post-marketing clinical trials;

•untitled or warning letters or other adverse publicity;

•withdrawal of the products from the market

•suspended or withdrawn regulatory approvals;

•refusal or delays to approve pending applications or supplements to approved applications that we submit;

•recall of products;

•refusal to permit the import or export of our products;

•product seizure;

•fines, restitution or disgorgement of profits or revenue;

•injunctions; or

•imposition of civil or criminal penalties.

If such regulatory actions are taken, our value and our operating results will be adversely affected. Additionally, if the FDA, the EC or any other comparable international regulatory authorities withdraws its approval of a product, we will be unable to generate revenues from the sale of that product in the relevant jurisdiction, our potential for generating positive cash flow will be diminished and the capital necessary to fund our operations will be increased. Accordingly, we continue to expend significant time, money and effort in all areas of regulatory compliance, including manufacturing, production, product surveillance, post-marketing studies and quality control.

*To obtain regulatory approval to market our products, preclinical studies and costly and lengthy clinical trials are required and the results of the studies and trials are highly uncertain. Likewise, preliminary, initial or interim data from clinical trials should be considered carefully and with caution because the final data may be materially different from the preliminary, initial or interim data, particularly as more patient data become available.

As part of the drug development process we must conduct, at our own expense, preclinical studies in the laboratory, including studies in animals, and clinical trials on humans for each product candidate. The number of preclinical studies and clinical trials that regulatory authorities require varies depending on the product candidate, the disease or condition the drug is being developed to address and regulations applicable to the particular drug. Generally, new drugs for diseases or conditions that affect larger patient populations, are less severe, or are treatable by alternative strategies must be validated through additional preclinical and clinical trials and/or clinical trials with higher enrollments. With respect to our early-stage product candidates, we may need to perform multiple preclinical studies using various doses and formulations before we can begin clinical trials, which could result in delays to our development timeline. Furthermore, even if we obtain favorable results in preclinical studies, the results in humans may be significantly different. After we have conducted preclinical studies, we must demonstrate that our product candidates are safe and efficacious for the intended indication and for use in the targeted human patients in order to receive regulatory approval for commercial sale. Clinical testing is expensive and can take many years to complete, and its outcome is inherently uncertain. Failure can occur at any time during the clinical trial process. The results of preclinical studies and early clinical trials of our product candidates may not be predictive of the results of later-stage clinical trials, and favorable data from interim analyses do not ensure the final results of a trial will be favorable. From time to time, we have and may in the future publish or report preliminary, initial or

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interim data from our clinical trials. Preliminary, initial or interim data from our clinical trials may not be indicative of the final results of the trial and are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and/or more patient data become available. In this regard, such data may show initial evidence of clinical benefit, but as patients continue to be followed and more patient data become available, there is a risk that any therapeutic effects will not be durable in patients and/or will decrease over time or cease entirely. Preliminary, initial or interim data also remain subject to audit and verification procedures that may result in the final data being materially different from such preliminary, initial or interim data. As a result, preliminary, initial or interim data should be considered carefully and with caution until the final data are available.

Product candidates may fail to show the desired safety and efficacy traits despite having progressed through preclinical studies and initial clinical trials, or despite having favorable data in connection with an interim analysis. A number of companies in the biopharmaceutical industry have suffered significant setbacks in advanced clinical trials due to lack of efficacy or adverse safety profiles, notwithstanding promising results in earlier trials. Also, as noted above, we do not always follow the advice of regulatory authorities or comply with all of their requests regarding the design of our clinical programs. In those cases, we may choose a development program that is inconsistent with the advice of regulatory authorities, which may limit the jurisdictions where we conduct clinical trials and/or adversely affect our ability to obtain approval in those jurisdictions where we do not follow the regulatory advice.

Adverse or inconclusive clinical results could stop us from obtaining regulatory approval of our product candidates. Additional factors that can cause delay or termination of our clinical trials include:

•slow or insufficient patient enrollment;

•slow recruitment of, and completion of necessary institutional approvals at, clinical sites;

•budgetary constraints or prohibitively high clinical trial costs;

•longer treatment time required to demonstrate efficacy;

•lack of sufficient supplies of the product candidate;

•adverse medical events or side effects in treated patients, including immune reactions;

•lack of effectiveness of the product candidate being tested;

•availability of competitive therapies to treat the same indication as our product candidates;

•regulatory requests for additional clinical trials or preclinical studies;

•deviations in standards for Good Clinical Practice (GCP); and

•disputes with or disruptions in our relationships with clinical trial partners, including CROs, clinical laboratories, clinical sites, and principal investigators.

Government price controls or other changes in pricing regulation could restrict the amount that we are able to charge for our current and future products, which would adversely affect our revenues and results of operations.

We expect that coverage and reimbursement may be increasingly restricted in all the markets in which we sell our products. The escalating cost of healthcare has led to increased pressure on the healthcare industry to reduce costs. In particular, drug pricing by pharmaceutical companies has been under scrutiny for many years and continues to be subject to intense political and public debate in the U.S. and abroad. Governmental and private third-party payers have proposed healthcare reforms and cost reductions. A number of federal and state proposals to control the cost of healthcare, including the cost of drug treatments, have been made in the U.S. Specifically, there have been several recent U.S. congressional inquiries and proposed bills and enacted legislation designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drugs. Further, Congress and the executive branch have each indicated that they will continue to seek new legislative and/or administrative measures to control drug costs. In some international markets, the government controls the pricing, which can affect the profitability of drugs. Current government regulations and possible future legislation regarding healthcare may affect coverage and reimbursement for medical treatment by third-party payers, which may render our products not commercially viable or may adversely affect our future revenues and gross margins.

International operations are also generally subject to extensive price and market regulations, and there are many proposals for additional cost-containment measures, including proposals that would directly or indirectly impose additional price controls or mandatory price cuts or reduce the value of our intellectual property portfolio. As part of these cost containment measures, some countries have imposed and continue to propose revenue caps limiting the annual volume of sales of our products. Some of these caps are significantly below the actual demand in certain countries, and if the trend regarding revenue caps continues, our future revenues and gross margins may be adversely affected. For example, in the EU, governments influence the price of medicinal products through their pricing and reimbursement rules and control of national healthcare systems that fund a large part of the cost of those products to consumers. EU Member States are free to restrict the range of medicinal products for

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which their national health insurance systems provide reimbursement and to control the prices of medicinal products for human use. Some jurisdictions operate positive and negative list systems under which products may only be marketed once a reimbursement price has been agreed to by the government. An EU Member State may approve a specific price for the medicinal product, or it may instead adopt a system of direct or indirect controls on the profitability of the company placing the medicinal product on the market, including volume-based arrangements, caps and reference pricing mechanisms. Other EU Member States allow companies to fix their own prices for medicines but monitor and control company profits. The downward pressure on healthcare costs in general, particularly prescription medicines, has become very intense. Pharmaceutical products may face competition from lower-priced products in foreign countries that have placed price controls on pharmaceutical products and may also compete with imported foreign products. Furthermore, there is no assurance that a product will be considered medically reasonable and necessary for a specific indication or cost-effective by third-party payers. There is also no assurance that an adequate level of reimbursement will be established even if coverage is available or that the third-party payers’ reimbursement policies will not adversely affect our business.

We cannot predict the extent to which our business may be affected by these or other potential future legislative or regulatory developments. However, future price controls or other changes in pricing regulation or negative publicity related to our product pricing or the pricing of pharmaceutical drugs generally could restrict the amount that we are able to charge for our current and future products or our sales volume, which would adversely affect our revenues and results of operations.

*Government healthcare reform could increase our costs and adversely affect our revenues and results of operations.

Our industry is highly regulated and changes in law may adversely impact our business, operations or financial results. In the U.S., there have been and continue to be a number of legislative initiatives to contain healthcare costs. In the U.S., there have been several recent congressional inquiries, proposed and enacted federal and state legislation and executive action designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the cost of drugs under Medicare, and reform government program reimbursement methodologies for drug products. Any reduction in reimbursement from Medicare and other government programs may result in a similar reduction in payments from private payers. Recently, healthcare reform initiatives culminated in the enactment of the Inflation Reduction Act (IRA) in August 2022, which will, among other things, allow U.S. Department of Health and Human Services (HHS) to negotiate the selling price of certain drugs and biologics that the CMS reimburses under Medicare Part B and Part D, although only high-expenditure single-source drugs that have been approved for at least 7 years (11 years for biologics) can be selected by CMS for negotiation. The IRA permits the Secretary of HHS to implement many of these provisions through guidance, as opposed to regulation, for the initial years. HHS has and will continue to issue and update guidance as these programs are implemented. Manufacturers that fail to comply with the IRA may be subject to various penalties, including civil monetary penalties.

Prior to the IRA, the Patient Protection and Affordable Care Act of 2010, as amended by the Health Care and Education Reconciliation Act of 2010 (collectively, the PPACA) is a sweeping measure intended to, among other things, expand healthcare coverage within the U.S., primarily through the imposition of health insurance mandates on employers and individuals and expansion of the Medicaid program. Several provisions of the law have affected us and increased certain of our costs. Since its enactment, there have been executive, judicial and congressional challenges to certain aspects of the PPACA. Although the PPACA has generally been upheld thus far, it is unclear how continued challenges to the law may impact the PPACA and our business. In addition, other legislative changes have been adopted since the PPACA was enacted. Some of these changes have resulted in additional reductions in Medicare and other healthcare funding, which could have a material adverse effect on our customers and, accordingly, our financial operations.

In addition, individual states in the U.S. have also increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, price disclosure and reporting requirements, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. Moreover, regional healthcare authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other healthcare programs.

Likewise, in many EU Member States, legislators and other policymakers continue to propose and implement healthcare cost-containing measures in response to the increased attention being paid to healthcare costs in the EU. Certain of these changes could impose limitations on the prices we will be able to charge for our products and any approved product candidates or the amounts of reimbursement available for these products from governmental and private third-party payers, may increase the tax obligations on pharmaceutical companies or may facilitate the introduction of generic competition with respect to our products. Further, an increasing number of EU Member States and other non-U.S. countries use prices for medicinal products established in other countries as “reference prices” to help determine the price of the product in their own territory. If the price of one of our products decreases substantially in a reference price country, it could impact the price for that product in other countries. Consequently, a downward trend in prices of our products in some countries could contribute to similar downward trends elsewhere, which would have a material adverse effect on our revenues and results of operations. Moreover, in order to obtain

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reimbursement for our products in some countries, we may be required to conduct clinical trials that compare the cost-effectiveness of our products to other available therapies.

We anticipate that the IRA, PPACA and other healthcare reform measures that may be adopted in the future in the U.S. or abroad, may result in more rigorous coverage criteria and an additional downward pressure on the reimbursement our customers may receive for our products. Recently there has been heightened governmental scrutiny in countries worldwide over the manner in which manufacturers set prices for their marketed products. Legally mandated price controls on payment amounts by governmental and private third-party payers or other restrictions could harm our business, results of operations, financial condition and prospects. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize our products.

*If we fail to obtain or maintain orphan drug exclusivity for some of our products, our competitors may obtain approval to sell the same drugs to treat the same conditions and our revenues will be reduced.

As part of our business strategy, we have developed and may in the future develop some drugs that may be eligible for FDA and EU orphan drug designation. Under the Orphan Drug Act, the FDA may designate a product as an orphan drug if it is intended to treat a rare disease or condition, defined as a patient population of fewer than 200,000 in the U.S. In the EU, pursuant to the Orphan Regulation, orphan drug designation is available if a sponsor can establish that: (1) the medicine is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than five in 10,000 people in the EU at the time the application is made, or, (2) that it is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition in the EU and that without incentives derived from the orphan status, it is unlikely that the marketing of the medicine in the EU would generate sufficient return to justify the necessary investment. In both cases, the applicant must demonstrate that there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorized in the EU or, if such method exists, the medicine will be of significant benefit to those affected by that condition.

In the U.S., the company that first obtains FDA approval for a designated orphan drug for a given rare disease receives marketing exclusivity for use of that drug for the stated condition for a period of seven years. Orphan drug exclusive marketing rights may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantity of the drug. In addition, the FDA may approve another drug during a period of orphan drug exclusivity if the second drug is found to be clinically superior to the first drug. In the EU, a ten-year period of market exclusivity (extendable to twelve years for orphan drugs that have complied with an agreed Pediatric Investigation Plan (PIP) pursuant to Regulation 1901/2006), during which similar medicines for the same indication cannot be placed on the market, is granted. Market authorizations (MAs) may also be granted to a similar medicinal product with the same orphan indication if: (i) the applicant can establish that the second medicinal product, although similar to the orphan medicinal product already authorized is safer, more effective or otherwise clinically superior to the orphan medicinal product already authorized; (ii) the MA holder for the first orphan medicinal product grants its consent; or (iii) if the MA holder of the orphan medicinal product is unable to supply sufficient quantities. MAs may also be granted for the same therapeutic indication in relation to products that are not similar. The period of market exclusivity may, in addition, be reduced to six years if, at the end of the fifth year, it can be demonstrated on the basis of available evidence that the criteria for its designation as an orphan medicine are no longer satisfied, for example if the original orphan medicinal product has become sufficiently profitable not to justify maintenance of market exclusivity. Because the extent and scope of patent protection for some of our products is limited, orphan drug designation is especially important for our products that are eligible for orphan drug designation. For eligible products, we plan to rely on the exclusivity period under the Orphan Drug Act and/or the Orphan Regulation, as applicable, to maintain a competitive position. If we do not obtain orphan drug exclusivity for our products that do not have broad patent protection, our competitors may then sell the same drug to treat the same condition and our revenues will be reduced.

Even though we have obtained orphan drug designation for certain of our product candidates and even if we obtain orphan drug designation for our future product candidates, due to the uncertainties associated with developing biopharmaceutical products, we may not be the first to obtain marketing approval for any particular orphan indication, which means that we may not obtain orphan drug exclusivity and could also potentially be blocked from approval of certain product candidates until the competitor product’s orphan drug exclusivity period expires. Moreover, with respect to certain biologics and gene therapies, there may be some uncertainty regarding how similarity between product candidates designed to treat the same rare disease or condition may affect such product candidates’ orphan drug exclusivities. For biologics and gene therapies, the FDA’s determination of whether a drug is the same drug or a different drug will be based on the principal molecular structural features of the products. For gene therapy products, the FDA has stated in guidance that it generally intends to consider certain key features such as transgenes and vectors used in gene therapy products to be principal molecular structural features. Further, even if we obtain orphan drug exclusivity for a product, that exclusivity may not effectively protect the product from competition because different drugs can be approved for the same condition and the same drug can be approved for different conditions and potentially used off-label in the orphan indication. Even after an orphan drug is approved and granted orphan drug exclusivity, the FDA can subsequently approve the same drug for the same condition if the FDA concludes that the later drug is safer or more effective or makes a major contribution to patient care. Orphan drug designation neither shortens the development time or regulatory review time of a drug, nor gives the drug any advantage in the regulatory review or approval process.

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*We may face competition from biosimilars approved through an abbreviated regulatory pathway.

Our ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ, ROCTAVIAN and VIMIZIM products are regulated by the FDA as biologics under the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act (the PHS Act). Biologics require the submission of a BLA and approval by the FDA prior to being marketed in the U.S. The Biologics Price Competition and Innovation Act of 2009 (BPCIA) created a regulatory pathway under the PHS Act for the abbreviated approval of biological products that are demonstrated to be “biosimilar” or “interchangeable” with an FDA-approved biological product. A similar abridged MA process is available to biosimilar products in the EU. In particular, applicants for MAs of biosimilars are required to demonstrate through comprehensive comparability studies with the reference biological medicine that: a) their biological medicine is highly similar to the reference medicine, notwithstanding natural variability inherent to all biological medicines; and b) there are no clinically meaningful differences between the biosimilar and the reference medicine in terms of safety, quality and efficacy.

In the U.S., in order to meet the standard of interchangeability, a sponsor must demonstrate that the biosimilar product can be expected to produce the same clinical result as the reference product, and for a product that is administered more than once, that the risk of switching between the reference product and biosimilar product is not greater than the risk of maintaining the patient on the reference product. The BPCIA establishes a period of 12 years of exclusivity for reference products. In the EU, a medicinal product containing a new active substance benefits from eight years of data exclusivity, during which biosimilar applications referring to the data of that product may not be accepted by the regulatory authorities, and a further two years of market exclusivity, during which such biosimilar products may not be placed on the market. The two-year period may be extended to three years if during the first eight years a new therapeutic indication with significant clinical benefit over existing therapies is approved. Our products approved under BLAs in the U.S. or as a result of Marketing Authorization Applications (MAAs) in the EU, as well as our product candidates that may be approved in the future, could be reference products for biosimilar marketing applications.

*Changes in funding for the FDA, the EMA, other comparable regulatory authorities and other government agencies or government shutdowns could hinder the ability of such authorities and agencies to hire and retain key leadership and other personnel or otherwise prevent those authorities and agencies from performing normal functions on which the operation of our business may rely, which could negatively impact our business.

Changes in funding levels of regulatory authorities and government agencies can affect their ability to hire and retain key personnel and carry out their normal functions that support our business. For example, the ability of the FDA or the EMA to timely review and approve INDs or MAAs for our product candidates may be hindered by a lack of resources and qualified personnel. In addition, funding of other regulatory authorities and government agencies on which our operations rely, including those that fund research and development activities, is subject to the political budget process, which is inherently fluid and unpredictable.

Government shutdowns could also impact the ability of regulatory authorities and government agencies to function normally and support our operations. For example, the U.S. federal government has shut down repeatedly since 1980, including for a period of 35 days beginning on December 22, 2018. During a shutdown, certain regulatory authorities and agencies, such as the FDA, have had to furlough key personnel and stop critical activities. If a prolonged government shutdown occurs, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business.

Risks Related to Our Gene Therapy Programs

*Our gene therapy products and product candidates are based on a novel technology, which presents additional development, manufacturing, regulatory and treatment risks in relation to our other, more traditional drug development programs.

In addition to the risks set forth in this Risk Factors section associated with more traditional pharmaceutical drugs, there are additional, unique development, manufacturing, regulatory and treatment risks associated with gene therapy products and product candidates like ROCTAVIAN and BMN 331.

The goal of gene therapy is to be able to correct an inborn genetic defect through administration of therapeutic genetic material containing non-defective gene copies. The gene copies are designed to reside permanently in a patient, allowing the patient to produce an essential protein or ribonucleic acid (RNA) molecule that a healthy person would normally produce. There is a risk, however, that the new gene copies will produce too little or too much of the desired protein or RNA. Although administration of a gene therapy product like ROCTAVIAN is intended to correct an inborn genetic defect for at least several years, there is a risk that the therapeutic effect will not be durable and production of the desired protein or RNA will decrease more quickly or cease entirely earlier than expected. If the therapeutic effect decreases significantly or ceases entirely, it is uncertain whether redosing is possible or would be effective. Furthermore, because gene therapy treatment is irreversible, there may be challenges in managing side effects, particularly those caused by potential overproduction of the desired protein. Adverse effects would not be able to be reversed or relieved by stopping dosing, and we may have to develop additional clinical safety procedures. Additionally, because the new gene copies are designed to reside permanently in a patient, there is a risk that they will disrupt other normal biological

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molecules and processes, including other healthy genes, and we may not learn the nature and magnitude of these side effects until long after clinical trials have been completed.

Moreover, gene therapy products are relatively novel and complex and have only in limited cases been manufactured at scales sufficient for pivotal trials and commercialization. Few pharmaceutical contract manufacturers specialize in gene therapy products and those that do are still developing appropriate processes and facilities for large-scale production. We invested a considerable amount of capital building our own commercial gene therapy manufacturing facility, which may be subject to significant impairment if our gene therapy programs are unsuccessful. As we develop, seek to optimize and operate the gene therapy manufacturing process, we will likely face technical and scientific challenges, considerable capital costs, and potential difficulty in recruiting and hiring experienced, qualified personnel. There may also be unexpected technical or operational issues during clinical or commercial manufacturing campaigns. As a result, we could experience manufacturing delays that prevent us from completing our clinical studies in a timely manner, if at all, or commercializing our gene therapy products on a profitable basis, if at all.

Furthermore, despite our recent success obtaining regulatory approval for ROCTAVIAN in the U.S. and conditional approval in the EU, we may experience regulatory challenges for other gene therapy product candidates that cause significant delays or unanticipated costs, or that cannot be solved. Although numerous companies are currently advancing gene therapy product candidates through clinical trials, the FDA and EC have only approved a relatively small number of vector-based gene therapy products thus far. As a result, it is difficult to determine how long it will take or how much it will cost to obtain regulatory approvals for our future gene therapy product candidates in any jurisdiction. Regulatory requirements governing gene and cell therapy products are still evolving and may continue to change in the future. For example, in October 2020, it was reported that the Director of the Center for Biologics Evaluation and Research, the center of the FDA responsible for reviewing marketing applications for gene therapies, stated that the FDA will assess the importance of durability of effect differently for a gene therapy that treats a disease that has no other available therapies versus a condition for which there are multiple approved treatments. Additionally, in September 2021, the FDA held a Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) to discuss toxicity risks of adeno-associated virus (AAV) vectors for gene therapy and to seek the CTGTAC’s insight into strategies to evaluate and mitigate risks in the context of AAV vector-based product design and quality, preclinical studies, and clinical trials. ROCTAVIAN and BMN 331 are AAV vector-based. Further, the FDA continues to develop and publish new guidance and policies, such as the publication of four draft or final gene therapy-specific guidance documents in 2022. These guidance documents and other recent policy statements demonstrate that regulatory requirements for gene therapies are likely to continue to evolve based upon factors such as the intended disease or class of diseases, product type or mechanism of action, as well as broader considerations such as the kinds of evidence that will be required for gene therapy products to take advantage of expedited development programs. Delay or failure to obtain, or unexpected costs in obtaining, the regulatory approval necessary to bring our gene therapy product candidates to market could have a negative effect on our business and financial condition.

As compared to our other, more traditional products, gene therapy products may present additional problems with respect to the pricing, coverage, and reimbursement and acceptance of the product.

In addition to the risks set forth in this Risk Factors section associated with commercializing more traditional pharmaceutical drugs, there are additional, unique commercial risks associated with gene therapy products like ROCTAVIAN. Due to the relative novelty of gene therapy and the potential to provide extended duration therapeutic treatment with a one-time administration, we face uncertainty with respect to the pricing, coverage and reimbursement of these products. In order to recover our research and development costs and commercialize one-time treatments on a profitable basis, the cost of a single administration of ROCTAVIAN is substantial, and it is likely other gene therapy products would also require relatively high prices. Therefore, coverage and reimbursement by governments and other third-party payers is essential for the vast majority of patients to be able to afford ROCTAVIAN or other gene therapy products that we may commercialize in the future. Accordingly, sales of our gene therapy products will depend substantially on the extent to which its cost will be paid by third-party payers. Even if coverage is provided, the reimbursement amounts approved by third-party payers may not be high enough to allow us to realize sufficient revenues from our investment in the development of our gene therapy products.

With respect to ROCTAVIAN specifically, we have entered into, and plan to enter into additional, outcomes-based agreements for the product with third-party payers to assist with realizing the value and sharing the risk of a one-time treatment, which make us subject to potential repayments if a patient does not respond to therapy or the therapeutic effect of the drug falls below specified thresholds. Although we will record reserves for potential refunds under the outcomes-based agreements for ROCTAVIAN in the same period as sales, our revenues and financial results could be adversely affected if our assumptions underlying our refund reserves differ from actual experience or otherwise underestimate refund obligations. Additionally, the increased complexity of reimbursement with outcomes-based arrangements heightens the risk that our price reporting may be inaccurate or delayed, which may result in fines and liability.

We also face uncertainty as to whether gene therapy will gain the acceptance of the public or the medical community. The commercial success of ROCTAVIAN or any other gene therapy product candidate that may be approved in the future will depend, in part, on the acceptance of physicians, patients and third-party payers of gene therapy products in general, and our product in particular, as medically necessary, cost-effective and safe. In particular, our success will depend upon physicians prescribing our

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product in lieu of existing treatments they are already familiar with and for which greater clinical data may be available. Moreover, physicians and patients may delay acceptance of one of our gene therapy treatments until the product has been on the market for a certain amount of time. Negative public opinion or more restrictive government regulations could have a negative effect on our business and financial condition and may delay or impair the successful commercialization of, and demand for, ROCTAVIAN or future gene therapy products.

Financial and Financing Risks

If we incur operating losses or are unable to sustain positive cash flows for a period longer than anticipated, we may be unable to continue our operations at planned levels and may be forced to reduce our operations.

Since we began operations in March 1997, we have been engaged in substantial research and development and capital investments, and we have operated at a net loss for each year since our inception, with the exceptions of 2008, 2010, 2020 and 2022. Our future profitability and cash flows depend on our marketing and selling of our products, the receipt of regulatory approval of our product candidates, our ability to successfully manufacture and market any products, either by ourselves or jointly with others, our spending on our development programs, the impact of any possible future business development transactions and other risks set forth in this Risk Factors section. The extent of our future losses and the timing of profitability and positive cash flows are highly uncertain. If we fail to become profitable or are unable to sustain profitability and positive cash flows on a continuing basis, then we may be unable to continue our operations at planned levels and be forced to reduce our operations.

*If we fail to obtain the capital necessary to fund our operations, our financial results and financial condition will be adversely affected and we will have to delay or terminate some or all of our product development programs.

As of September 30, 2023, we had cash, cash equivalents and investments totaling $1.67 billion and debt obligations of $1.1 billion (undiscounted), which consisted of our 0.599% senior subordinated convertible notes due in 2024 (the 2024 Notes) and our 1.25% senior subordinated convertible notes due in 2027 (the 2027 Notes). The 2024 Notes and the 2027 Notes (collectively, the Notes), if not converted, will be required to be repaid in cash at maturity in August 2024 and May 2027, respectively. We will need cash not only to pay the ongoing interest due on the Notes during their term, but also to repay the principal amount of the Notes if not converted.

We may require additional financing to fund the repayment of the Notes, future milestone payments and our future operations, including the commercialization of our products and product candidates currently under development, preclinical studies and clinical trials, and potential licenses and acquisitions. We may be unable to raise additional financing due to a variety of factors, including our financial condition, the status of our product programs, and the general condition of the financial markets. If we fail to raise any necessary additional financing we may have to delay or terminate some or all of our product development programs and our financial condition and operating results will be adversely affected.

We expect to continue to spend substantial amounts of capital for our operations for the foreseeable future. The amount of capital we will need depends on many factors, including:

•our ability to successfully market and sell our products;

•the time and cost necessary to develop commercial manufacturing processes, including quality systems, and to build or acquire manufacturing capabilities the progress and success of our preclinical studies and clinical trials (including studies and the manufacture of materials);

•the timing, number, size and scope of our preclinical studies and clinical trials;

•the time and cost necessary to obtain regulatory approvals and the costs of post-marketing studies which may be required by regulatory authorities;

•the progress of research programs carried out by us;

•any changes made to, or new developments in, our existing collaborative, licensing and other commercial relationships or any new collaborative, licensing and other commercial relationships that we may establish;

•Sanofi’s ability to continue to successfully commercialize ALDURAZYME; and

•whether our convertible debt is converted to common stock in the future.

Moreover, our fixed expenses such as rent, license payments, interest expense and other contractual commitments are substantial and may increase in the future. These fixed expenses may increase because we may enter into:

•additional licenses and collaborative agreements;

•additional contracts for product manufacturing; and

•additional financing facilities or arrangements.

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We will need to raise additional funds from equity or debt securities, loans or collaborative agreements if we are unable to satisfy our liquidity requirements. The sale of additional equity and/or equity-linked securities will result in additional dilution to our stockholders. Furthermore, additional financing may not be available in amounts or on terms satisfactory to us or at all. This could result in the delay, reduction or termination of our research, which could harm our business.

*We have incurred substantial indebtedness that may decrease our business flexibility, access to capital, and/or increase our borrowing costs, which may adversely affect our operations and financial results.

As of September 30, 2023, we had $1.1 billion (undiscounted) principal amount of indebtedness, including $495.0 million (undiscounted) principal amount of indebtedness under the 2024 Notes and $600.0 million (undiscounted) principal amount of indebtedness under the 2027 Notes. Our indebtedness may:

•limit our ability to borrow additional funds for working capital, capital expenditures, acquisitions or other general business purposes;

•limit our ability to use our cash flow or obtain additional financing for future working capital, capital expenditures, acquisitions or other general business purposes;

•require us to use a substantial portion of our cash flow from operations to make debt service payments;

•limit our flexibility to plan for, or react to, changes in our business and industry;

•place us at a competitive disadvantage compared to our less leveraged competitors; and

•increase our vulnerability to the impact of adverse economic and industry conditions.

If we default under any series of the Notes, such series of Notes could become immediately due and payable and it could lead to defaults under the other series of Notes.

*In addition, our ability to refinance our indebtedness will depend on the capital markets and our financial condition at such time.

Our outstanding indebtedness consists primarily of the 2024 Notes and 2027 Notes, which, if not converted, will be required to be repaid in cash at maturity in August 2024 and May 2027, respectively. While we could seek to obtain additional third-party financing to pay for any amounts due in cash upon maturity of the Notes, we cannot be sure that such third-party financing will be available on commercially reasonable terms, if at all.

Manufacturing Risks

*If we fail to comply with manufacturing regulations, our financial results and financial condition will be adversely affected.

Before we can begin commercial manufacture of our products, regulatory authorities must approve marketing applications that identify authorized manufacturing facilities operated by us or our contract manufacturers that have passed regulatory inspection and manufacturing processes that are acceptable to the regulatory authorities. In addition, our pharmaceutical manufacturing facilities are continuously subject to scheduled and unannounced inspection by the FDA, and other comparable EU and other national and international regulatory authorities, before and after product approval, to monitor and ensure compliance with cGMP and other regulations. Our manufacturing facility in the U.S. has been approved by the FDA and the EC for the manufacture of PALYNZIQ and ROCTAVIAN and been approved by the FDA, the EC, and health agencies in other countries for the manufacture of ALDURAZYME, BRINEURA, NAGLAZYME, VIMIZIM and VOXZOGO. Our manufacturing facility in Shanbally, Cork, Ireland has been approved by the FDA, the EC, and regulatory authorities in other countries for the manufacture of VIMIZIM and BRINEURA. In addition, our third-party manufacturers’ facilities involved with the manufacture of our products have also been inspected and approved by various regulatory authorities. Although we are not involved in the day-to-day operations of our contract manufacturers, we are ultimately responsible for ensuring that our products are manufactured in accordance with cGMP regulations.

Due to the complexity of the processes used to manufacture our products and product candidates, we may be unable to continue to pass or initially pass federal, national or international regulatory inspections in a cost-effective manner. For the same reason, any potential third-party manufacturer of our products or our product candidates may be unable to comply with cGMP regulations in a cost-effective manner and may be unable to initially or continue to pass a federal, national or international regulatory inspection.

If we, or third-party manufacturers with whom we contract, are unable to comply with manufacturing regulations, we may be subject to delay of approval of our product candidates, warning or untitled letters, fines, unanticipated compliance expenses,

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recall or seizure of our products, total or partial suspension of production and/or enforcement actions, including injunctions, and criminal or civil prosecution. These possible sanctions would adversely affect our financial results and financial condition.

*If we are unable to successfully develop and maintain manufacturing processes for our product candidates to produce sufficient quantities at acceptable costs, we may be unable to support a clinical trial or be forced to terminate a program, or if we are unable to produce sufficient quantities of our products at acceptable costs, we may be unable to meet commercial demand, lose potential revenue, have reduced margins or be forced to terminate a program.

Due to the complexity of manufacturing our product candidates and products, we may not be able to manufacture sufficient quantities. Our inability to produce enough of our product candidate at acceptable costs may result in the delay or termination of development programs. With respect to our commercial portfolio, we may not be able to manufacture our products successfully with a commercially viable process or at a scale large enough to support their respective commercial markets or at acceptable margins. For example, demand for VOXZOGO in certain markets has outpaced our projections in recent quarters, and if the trend continues, we could face challenges meeting demand, we may need to postpone planned entry into additional markets until VOXZOGO inventory levels increase or we could be required to delay certain VOXZOGO development activities. If we face such inventory constraints, we could lose potential VOXZOGO revenues that may never be recouped and our VOXZOGO development program could be adversely impacted.

The development of commercially viable manufacturing processes typically is very difficult to achieve and is often very expensive and may require extended periods of time. Changes in manufacturing processes (including manufacturing cell lines), equipment or facilities (including moving manufacturing from one of our facilities to another one of our facilities or a third-party facility, or from a third-party facility to one of our facilities) may require us to complete clinical trials to receive regulatory approval of any manufacturing modifications.

Also, we may be required to demonstrate product comparability between a biological product made after a manufacturing change and the product made before implementation of the change through additional types of analytical and functional testing or may have to complete additional clinical studies. If we contract for manufacturing services with an unproven process, our contractor is subject to the same uncertainties, high standards and regulatory controls, and may therefore experience difficulty if further process development is necessary.

Even a developed manufacturing process can encounter difficulties. Problems may arise during manufacturing for a variety of reasons, including human error, mechanical breakdowns, problems with raw materials and cell banks, malfunctions of internal information technology systems, and other events that cannot always be prevented or anticipated. Many of the processes include biological systems, which add significant complexity, as compared to chemical synthesis. We expect that, from time to time, consistent with biotechnology industry expectations, certain production lots will fail to produce product that meets our quality control release acceptance criteria. To date, our historical failure rates for all of our product programs have been within our expectations, which are based on industry norms. If the failure rate increased substantially, we could experience increased costs, lost revenue, damage to customer relations, time and expense investigating the cause and, depending upon the cause, similar losses with respect to other lots or products. If problems are not discovered before the product is released to the market, recall and product liability costs may also be incurred.

In order to produce product within our time and cost parameters, we must continue to produce product within our expected success rate and yield expectations. Because of the complexity of our manufacturing processes, it may be difficult or impossible for us to determine the cause of any particular lot failure and we must effectively take corrective action in response to any failure in a timely manner.

We currently rely on third parties for portions of the manufacture of each of our products. If those manufacturers are unwilling or unable to fulfill their contractual obligations or satisfy demand outside of or in excess of the contractual obligations, we may be unable to meet demand for these products or sell these products at all and we may lose potential revenue. Further, the availability of suitable contract manufacturing capacity at scheduled or optimum times is not certain.

In addition, our manufacturing processes subject us to a variety of federal, national, state and local laws and regulations governing the use, generation, manufacture, storage, handling and disposal of hazardous materials and wastes resulting from their use. We incur significant costs in complying with these laws and regulations.

Supply interruptions may disrupt our inventory levels and the availability of our products and product candidates and cause delays in obtaining regulatory approval for our product candidates, or harm our business by reducing our revenues.

We depend on single-source suppliers for critical raw materials and a limited number of manufacturing facilities to manufacture our finished products and product candidates. Numerous factors could cause interruptions in the supply or manufacture of our products and product candidates, including:

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•timing, scheduling and prioritization of production by our contract manufacturers or a breach of our agreements by our contract manufacturers;

•labor interruptions;

•changes in our sources for manufacturing;

•the timing and delivery of shipments;

•our failure to locate and obtain replacement suppliers and manufacturers as needed on a timely basis; and

•conditions affecting the cost and availability of raw materials, including inflation.

If one of our suppliers or manufacturers fails or refuses to supply us with necessary raw materials or finished products or product candidates on a timely basis or at all, it would take a significant amount of time and expense to qualify a new supplier or manufacturer. We may not be able to obtain active ingredients or finished products from new suppliers or manufacturers on acceptable terms and at reasonable prices, or at all.

Any interruption in the supply of finished products could hinder our ability to distribute finished products to meet commercial demand and adversely affect our financial results and financial condition.

With respect to our product candidates, production of product is necessary to perform clinical trials and successful registration batches are necessary to file for approval to commercially market and sell product candidates. Delays in obtaining clinical material or registration batches could adversely impact our clinical trials and delay regulatory approval for our product candidates.

If our Manufacturing, Marketing and Sales Agreement with Sanofi were terminated, we could be prevented from continuing to commercialize ALDURAZYME or our ability to successfully commercialize ALDURAZYME would be delayed or diminished.

Either party may terminate the Manufacturing, Marketing and Sales Agreement (the MMS Agreement) between Sanofi and us related to ALDURAZYME for specified reasons, including if the other party is in material breach of the MMS Agreement, has experienced a change of control, as such term is defined in the MMS Agreement, or has declared bankruptcy and also is in breach of the MMS Agreement. Although we are not currently in breach of the MMS Agreement, there is a risk that either party could breach the MMS Agreement in the future. Either party may also terminate the MMS Agreement upon one-year prior written notice for any reason.

If the MMS Agreement is terminated for breach, the breaching party will transfer its interest in the BioMarin/Genzyme LLC to the non-breaching party, and the non-breaching party will pay a specified buyout amount for the breaching party’s interest in ALDURAZYME and in the BioMarin/Genzyme LLC. If we are the breaching party, we would lose our rights to ALDURAZYME and the related intellectual property and regulatory approvals. If the MMS Agreement is terminated without cause, the non-terminating party would have the option, exercisable for one year, to buy out the terminating party’s interest in ALDURAZYME and in the BioMarin/Genzyme LLC at a specified buyout amount. If such option is not exercised, all rights to ALDURAZYME will be sold and the BioMarin/Genzyme LLC will be dissolved. In the event of termination of the buyout option without exercise by the non-terminating party as described above, all right and title to ALDURAZYME is to be sold to the highest bidder, with the proceeds to be split between Sanofi and us in accordance with our percentage interest in the BioMarin/Genzyme LLC.

If the MMS Agreement is terminated by either party because the other party declared bankruptcy, the terminating party would be obligated to buy out the other party and would obtain all rights to ALDURAZYME exclusively. If the MMS Agreement is terminated by a party because the other party experienced a change of control, the terminating party shall notify the other party, the offeree, of its intent to buy out the offeree’s interest in ALDURAZYME and the BioMarin/Genzyme LLC for a stated amount set by the terminating party at its discretion. The offeree must then either accept this offer or agree to buy the terminating party’s interest in ALDURAZYME and the BioMarin/Genzyme LLC on those same terms. The party who buys out the other party would then have exclusive worldwide rights to ALDURAZYME. The Amended and Restated Collaboration Agreement between us and Sanofi will automatically terminate upon the effective date of the termination of the MMS Agreement and may not be terminated independently from the MMS Agreement.

If we were obligated or given the option to buy out Sanofi’s interest in ALDURAZYME and the BioMarin/Genzyme LLC, and thereby gain exclusive rights to ALDURAZYME, we may not have sufficient funds to do so and we may not be able to obtain the financing to do so. If we fail to buy out Sanofi’s interest, we may be held in breach of the agreement and may lose any claim to the rights to ALDURAZYME and the related intellectual property and regulatory approvals. We would then effectively be prohibited from developing and commercializing ALDURAZYME. If this happened, not only would our product revenues decrease, but our share price would also decline.

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Risks Related to International Operations

We conduct a significant amount of our sales and operations outside of the U.S., which subjects us to additional business risks that could adversely affect our revenues and results of operations.

A significant portion of the sales of our products are generated from countries other than the U.S., and we expect international markets will continue to be important for the sales of any products approved in the future. We have operations in Canada and in several European, Middle Eastern, Asian, and Latin American countries. We expect that we will continue to expand our international operations in the future. International operations inherently subject us to a number of risks and uncertainties, including:

•the increased complexity and costs inherent in managing international operations;

•diverse regulatory and compliance requirements, and changes in those requirements that could restrict our ability to manufacture, market and sell our products;

•political and economic instability, such as the instability caused by Russia’s invasion of Ukraine;

•diminished protection of intellectual property in some countries outside of the U.S.;

•trade protection measures and import or export licensing requirements;

•difficulty in staffing and managing international operations;

•differing labor regulations and business practices;

•potentially negative consequences from changes in or interpretations of tax laws;

•changes in international medical reimbursement policies and programs;

•financial risks such as longer payment cycles, difficulty collecting accounts receivable, exposure to fluctuations in foreign currency exchange rates and potential currency controls imposed by non-U.S. governments;

•regulatory and compliance risks that relate to maintaining accurate information and control over sales and distributors’ and service providers’ activities that may fall within the purview of the Foreign Corrupt Practices Act (the FCPA); and

•rapidly evolving global laws and regulations relating to data protection and the privacy and security of commercial and personal information.

Any of these factors may, individually or as a group, have a material adverse effect on our business and results of operations. For example, Russia’s invasion of Ukraine and the related impacts to Ukraine’s infrastructure and healthcare system has significantly impacted our ability to provide our therapies to patients in Ukraine. Sanctions issued by the U.S. and other countries against Russia and Belarus in response to the attack on Ukraine and related counter-sanctions issued by Russia have made it very difficult for us to operate in Russia and may have a material adverse impact on our ability to sell our products and/or collect receivables from customers in Russia and Belarus.

As we continue to expand our existing international operations, we may encounter new risks. For example, as we focus on building our international sales and distribution networks in new geographic regions, we must continue to develop relationships with qualified local distributors and trading companies. If we are not successful in developing and maintaining these relationships, we may not be able to grow sales in these geographic regions. These or other similar risks could adversely affect our revenues and profitability.

*A significant portion of our international sales are made based on special access programs, and changes to these programs could adversely affect our product sales and revenues in these countries.

We make a significant portion of our initial international sales of newly launched products through early access, special access or “named patient sales” programs in markets where we are not required to obtain regulatory approval before establishing these programs. For example, a significant portion of our international sales of VOXZOGO since the product’s launch have been made through such programs. The specifics of the programs vary from country to country. Generally, special approval must be obtained to initiate such programs, and in some cases, special approval must be obtained for each patient. The approval normally requires an application to regulatory authorities or a lawsuit accompanied by evidence of medical need.

These programs are not well defined in some countries and are subject to changes in requirements, funding levels, unmet medical need and classification of the disease treated by our product. Any change to these programs could adversely affect our ability to sell our products in those countries and delay sales. If the programs are not funded by the respective government, there could be insufficient funds to pay for all patients. Further, governments have and may continue to undertake unofficial measures to limit purchases of our products, including initially denying coverage for purchasers, delaying orders, requiring additional in-country

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testing and denying or taking excessively long to approve customs clearance. Any such actions could materially delay or reduce our revenues from such countries.

Without the special access programs, we would need to seek full product approval or official reimbursement to commercially market and sell our products in certain jurisdictions. This can be an expensive and time-consuming process and may subject our products to additional price controls. Because the number of patients is so small in some countries, it may not be economically feasible to seek, obtain and maintain a full product approval or official reimbursement, and therefore the sales in such country would be permanently reduced or eliminated. For all of these reasons, if the special access programs that we are currently using are eliminated or restricted, our revenues could be adversely affected.

Our international operations pose currency risks, which may adversely affect our operating results and net income.

A significant and growing portion of our revenues and earnings, as well as our substantial international net assets, are exposed to changes in foreign exchange rates. As we operate in multiple foreign currencies, including the Euro, the Brazilian Real, the Canadian Dollar, the Colombian Peso, the Argentine Peso and several other currencies, changes in those currencies relative to the U.S. Dollar (USD) will impact our revenues and expenses. If the USD were to weaken against another currency, assuming all other variables remained constant, our revenues would increase, having a positive impact on earnings, and our overall expenses would increase, having a negative impact on earnings. Conversely, if the USD were to strengthen against another currency (as was the case for many currencies in 2022), assuming all other variables remained constant, our revenues would decrease, having a negative impact on earnings, and our overall expenses would decrease, having a positive impact on earnings. In addition, because our financial statements are reported in USD, changes in currency exchange rates between the USD and other currencies have had, and will continue to have, an impact on our results of operations. Therefore, significant changes in foreign exchange rates can impact our results and our financial guidance.

We implement currency hedges intended to reduce our exposure to changes in certain foreign currency exchange rates. However, our hedging strategies may not be successful, and any of our unhedged foreign exchange exposures will continue to be subject to market fluctuations. These risks could cause a material adverse effect on our business, financial position and results of operations and could cause the market value of our common stock to decline.

U.S. export control and economic sanctions may adversely affect our business, financial condition and operating results. Moreover, compliance with such regulatory requirements may increase our costs and negatively impact our ability to sell our products and collect cash from customers.

Our products are subject to U.S. export control laws and regulations, including the U.S. Export Administration Regulations and various economic and trade sanctions regulations administered by the U.S. Treasury Department’s Office of Foreign Assets Control (OFAC). Exports of our products and solutions must be made in compliance with these laws and regulations. Changes to these laws and regulations, or to the countries, governments, persons or activities targeted by such laws, could result in decreased use of our products, or hinder our ability to export or sell our products to existing or potential customers, which would likely adversely affect our results of operations, financial condition or strategic objectives. For example, sanctions issued by the U.S. and other jurisdictions against Russia and Belarus in response to the invasion of Ukraine have made it very difficult for us to operate in Russia and may have a material adverse impact on our ability to sell our products and/or collect receivables from customers in Russia and Belarus. Moreover, if we fail to comply with these laws and regulations, we could be subject to substantial civil or criminal penalties, including the possible loss of export or import privileges and fines.

We rely on a general license from OFAC to sell our medicines for eventual use by hospital and clinic end-users in Iran. The use of this OFAC general license requires us to observe strict conditions with respect to products sold, end-user limitations and payment requirements. Although we believe we have maintained compliance with the general license requirements, there can be no assurance that the general license will not be revoked, the general license will be renewed in the future or we will remain in compliance with the general license. A violation of the OFAC general license could result in substantial fines, sanctions, civil or criminal penalties, competitive or reputational harm, litigation or regulatory action and other consequences that might adversely affect our results of operations, financial condition or strategic objectives.

Moreover, U.S. export control and economic sanctions may make operating in certain countries more difficult and expensive. For example, we may be unable to find distributors or financial institutions willing to facilitate the sale of our products and collection of cash from such sales in a cost-effective manner, if at all.

Failure to comply with applicable anti-corruption legislation could result in fines, criminal penalties and materially adversely affect our business, financial condition and results of operations.

We are required to comply with anti-corruption and anti-bribery laws in the jurisdictions in which we operate, including the FCPA in the U.S. and other similar laws in other countries in which we do business. We operate in a number of countries that are recognized to have a reputation for corruption and pose an increased risk of corrupt practices. We also regularly interact with

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government regulators in many countries, including those that are considered higher risk for corruption, in order to secure regulatory approval to manufacture and distribute our products. The anti-corruption and anti-bribery laws to which we are subject generally prohibit companies and their intermediaries from making improper payments to non-U.S. government officials or other persons for the purposes of influencing official decisions or obtaining or retaining business and/or other benefits. These laws also require us to make and keep books and records that accurately and fairly reflect our transactions and to devise and maintain an adequate system of internal accounting controls. As part of our business, we deal with state-owned business enterprises, the employees and representatives of which may be considered non-U.S. government officials for purposes of applicable anti-corruption laws.

Although we have adopted policies and procedures designed to ensure that we, our employees and third-party agents will comply with such laws, there can be no assurance that such policies or procedures will work effectively at all times or protect us against liability under these or other laws for actions taken by our employees, partners and other third parties with respect to our business. If we are not in compliance with anti-corruption laws and other laws governing the conduct of business with government entities and/or officials (including local laws), we may be subject to criminal and civil penalties and other remedial measures, which could harm our business, financial condition, results of operations, cash flows and prospects. Investigations of any actual or alleged violations of such laws or policies related to us could harm our business, financial condition, results of operations, cash flows and prospects.

Moreover, there has been enhanced scrutiny of company-sponsored patient assistance programs, including insurance premium and co-pay assistance programs and donations to third-party independent charities that provide such assistance. There has also been enhanced scrutiny by governments on reimbursement support offerings, clinical education programs and promotional speaker programs. If we, our third-party agents or donation recipients are deemed to have failed to comply with laws, regulations or government guidance in any of these areas, we could be subject to criminal or civil sanctions. Any similar violations by our competitors could also negatively impact our industry reputation and increase scrutiny over our business and our products.

We face credit risks from government-owned or sponsored customers outside of the U.S. that may adversely affect our results of operations.

Our product sales to government-owned or supported customers in various countries outside of the U.S. are subject to significant payment delays due to government funding and reimbursement practices. This has resulted and may continue to result in an increase in days sales outstanding due to the average length of time that we have accounts receivable outstanding. If significant changes were to occur in the reimbursement practices of these governments or if government funding becomes unavailable, we may not be able to collect on amounts due to us from these customers and our results of operations would be adversely affected.

Intellectual Property Risks

If we are unable to protect our intellectual property, we may not be able to compete effectively or preserve our market shares.

Where appropriate, we seek patent protection for certain aspects of our technology. Patent protection may not be available for some of the products we are developing. If we must spend significant time and money protecting or enforcing our patents, designing around patents held by others or licensing, potentially for large fees, patents or other proprietary rights held by others, our business and financial prospects may be harmed.

The patent positions of biopharmaceutical products are complex and uncertain. The scope and extent of patent protection for some of our products and product candidates are particularly uncertain because key information on some of our product candidates has existed in the public domain for many years. The composition and genetic sequences of animal and/or human versions of ALDURAZYME, NAGLAZYME and many of our product candidates have been published and are believed to be in the public domain. The chemical structure of 6R-BH4 (the active ingredient in KUVAN) has also been published. Publication of this information may prevent us from obtaining or enforcing patents relating to our products and product candidates, including without limitation composition-of-matter patents, which are generally believed to offer the strongest patent protection.

We own or have licensed patents and patent applications related to our products. However, these patents and patent applications do not ensure the protection of our intellectual property for a number of reasons, including without limitation the following:

•With respect to pending patent applications, unless and until actually issued, the protective value of these applications is impossible to determine. We do not know whether our patent applications will result in issued patents.

•Patents have limited duration and expire.

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•Enforcing patents is expensive and may absorb significant time of our management. Management would spend less time and resources on developing products, which could increase our operating expenses and delay product programs.

•Receipt of a patent may not provide much, if any, practical protection. For example, if we receive a patent with a narrow scope, then it will be easier for competitors to design products that do not infringe on our patent.

•The Leahy-Smith America Invents Act of 2011, which reformed certain patent laws in the U.S., may create additional uncertainty. Among the significant changes are switching from a “first-to-invent” system to a “first-to-file” system, and the implementation of new procedures that permit competitors to challenge our patents in the U.S. Patent and Trademark Office after grant.

It is also unclear whether our trade secrets are adequately protected. Our current and former employees, consultants or contractors may unintentionally or willfully disclose trade secrets to competitors. Enforcing a claim that someone else illegally obtained and is using our trade secrets, as with patent litigation, is expensive and time consuming, requires significant resources and has an unpredictable outcome. In addition, courts outside of the U.S. are sometimes less willing to protect trade secrets. Furthermore, our competitors may independently develop equivalent knowledge, methods and know-how, in which case we would not be able to enforce our trade secret rights against such competitors.

In the EU, materials we submit to the EMA in connection with our clinical trials that were traditionally regarded as confidential, proprietary information, such as study protocols, information regarding manufacturing methods and controls, and intermediate data analyses, are now subject to public disclosure. Moreover, clinical trial data submitted to the EMA in our MAAs are also available to the public. We are only permitted to redact from public disclosures commercially confidential information, a standard which is construed narrowly and subject to the interpretation and final decision of the EU regulatory authorities. EU regulations have resulted and will continue to result in the EMA’s public disclosure of certain of our proprietary information related to recently completed and future clinical trials and MAA submissions. The move toward public disclosure of such development information could adversely affect our business in many ways, including, for example, resulting in the disclosure of our confidential methodologies for development of our products, preventing us from obtaining intellectual property right protection for innovations, requiring us to allocate significant resources to prevent other companies from violating our intellectual property rights, adding even more complexity to processing health data from clinical trials consistent with applicable data privacy regulations, increasing scrutiny of our product candidates and products, and enabling competitors to use our clinical trial information and data to gain approvals for their own products.

Competitors may interfere with our patent process in a variety of ways. Competitors may claim that they invented the claimed invention prior to us or that they filed their application for a patent on a claimed invention before we did. Competitors may also claim that we are infringing on their patents and therefore we cannot practice our technology. Competitors may also contest our patents by showing the patent examiner or a court that the invention was not original, was not novel or was obvious, for example. In litigation, a competitor could claim that our issued patents are not valid or are unenforceable for a number of reasons. If a court agrees, we would not be able to enforce that patent. Moreover, generic manufacturers may use litigation and regulatory means to obtain approval for generic versions of our products notwithstanding our filed patents or patent applications.

If we are unable to protect our intellectual property, third parties could develop competing products, which could adversely affect our revenues and financial results generally.

*Competitors and other third parties may have developed intellectual property that could limit our ability to market and commercialize our products and product candidates, if approved.

Similar to us, competitors continually seek intellectual property protection for their technology. Several of our products and development programs, such as ROCTAVIAN, focus on therapeutic areas that have been the subject of extensive research and development by third parties for many years. Due to the amount of intellectual property in our field of technology, we cannot be certain that we do not infringe intellectual property rights of competitors or that we will not infringe intellectual property rights of competitors granted or created in the future. For example, if a patent holder believes our product infringes its patent, the patent holder may sue us even if we have received patent protection for our technology. If someone else claims we infringe their intellectual property, we would face a number of issues, including the following:

•Defending a lawsuit takes significant executive resources and can be very expensive.

•If a court decides that our product infringes a competitor’s intellectual property, we may have to pay substantial damages.

•With respect to patents, in addition to requiring us to pay substantial damages, a court may prohibit us from making, selling, offering to sell, importing or using our product unless the patent holder licenses the patent to us. The patent holder is not required to grant us a license. If a license is available, it may not be available on commercially reasonable terms. For example, we may have to pay substantial royalties or grant cross licenses to our patents and patent applications.

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•We may need to redesign our product so it does not infringe the intellectual property rights of others.

•Redesigning our product so it does not infringe the intellectual property rights of competitors may not be possible or could require substantial funds and time.

We may also support and collaborate in research conducted by government organizations, hospitals, universities or other educational institutions. These research partners may be unwilling to grant us any exclusive rights to technology or products derived from these collaborations.

If we do not obtain required licenses or rights, we could encounter delays in our product development efforts while we attempt to design around other patents or may be prohibited from making, using, importing, offering to sell or selling products requiring these licenses or rights. There is also a risk that disputes may arise as to the rights to technology or products developed in collaboration with other parties. If we are not able to resolve such disputes and obtain the licenses or rights we need, we may not be able to develop or market our products.

Risks Related to Ownership of Our Securities

Our stock price has been and may in the future be volatile, and an investment in our stock could suffer a decline in value.

Our stock price has been and may in the future be volatile. Our valuation and stock price may have no meaningful relationship to current or historical earnings, asset values, book value or many other criteria based on conventional measures of stock value. The market price of our common stock will fluctuate due to factors including:

•product sales and profitability of our products;

•manufacturing, supply or distribution of our product candidates and products

•progress of our product candidates through the regulatory process and our ability to successfully commercialize any such products that receive regulatory approval;

•results of clinical trials, announcements of technological innovations or new products by us or our competitors;

•generic competition to KUVAN tablets and powder described above in this Risk Factors section or potential generic competition from future competitors;

•government regulatory action affecting our product candidates, our products or our competitors’ product candidates and products in both the U.S. and non-U.S. countries;

•developments or disputes concerning patent or proprietary rights;

•general market conditions and fluctuations for the emerging growth and pharmaceutical market sectors;

•economic conditions in the U.S. or abroad;

•negative publicity about us or the pharmaceutical industry;

•changes in the structure of healthcare payment systems;

•cybersecurity incidents experienced by us or others in our industry;

•broad market fluctuations in the U.S., the EU or in other parts of the world;

•actual or anticipated fluctuations in our operating results, including due to timing of large periodic orders for our products by governments in certain countries;

•changes in company assessments or financial estimates by securities analysts;

•acquisitions of products, businesses, or other assets; and

•sales of our shares of stock by us, our significant stockholders, or members of our management or Board of Directors.

Furthermore, the stock markets have recently experienced extreme price and volume fluctuations that have affected and continue to affect the market prices of equity securities of many companies. In some cases, these fluctuations have been unrelated or disproportionate to the operating performance of those companies. In the past, companies that have experienced volatility in the market price of their stock have been subject to securities class action litigation. For example, in September 2020, after a substantial drop in our stock price that followed an announcement providing a regulatory update regarding ROCTAVIAN, we and certain of our officers were sued in a putative class action lawsuit alleging violations of the federal securities laws for allegedly making materially false or misleading statements. In addition, in October 2021, after a drop in our stock price that followed an announcement providing a regulatory update regarding BMN 307, we and certain of our current and former officers were sued in a

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putative class action lawsuit alleging violations of the federal securities laws for allegedly making materially false or misleading statements. We may be the target of additional litigation of this type in the future as well. Securities litigation against us could result in substantial costs and divert our management’s time and attention from other business concerns, which could harm our business.

In addition, our stock price can be materially adversely affected by factors beyond our control, such as disruptions in global financial markets or negative trends in the biotechnology sector of the economy, even if our business is operating well.

Conversion of the Notes will dilute the ownership interest of existing stockholders, including holders who had previously converted their Notes, or may otherwise depress the price of our common stock.

The conversion of some or all of the Notes will dilute the ownership interests of existing stockholders. Any sales in the public market of the common stock issuable upon such conversion could adversely affect prevailing market prices of our common stock. In addition, the existence of the Notes may encourage short selling by market participants because the conversion of the Notes could be used to satisfy short positions, or anticipated conversion of the Notes into shares of our common stock could depress the price of our common stock.

The fundamental change repurchase feature of the Notes may delay or prevent an otherwise beneficial attempt to take us over.

The terms of the Notes require us to offer to repurchase the Notes in the event of a fundamental change (as defined in each indenture governing the Notes). A takeover of us would trigger options by the respective holders of the applicable Notes to require us to repurchase such Notes. This may have the effect of delaying or preventing a takeover of us that would otherwise be beneficial to our stockholders or investors in the Notes.

Anti-takeover provisions in our charter documents and under Delaware law may make an acquisition of us, which may be beneficial to our stockholders, more difficult.

We are incorporated in Delaware. Certain anti-takeover provisions of Delaware law and our charter documents as currently in effect may make a change in control of us more difficult, even if a change in control would be beneficial to the stockholders. Our anti-takeover provisions include provisions in our restated certificate of incorporation and amended and restated bylaws providing that stockholders’ meetings may only be called by our Chairman, the lead independent director or the majority of our Board of Directors and that the stockholders may not take action by written consent and requiring that stockholders that desire to nominate any person for election to our Board of Directors or to make any proposal with respect to business to be conducted at a meeting of our stockholders be submitted in appropriate form to our Secretary within a specified period of time in advance of any such meeting. Additionally, our Board of Directors has the authority to issue shares of preferred stock and to determine the terms of those shares of stock without any further action by our stockholders. The rights of holders of our common stock are subject to the rights of the holders of any preferred stock that may be issued. The issuance of preferred stock could make it more difficult for a third party to acquire a majority of our outstanding voting stock. Delaware law also prohibits corporations from engaging in a business combination with any holders of 15% or more of their capital stock until the holder has held the stock for three years unless, among other possibilities, our Board of Directors approves the transaction. Our Board of Directors may use these provisions to prevent changes in the management and control of us. Also, under applicable Delaware law, our Board of Directors may adopt additional anti-takeover measures in the future.

Our amended and restated bylaws designate the Court of Chancery of the State of Delaware and the federal district courts of the U.S. as the exclusive forums for the adjudication of certain disputes, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers, or employees.

Our amended and restated bylaws provide that the Court of Chancery of the State of Delaware is the sole and exclusive forum for the following types of actions or proceedings under Delaware statutory or common law:

•any derivative claim or cause of action brought on our behalf;

•any claim or cause of action for breach of a fiduciary duty owed by any director, officer or other employee of BioMarin to us or our stockholders;

•any claim or cause of action against us or any of our directors, officers or other employees arising pursuant to any provision of the General Corporation Law of the State of Delaware, our restated certificate of incorporation or our amended and restated bylaws; any claim or cause of action seeking to interpret, apply, enforce or determine the validity of our restated certificate of incorporation or our amended and restated bylaws;

•any claim or cause of action as to which the General Corporation Law of the State of Delaware confers jurisdiction to the Court of Chancery of the State of Delaware; and

•any claim or cause of action against us or any of our directors, officers or other employees that is governed by the internal affairs doctrine.

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This exclusive-forum provision would not apply to suits brought to enforce a duty or liability created by the Securities Act of 1933, as amended, the Exchange Act or any other claim for which the U.S. federal courts have exclusive jurisdiction. In addition, our amended and restated bylaws provide that the federal district courts of the U.S. of America will be the exclusive forum for resolving any complaint asserting a cause of action arising under the Securities Act.

While the Delaware courts have determined that such choice of forum provisions are facially valid, a stockholder may nevertheless seek to bring a claim in a venue other than those designated in the exclusive forum provisions. In such instance, we would expect to vigorously assert the validity and enforceability of the exclusive forum provisions of our amended and restated bylaws. This may require significant additional costs associated with resolving such action in other jurisdictions and there can be no assurance that the provisions will be enforced by a court in those other jurisdictions.

These exclusive forum provisions may limit a stockholder’s ability to bring a claim in a judicial forum that it finds favorable for disputes with us or our directors, officers, or other employees, which may discourage lawsuits against us and our directors, officers, and other employees. If a court were to find either of our exclusive forum provisions to be inapplicable or unenforceable in an action, we may incur further significant additional costs associated with resolving the dispute in other jurisdictions, all of which could seriously harm our business. Our amended and restated bylaws further provide that any person or entity that acquires any interest in shares of our capital stock will be deemed to have notice of and consented to the provisions of such provisions.

General Risk Factors

*We depend upon our key personnel and our ability to attract and retain qualified employees.

Our future growth and success will depend in large part on our continued ability to attract, retain, manage and motivate our employees. The loss of the services of a significant portion of our workforce or any member of our senior management or the inability to hire or retain qualified personnel could adversely affect our ability to execute our business plan and harm our operating results.

Because of the specialized nature of our business, we rely heavily on our ability to attract and retain qualified scientific, technical and managerial personnel. In particular, the loss of one or more of our senior executive officers could be detrimental to us if we do not have an adequate succession plan or if we cannot recruit suitable replacements in a timely manner. While our senior executive officers are parties to employment agreements with us, these agreements do not guarantee that they will remain employed with us in the future. In addition, in many cases, these agreements do not restrict our senior executive officers’ ability to compete with us after their employment is terminated. In November 2023, we announced the retirement of Jean-Jacques Bienaimé, our then-current President and Chief Executive Officer, and the appointment of Alexander Hardy as President and Chief Executive Officer, each effective December 1, 2023. This change in our management team and senior leadership could cause retention and morale concerns among current employees, as well as operational risks. If Mr. Hardy’s succession as President and Chief Executive Officer is not managed successfully, including his ability to lead a team that can effectively implement our strategic plans, it could disrupt our business and affect our financial condition and operating results.

The competition for qualified personnel in the pharmaceutical field is intense, and there is a limited pool of qualified potential employees to recruit. This competition was exacerbated during the COVID-19 pandemic, and we have recently experienced increased employee turnover like many other employers in the U.S. during the “great resignation.” Due to the intense competition for talent, we may be unable to continue to attract and retain qualified personnel necessary for the development of our business or to recruit suitable replacement personnel. If we are unsuccessful in our recruitment and retention efforts, our business may be harmed.

Our success depends on our ability to manage our growth.

Our two newest products, VOXZOGO and ROCTAVIAN, address potentially larger patient populations than most of our other products, and product candidates that we are currently developing or may license or acquire in the future may be intended for similarly larger patient populations than we have historically targeted. In order to continue development of such product candidates and marketing of products with larger markets, we will need to continue expanding our operations. To manage expansion effectively, we need to continue to develop and improve our research and development capabilities, manufacturing and quality capacities, sales and marketing capabilities, financial and administrative systems and standard processes for global operations. Our staff, financial resources, systems, procedures or controls may be inadequate to support our operations and may increase our exposure to regulatory, competitive, and corruption risks and our management may be unable to manage successfully current or future market opportunities or our relationships with customers and other third parties.

*New tax laws or regulations that are enacted or existing tax laws and regulations that are interpreted, modified or applied adversely to us or our customers may have a material adverse effect on our business and financial condition.

New tax laws or regulations could be enacted at any time, and existing tax laws or regulations could be interpreted, modified or applied in a manner that is adverse to us or our customers, which could adversely affect our business and financial

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condition. For example, the Tax Cuts and Jobs Act, the Coronavirus Aid, Relief, and Economic Security Act and the Inflation Reduction Act enacted many significant changes to the U.S. tax laws. Future guidance from the Internal Revenue Service and other tax authorities with respect to such legislation may affect us, and certain aspects of such legislation could be repealed or modified in future legislation. In addition, it is uncertain if and to what extent various states will conform to federal tax laws. Any future tax legislation could increase our U.S. tax expense and could have a material adverse impact on our business and financial condition.

Moreover, changes in the tax laws of jurisdictions in which we conduct business could arise, including as a result of the base erosion and profit shifting (BEPS) project that is being led by the Organization for Economic Co-operation and Development (OECD), and other initiatives led by the OECD or the EC. For example, the OECD, which represents a coalition of member countries including the U.S. and other countries in which we have operations, is working on proposals, commonly referred to as “BEPS 2.0”, which, if and to the extent implemented, would make important changes to the international tax system. These proposals are based on two “pillars”, Pillar One focuses on the allocation of taxing rights in respect of certain profits of multinational enterprises with annual global revenue above 20 billion euros and profitability above 10% to the jurisdictions within which they carry on business (based on the thresholds, we currently expect to be outside the scope of the Pillar One proposals, but could fall within their scope in the future) and Pillar Two imposes a minimum effective tax rate of 15% on certain multinational enterprises that have consolidated revenues of at least 750 million euros in at least two out of the last four years (based on the thresholds, we currently expect that we are likely to fall within the scope of the Pillar Two proposals). The EU has adopted a Council Directive requiring aspects of the Pillar Two proposal to be transposed into the national laws of EU Member States by December 31, 2023, and a number of other countries in which we conduct business also have enacted, or are in the process of enacting, such rules. The OECD has issued administrative guidance providing transition and safe harbor rules around the implementation of Pillar Two. We are monitoring developments and evaluating the impacts these new rules will have on our tax rate, including eligibility to qualify for these safe harbor rules. It is not uncommon for taxing authorities in different countries to have conflicting views, for instance, with respect to, among other things, the manner in which the arm’s length standard is applied for transfer pricing purposes, or with respect to the valuation of intellectual property. If tax authorities successfully challenge our transfer prices as not reflecting arm’s length transactions, they could require us to adjust our transfer prices and thereby reallocate our income to reflect these revised transfer prices, resulting in a higher tax liability. In addition, if a country from which income is reallocated does not agree with the reallocation, both that country and the other country to which the income was allocated could tax the same income, potentially resulting in double taxation. If tax authorities were to allocate income to a higher tax jurisdiction, subject our income to double taxation or assess interest and penalties, it would increase our consolidated tax liability, which could adversely affect our business, financial condition, results of operations and cash flows.

*If we are found in violation of healthcare laws or privacy and data protection laws, we may be required to pay penalties, be subjected to scrutiny by regulators or governmental entities, or be suspended from participation in government healthcare programs, which may adversely affect our business, reputation, financial condition and results of operations.

We are subject to various healthcare laws and regulations in the U.S. and internationally, including anti-kickback laws, false claims laws, data privacy and security laws, and laws related to ensuring compliance. In the U.S., the federal Anti-Kickback Statute makes it illegal for any person or entity, including a pharmaceutical company, to knowingly and willfully offer, solicit, pay or receive any remuneration, directly or indirectly, in exchange for or to induce the referral of business, including the purchase, order or prescription of a particular drug, for which payment may be made under federal healthcare programs, such as Medicare and Medicaid. Under the federal Anti-Kickback Statute and related regulations, certain arrangements are deemed not to violate the federal Anti-Kickback Statute if they fit within a statutory exception or regulatory safe harbor. However, the exceptions and safe harbors are drawn narrowly, and practices that involve remuneration not intended to induce prescribing, purchases or recommendations may be subject to scrutiny if they do not qualify for an exception or safe harbor. Our practices may not in all cases meet all of the criteria for safe harbor protection from Anti-Kickback liability, although we seek to comply with these safe harbors. Many states have adopted laws similar to the federal Anti-Kickback Statute, some of which apply to referral of patients for healthcare services reimbursed by any source, not just governmental payers.

Federal and state false claims laws, including the civil False Claims Act and the Civil Monetary Penalties Law, prohibit any person or entity from knowingly presenting, or causing to be presented, a false claim for payment to the federal government, or knowingly making, or causing to be made, a false statement to have a false claim paid, or knowingly making, using, or causing to be made or used, a false record or statement to avoid, decrease or conceal an obligation to pay money to the federal government. In addition, certain marketing practices, including off-label promotion, may also violate false claims laws.

Under the Health Insurance Portability and Accountability Act of 1996 (HIPAA), we also are prohibited from, among other things, knowingly and willfully executing a scheme to defraud any healthcare benefit program, including private payers, or knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent statement in connection with the delivery of or payment for healthcare benefits, items or services.

In addition, recent healthcare reform legislation has strengthened these laws in the U.S. For example, the PPACA, among other things, amends the intent requirement of the federal Anti-Kickback Statute and criminal healthcare fraud statutes. A person or

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entity no longer needs to have actual knowledge of these statutes or specific intent to violate them in order to commit a violation. Moreover, the PPACA provides that the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the civil False Claims Act.

HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act and its implementing regulations, also imposes obligations, including mandatory contractual terms, on certain types of individuals and entities, with respect to safeguarding the privacy, integrity, availability, security and transmission of individually identifiable health information. Many state and non-U.S. laws also govern the privacy and security of health information. They often differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts. The global data protection landscape is rapidly evolving, and implementation standards and enforcement practices are likely to remain uncertain for the foreseeable future. In the U.S., California enacted the California Consumer Privacy Act (CCPA), which took effect on January 1, 2020. The CCPA gives California consumers expanded rights to access and delete their personal information, opt out of certain personal information sales, and receive detailed information about how their personal information is used. The CCPA provides for civil penalties for violations, as well as a private right of action for data breaches that is expected to increase data breach litigation. The CCPA was expanded substantially on January 1, 2023 when the California Privacy Rights Act of 2020 (CPRA) took effect and amended the CCPA. Following the CPRA amendments, the CCPA, among other things, gives consumers the ability to limit use of information deemed to be sensitive, increase the maximum penalties for violations concerning consumers under age 16, expands an individual’s private right of action and establishes the California Privacy Protection Agency to implement and enforce the new law and impose administrative fines.

In addition to California, other U.S. states have recently adopted consumer data protection and privacy laws, and more U.S. states may do so in the future. This creates the potential for a patchwork of overlapping but different state laws and could mark the beginning of a trend toward more stringent privacy legislation in the U.S., which could increase our potential liability and adversely affect our business, financial condition, and results of operations. Many other states are considering proposed comprehensive data privacy legislation and all 50 states have passed some form of legislation relating to privacy or cybersecurity.

Aspects of the CCPA, CPRA and similar laws in other states and their interpretation and enforcement remain uncertain. The potential effects of these laws are far-reaching and may require us to modify our data processing practices and policies and to incur substantial costs and expenses in an effort to comply. Complying with these or other similar laws, regulations, amendments to or re-interpretations of existing laws and regulations, and contractual or other obligations relating to privacy, data protection, data transfers, data localization, or information security may require us to make changes to our services to enable us or our customers to meet new legal requirements, incur substantial operational costs, modify our data practices and policies, and restrict our business operations. Any actual or perceived failure by us to comply with these laws, regulations, or other obligations may lead to significant fines, penalties, regulatory investigations, lawsuits, significant costs for remediation, damage to our reputation, or other liabilities.

The European Regulation 2016/679, known as the General Data Protection Regulation (GDPR), as well as EEA Member State legislations supplementing such regulation, apply to the processing of personal data of individuals located in the EEA, including health-related information, by companies located in the EEA, or in certain circumstances, by companies located outside of the EEA. These laws impose strict obligations on the ability to collect, record, store, disclose, use and transmit personal data, including health-related information. These include several requirements relating to (i) obtaining, in some situations, the informed consent of the individuals to whom the personal data relates, (ii) the information provided to the individuals about how their personal information is used, (iii) ensuring the security and confidentiality of the personal data, (iv) the obligation to notify regulatory authorities and affected individuals of personal data breaches, (v) extensive internal privacy governance obligations, and (vi) obligations to honor rights of individuals in relation to their personal data (for example, the right to access, correct and delete their data). Switzerland has adopted similar restrictions.

The GDPR and other European data protection laws generally restrict the transfer of personal information from Europe, including the EEA and Switzerland, to the U.S. and most other countries unless the parties to the transfer have implemented specific safeguards to protect the transferred personal information. One of the primary safeguards allowing U.S. companies to import personal information from the EEA has been the EC’s Standard Contractual Clauses (SCCs). However, the Court of Justice of the EU (CJEU) issued a decision that called into question whether the SCCs can lawfully be used for transfers of personal information from Europe to the United States or most other countries. At present, there are few, if any, viable alternatives to the SCCs, on which we have relied for personal information transfers from Europe to the United States and other “third countries.” After the mentioned CJEU judgment, new sets of SCCs were published on June 4, 2021. Most importantly, the use of SCCs does not any longer automatically ensure compliance with the GDPR. Instead, companies remain required to conduct a data transfer impact assessment for each transfer, which adds a compliance burden.

Potential pecuniary fines for noncompliance with the GDPR may be up to the greater of €20 million or 4% of annual global revenue. The GDPR has increased our responsibility and liability in relation to personal data that we process and has increased our compliance costs. The EU regulations that make certain materials we submit to the EMA in connection with our clinical trials subject to public disclosure have increased the risk that we may unintentionally disclose personal information protected under the GDPR and thereby incur associated penalties and suffer reputational damage.

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In addition to the U.S. and European countries, other countries in which we operate have also enacted data privacy laws or may do so in the future. For example, Brazil’s General Data Protection Law (LGPD), which is modeled on the GDPR, took effect on August 16, 2020.

Substantial new laws and regulations affecting compliance have also been adopted in the U.S. and certain non-U.S. countries, which may require us to modify our business practices with healthcare practitioners. For example, in the U.S., the PPACA, through the Physician Payments Sunshine Act, requires certain drug, biologicals and medical supply manufacturers to collect and report to CMS information on payments or transfers of value to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other health care professionals (such as physicians assistants and nurse practitioners), and teaching hospitals, as well as investment and ownership interests held by such physicians and their immediate family members during the preceding calendar year. In addition, there has been a recent trend of increased state regulation of payments made to physicians. Certain states and/or local jurisdictions mandate implementation of compliance programs, compliance with the Office of Inspector General Compliance Program Guidance for Pharmaceutical Manufacturers and the Pharmaceutical Research and Manufacturers of America (PhRMA) Code on Interactions with Healthcare Professionals, the registration of pharmaceutical sales representatives and/or the tracking and reporting of gifts, compensation and other remuneration to physicians, marketing expenditures, and drug pricing. Likewise, in many non-U.S. countries there is an increasing focus on the relationship between drug companies and healthcare practitioners. Recently enacted non-U.S. legislation creates reporting obligations on payments, gifts and benefits made to these professionals. Outside the U.S., interactions between pharmaceutical companies and health care professionals are also governed by strict laws, such as national anti-bribery laws of European countries, national sunshine rules, regulations, industry self-regulation codes of conduct and physicians’ codes of professional conduct. The shifting regulatory environment and the need to implement systems to comply with multiple jurisdictions with different compliance and/or reporting requirements increases the costs of maintaining compliance and the possibility that we may violate one or more of the requirements and be subject to fines or sanctions.

Due to the breadth of the healthcare and privacy and data protection laws described above, the narrowness of available statutory and regulatory exceptions and safe harbors and the increased focus by law enforcement authorities in enforcing such laws, our business activities could be subject to challenge under one or more of such laws. If we are found in violation of one of these laws, we may be subject to significant criminal, civil or administrative sanctions, including damages, fines, disgorgement, imprisonment, contractual damages, reputational harm, public reprimands, diminished profits and future earnings, additional reporting requirements and oversight if we become subject to a corporate integrity agreement or similar agreement to resolve allegations of non-compliance with these laws, curtailment of our operations, and debarment, suspension or exclusion from participation in government healthcare programs, any of which could adversely affect our business, financial condition and results of operations.

If product liability lawsuits are successfully brought against us, we may incur substantial liabilities.

We are exposed to the potential product liability risks inherent in the testing, manufacturing and marketing of human pharmaceuticals. We currently maintain insurance against product liability lawsuits for the commercial sale of our products and for the clinical trials of our product candidates. Pharmaceutical companies must balance the cost of insurance with the level of coverage based on estimates of potential liability. Historically, the potential liability associated with product liability lawsuits for pharmaceutical products has been unpredictable. Although we believe that our current insurance is a reasonable estimate of our potential liability and represents a commercially reasonable balancing of the level of coverage as compared to the cost of the insurance, we may be subject to claims in connection with our clinical trials and commercial use of our products and product candidates for which our insurance coverage may not be adequate and we may be unable to avoid significant liability if any product liability lawsuit is brought against us. If we are the subject of a successful product liability claim that exceeds the limits of any insurance coverage we obtain, we may incur substantial charges that would adversely affect our earnings and require the commitment of capital resources that might otherwise be available for the development and commercialization of our product programs.

*We rely significantly on information technology systems and any failure, inadequacy, interruption or security lapse of that technology, including any cybersecurity incidents, could harm our ability to operate our business effectively and have a material adverse effect on our business, reputation, financial condition, and results of operations.

We rely significantly on our information technology systems to effectively manage and maintain our operations, inventory and internal reports, to manufacture and ship products to customers and to timely invoice them. Any failure, inadequacy or interruption of that infrastructure or security lapse (whether intentional or inadvertent) of that technology, including cybersecurity incidents or attacks, could harm our ability to operate our business effectively. Our ability to manage and maintain our operations, inventory and internal reports, to manufacture and ship our products to customers and timely invoice them depends significantly on our enterprise resource planning, production management and other information systems. Our technology systems, including our cloud technologies, continue to increase in multitude and complexity, making them potentially vulnerable to breakdown, cyberattack and other disruptions. Potential problems and interruptions associated with the implementation of new or upgraded technology systems or with maintenance or adequate support of existing systems could disrupt or reduce the efficiency of our operations and expose us to greater risk of security breaches. Cybersecurity incidents resulting in the failure of our enterprise resource planning

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system, production management or other systems to operate effectively or to integrate with other systems, or a breach in security or other unauthorized access or unavailability of these systems or those of any third parties in our supply chain or on whom we otherwise depend, have occurred in the past and may affect our ability in the future to manage and maintain our operations, inventory and internal reports, and result in delays in product fulfillment and reduced efficiency of our operations.

As part of our business, we collect, store, and transmit large amounts of confidential information, proprietary data, intellectual property, and personal data. The information and data processed and stored in our technology systems, and those of our research collaborators, CROs, contract manufacturers, suppliers, distributors, or other third parties on whom we depend to operate our business, may be vulnerable to loss, damage, denial-of-service, unauthorized access or misappropriation. Data security breaches may be the result of unauthorized or unintended activity (or lack of activity) by our employees, contractors, or others with authorized access to our network or malware, hacking, business email compromise, phishing, ransomware or other cyberattacks directed by third parties. While we have implemented measures to protect our information and data stored in our technology systems and those of the third parties that we rely on, our efforts may not be successful.

We have experienced and may continue to experience cybersecurity incidents, although to our knowledge we have not experienced any material incident or interruption to date. If such a significant event were to occur, it could result in a material disruption of our development programs and commercial operations, including due to a loss, corruption or unauthorized disclosure of our trade secrets, personal data or other proprietary or sensitive information. Further, these cybersecurity incidents can lead to the public disclosure of personal information (including sensitive personal information) of our employees, clinical trial patients and others and result in demands for ransom or other forms of blackmail. Such attacks, including phishing attacks and attempts to misappropriate or compromise confidential or proprietary information or sabotage enterprise IT systems, are of ever-increasing levels of sophistication and are made by groups and individuals with a wide range of motives (including industrial espionage) and expertise, including by organized criminal groups, “hacktivists”, nation states and others. Moreover, the costs to us to investigate and mitigate cybersecurity incidents could be significant. For example, the loss of clinical trial data could result in delays in our product development or regulatory approval efforts and significantly increase our costs to recover or reproduce the data. Any security breach that results in the unauthorized access, use or disclosure of personal data may require us to notify individuals, governmental authorities, credit reporting agencies, or other parties pursuant to privacy and security laws and regulations or other obligations. Such a security compromise could harm our reputation, erode confidence in our information security measures, and lead to regulatory scrutiny. To the extent that any disruption or security breach resulted in a loss of, or damage to, our data or systems, or inappropriate disclosure of confidential, proprietary or personal information, we could be exposed to a risk of loss, enforcement measures, penalties, fines, indemnification claims, litigation and potential civil or criminal liability, which could materially adversely affect our business, financial condition and results of operations.

Not all our contracts contain limitations of liability, and even where they do, there can be no assurance that limitations of liability in our contracts are sufficient to protect us from liabilities, damages, or claims related to our data privacy and security obligations. We cannot be sure that our insurance coverage will be adequate or sufficient to protect us from or to mitigate liabilities arising out of our privacy and security practices, that such coverage will continue to be available on commercially reasonable terms or at all, or that such coverage will pay future claims.

Further, the SEC has adopted new rules that require us to provide greater disclosures around proactive security protections that we employ and reactive issues (e.g., security incidents). Any such disclosures, including those under state data breach notification laws, can be costly, and the disclosures we make to comply with, or the failure to comply with, such requirements could lead to adverse consequences.

*If a natural disaster, terrorist or criminal activity or other unforeseen event caused significant damage to our facilities or those of our third-party manufacturers and suppliers or significantly disrupted our operations or those of our third-party manufacturers and suppliers, we may be unable to meet demand for our products and lose potential revenue, have reduced margins, or be forced to terminate a program.

The occurrence of an earthquake or other catastrophic disaster could cause damage to our facility and equipment, or that of our third-party manufacturers or single-source suppliers, which could materially impair the ability for us or our third-party manufacturers to manufacture our products and product candidates. Our Galli Drive facility, located in Novato, California, is currently our only manufacturing facility for ALDURAZYME, NAGLAZYME, VOXZOGO and PALYNZIQ and is one of two manufacturing facilities for BRINEURA and VIMIZIM. Our gene therapy manufacturing facility is also located in Novato, California, and it is currently our only manufacturing facility to support ongoing ROCTAVIAN clinical development activities and commercial demand for ROCTAVIAN. These facilities are located in the San Francisco Bay Area near known earthquake fault zones and are vulnerable to significant damage from earthquakes. We, the third-party manufacturers with whom we contract and our single-source suppliers of raw materials, which include many of our critical raw materials, are also vulnerable to damage from other types of disasters, including fires, explosions, floods, and similar events. If any disaster were to occur, or any terrorist or criminal activity caused significant damage to our facilities or the facilities of our third-party manufacturers and suppliers, our ability to manufacture our products, or to have our products manufactured, could be seriously, or potentially completely, impaired, and our commercialization efforts and revenues could be seriously impaired.

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Moreover, other unforeseen events, such as power outages, could significantly disrupt our operations or those of our third-party manufacturers and suppliers, which could result in significant delays in the manufacture of our products and adversely impact our commercial operations and revenues. Pacific Gas and Electric Company, the electric utility in the San Francisco Bay Area where many of our facilities are located, commenced widespread blackouts during the fall of 2019 to avoid and contain wildfires sparked during strong wind events by downed power lines or equipment failures. While we have not experienced damage to our facilities or material disruption to our operations as a result of these power outages, ongoing blackouts, particularly if prolonged or frequent, could impact our business going forward. The insurance that we carry, the inventory that we maintain and our risk mitigation plans may not be adequate to cover our losses resulting from disasters or other business interruptions.

*Our business is affected by macroeconomic conditions.

Various macroeconomic factors could adversely affect our business and the results of our operations and financial condition, including changes in inflation, interest rates, or foreign currency exchange rates, banking crises, natural disasters, geopolitical instability resulting from war, terrorism and other violence, lasting effects of the COVID-19 pandemic or other global public health threats and overall economic conditions and uncertainties, including those resulting from the current and future conditions in the global financial markets and volatility and disruptions in the equity and debt markets. For instance, COVID-19 previously adversely affected our ability to source materials and supplies. Inflation (such as that recently observed in the U.S. and elsewhere) has increased our business costs and could become more significant in the future, and it may not be feasible to pass price increases on to our customers due to the process by which healthcare providers are reimbursed for our products by the government. Interest rates, the liquidity of the credit markets and the volatility of the capital markets could also affect the value of our investments and our ability to liquidate our investments in order to fund our operations. We purchase or enter into a variety of financial instruments and transactions, including investments in commercial paper, the extension of credit to corporations, institutions and governments and hedging contracts. If any of the issuers or counterparties to these instruments were to default on their obligations, it could materially reduce the value of the transaction and adversely affect our cash flows.

We sell our products in countries that face economic volatility and weakness. Although we have historically collected receivables from customers in those countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to be unable to pay for our products. Additionally, if one or more of these countries were unable to purchase our products, our revenues would be adversely affected.

Interest rates and the ability to access credit markets could also adversely affect the ability of our customers/distributors to purchase, pay for and effectively distribute our products, which could limit our ability to obtain sufficient materials and supplies necessary for production of our therapies. Similarly, these macroeconomic factors could affect the ability of our contract manufacturers, sole-source or single-source suppliers to remain in business or otherwise manufacture or supply product. Failure by any of them to remain a going concern could affect our ability to manufacture products.

Additionally, the lasting effects of the COVID-19 pandemic could continue to adversely affect our business, results of our operations, and financial condition. The COVID-19 pandemic impacted our global revenue sources and overall business operations by, for example, presenting challenges to our ability to find adequate resources to staff clinical trials, impacting patients’ ability to participate in trials, affecting our ability to source materials and supplies for trials and otherwise delaying trials, and the pandemic could continue to adversely impact our financial results and our business generally in 2023. Effects of any future pandemic or other global public health threat on all aspects of our business and operations and the duration of such effects are highly uncertain and difficult to predict. Moreover, while the long-term economic impact of the COVID-19 pandemic may be difficult to predict, the pandemic has resulted in, and may continue to result in, significant disruption of global financial markets, which could reduce our ability to access capital and could negatively affect our liquidity and the liquidity and stability of markets for our common stock and Notes. In addition, a recession, further market correction or depression resulting from a future global public health threat could materially adversely affect our business and the value of our common stock and Notes.

To the extent macroeconomic conditions continue to adversely affect our business and financial results, they may also have the effect of heightening many of the other risks described in this Risk Factors section, such as those relating to our conducting a significant amount of our sales and operations outside of the U.S., exposure to changes in foreign exchange rates, our need to generate sufficient cash flows to service our indebtedness and finance our operations and the volatility of our stock price.

Item 2.    Unregistered Sales of Equity Securities and Use of Proceeds

None.

Item 3.    Defaults Upon Senior Securities

None.

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Item 4.    Mine Safety Disclosures

Not applicable.

Item 5.    Other Information

Rule 10b5-1 Trading Plans

During the three months ended September 30, 2023, our directors and officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted or terminated the contracts, instructions or written plans for the purchase or sale of BioMarin securities set forth in the table below.

Type of Trading Arrangement
Name	Position	Action	Adoption/Termination Date	Rule 10b5-1*	Non- Rule 10b5-1**	Total Shares of Common Stock to be Sold***	Expiration Date
Jean-Jacques Bienaimé	Chairman and Chief Executive Officer	Adoption	August 7, 2023	X		up to 191,000	June 3, 2024
G. Eric Davis	Executive Vice President, Chief Legal Officer and Secretary	Adoption	September 13, 2023	X		up to 164,429****	August 1, 2024
Henry J. Fuchs, M.D.	President of Worldwide Research & Development	Adoption	August 17, 2023	X		up to 64,659	November 15, 2024
Brian R. Mueller	Executive Vice President and Chief Financial Officer	Adoption	August 23, 2023	X		up to 12,495	July 30, 2024

*    Contract, instruction or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act.

**     “Non-Rule 10b5-1 trading arrangement” as defined in Item 408(c) of Regulation S-K under the Exchange Act.

***    Represents the maximum number of shares that may be sold pursuant to the 10b5-1 arrangement. The number of shares sold will be dependent on the satisfaction of certain conditions as set forth in the written plan.

**** Includes up to 41,680 shares subject to performance-based restricted stock units previously granted to Mr. Davis that may vest and be released to Mr. Davis on or before August 1, 2024 upon the satisfaction of the applicable vesting conditions. The actual number of shares that will be released to Mr. Davis pursuant to the performance-based restricted stock units and sold under the Rule 10b5-1 trading arrangement will be net of the number of shares withheld to satisfy tax withholding obligations arising from the vesting of such shares and is not yet determinable.

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Item 6.    Exhibits

Exhibit Number	Description
2.1	Amended and Restated Termination and Transition Agreement, dated as of December 23, 2015, between BioMarin Pharmaceutical Inc. and Ares Trading S.A., previously filed with the SEC on January 7, 2016 as Exhibit 2.1 to the Company’s Current Report on Form 8-K (File No. 000-26727), which is incorporated herein by reference. Portions of this exhibit (indicated by asterisks) have been omitted pursuant to a request for confidential treatment. Omitted portions have been filed separately with the SEC.
2.2	Termination and Transition Agreement, dated as of October 1, 2015, between BioMarin Pharmaceutical Inc. and Ares Trading S.A., previously filed with the SEC on January 7, 2016 as Exhibit 2.3 to the Company’s Current Report on Form 8-K (File No. 000-26727), which is incorporated herein by reference. Portions of this exhibit (indicated by asterisks) have been omitted pursuant to a request for confidential treatment. Omitted portions have been filed separately with the SEC.
2.3	First Amendment, dated as of December 12, 2016, to the Amended and Restated Termination and Transition Agreement, dated as of December 23, 2015 and effective as of October 1, 2015, between BioMarin Pharmaceutical Inc. and Ares Trading S.A., previously filed with the SEC on February 27, 2017 as Exhibit 2.6 to the Company’s Annual Report on Form 10-K (File No. 000-26727), which is incorporated herein by reference. Portions of this exhibit (indicated by asterisks) have been omitted pursuant to a request for confidential treatment. Omitted portions have been filed separately with the SEC.
3.1	Restated Certificate of Incorporation of BioMarin Pharmaceutical Inc., previously filed with the SEC on June 12, 2017 as Exhibit 3.2 to the Company’s Current Report on Form 8-K (File No. 000-26727), which is incorporated herein by reference.
3.2	Amended and Restated Bylaws of BioMarin Pharmaceutical Inc., previously filed with the SEC on December 21, 2022 as Exhibit 3.1 to the Company’s Current Report on Form 8-K (File No. 000-26727), which is incorporated herein by reference.
31.1*	Certification of Chief Executive Officer pursuant to Rules 13a-14(a)/15d-14(a) of the Securities Exchange Act of 1934, as amended.
31.2*	Certification of Chief Financial Officer pursuant to Rules 13a-14(a)/15d-14(a) of the Securities Exchange Act of 1934, as amended.
32.1*+	Certification of Chief Executive Officer and Chief Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002. This Certification accompanies this report and shall not, except to the extent required by the Sarbanes-Oxley Act of 2002, be deemed filed for purposes of §18 of the Securities Exchange Act of 1934, as amended.
101.INS	XBRL Instance Document - the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document.
101.SCH*	Inline XBRL Taxonomy Extension Schema Document
101.CAL*	Inline XBRL Taxonomy Extension Calculation Document
101.DEF*	Inline XBRL Taxonomy Extension Definition Linkbase
101.LAB*	Inline XBRL Taxonomy Extension Labels Linkbase Document
101.PRE*	Inline XBRL Taxonomy Extension Presentation Link Document
104	XBRL tags for the cover page from the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, are embedded within the Inline XBRL document.

*    Filed herewith

+    The certifications attached as Exhibit 32.1 accompany this Quarterly Report on Form 10-Q pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, and shall not be deemed “filed” by the Registrant for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, and are not to be incorporated by reference into any of the Registrant’s filings under the Securities Act of 1933, as amended, irrespective of any general incorporation language contained in any such filing.

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Attached as Exhibit 101 to this report are documents formatted in XBRL (Extensible Business Reporting Language):

(i) Condensed Consolidated Balance Sheets as of September 30, 2023 and December 31, 2022, (ii) Condensed Consolidated Statements of Comprehensive Income for the three and nine months ended September 30, 2023 and 2022, (iii) Condensed Consolidated Statement of Stockholders’ Equity for the three and nine months ended September 30, 2023 and 2022, (iv) Condensed Consolidated Statements of Cash Flows for the nine months ended September 30, 2023 and 2022, and (v) Notes to Condensed Consolidated Financial Statements.

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SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.

	BIOMARIN PHARMACEUTICAL INC.
Dated: November 2, 2023	By	/S/ BRIAN R. MUELLER
		Brian R. Mueller Executive Vice President, Finance & Chief Financial Officer (Principal Financial Officer)

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EX-31.1

Exhibit 31.1

CERTIFICATION

I, Jean-Jacques Bienaimé, certify that:

1.I have reviewed this Quarterly Report on Form 10-Q of BioMarin Pharmaceutical Inc.;

2.Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3.Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4.The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

a)designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;

b)designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

c)evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and

d)disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and

5.The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

a)all significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

b)any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.

Date: November 2, 2023

/S/ JEAN-JACQUES BIENAIMÉ 

Jean-Jacques Bienaimé
Chief Executive Officer

EX-31.2

Exhibit 31.2

CERTIFICATION

I, Brian R. Mueller certify that:

1.I have reviewed this Quarterly Report on Form 10-Q of BioMarin Pharmaceutical Inc.;

2.Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;

3.Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;

4.The registrant’s other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:

a)designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;

b)designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;

c)evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and

d)disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and

5.The registrant’s other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):

a)all significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and

b)any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.

Date: November 2, 2023

/S/ BRIAN R. MUELLER

Brian R. Mueller
Executive Vice President, Finance &
Chief Financial Officer

EX-32.1

Exhibit 32.1

CERTIFICATION PURSUANT TO

18 U.S.C. SECTION 1350,

AS ADOPTED PURSUANT TO

SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002

We, Jean-Jacques Bienaimé and Brian R. Mueller, hereby certify, pursuant to 18 U.S.C. §1350, as adopted pursuant to §906 of the Sarbanes-Oxley Act of 2002, that BioMarin Pharmaceutical Inc.’s Quarterly Report on Form 10-Q for the period ended September 30, 2023, fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934 and the information contained in such Form 10-Q fairly presents, in all material respects, the financial condition and results of operations of BioMarin Pharmaceutical Inc.

/s/ JEAN-JACQUES BIENAIMÉ

Jean-Jacques Bienaimé
Chief Executive Officer

Date: November 2, 2023

/s/ BRIAN R. MUELLER

Brian R. Mueller
Executive Vice President, Finance &
Chief Financial Officer

Date: November 2, 2023

This certification accompanies the Quarterly Report on Form 10-Q to which it relates, is not deemed filed with the Securities and Exchange Commission and is not to be incorporated by reference into any filing of BioMarin Pharmaceutical Inc. under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended (whether made before or after the date of the Quarterly Report on Form 10-Q), irrespective of any general incorporation language contained in such filing.

XML

Cover Page - shares	9 Months Ended
	Sep. 30, 2023	Oct. 30, 2023
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2023
Document Transition Report	false
Entity File Number	000-26727
Entity Registrant Name	BioMarin Pharmaceutical Inc
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	68-0397820
Entity Address, Address Line One	770 Lindaro Street
Entity Address, City or Town	San Rafael
Entity Address, State or Province	CA
Entity Address, Postal Zip Code	94901
City Area Code	415
Local Phone Number	506-6700
Title of 12(b) Security	Common Stock, par value $0.001
Trading Symbol	BMRN
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		188,368,171
Amendment Flag	false
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q3
Entity Central Index Key	0001048477
Current Fiscal Year End Date	--12-31

XML

CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022	[1]
Current assets:
Cash and cash equivalents	$ 785,414	$ 724,531
Short-term investments	340,431	567,006
Accounts receivable, net	572,498	461,316
Inventory	1,032,159	894,083
Other current assets	224,806	104,521
Total current assets	2,955,308	2,751,457
Noncurrent assets:
Long-term investments	548,002	333,835
Property, plant and equipment, net	1,067,156	1,073,366
Intangible assets, net	302,476	338,569
Goodwill	196,199	196,199
Deferred tax assets	1,523,953	1,505,412
Other assets	165,069	176,236
Total assets	6,758,163	6,375,074
Current liabilities:
Accounts payable and accrued liabilities	669,825	572,959
Short-term convertible debt, net	493,398	0
Short-term contingent consideration	0	15,925
Total current liabilities	1,163,223	588,884
Noncurrent liabilities:
Long-term convertible debt, net	592,586	1,083,019
Other long-term liabilities	105,457	100,015
Total liabilities	1,861,266	1,771,918
Stockholders’ equity:
Common stock, $0.001 par value: 500,000,000 shares authorized; 188,339,482 and 186,250,719 shares issued and outstanding, respectively	188	186
Additional paid-in capital	5,542,774	5,404,895
Company common stock held by the Nonqualified Deferred Compensation Plan (the NQDC)	(10,393)	(8,859)
Accumulated other comprehensive income (loss)	6,257	(3,867)
Accumulated deficit	(641,929)	(789,199)
Total stockholders’ equity	4,896,897	4,603,156	[2]
Total liabilities and stockholders’ equity	$ 6,758,163	$ 6,375,074

[1] (1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

[2] The beginning balances for the nine-month periods were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 filed with the SEC on February 27, 2023.

XML

CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares	Sep. 30, 2023	Dec. 31, 2022
Statement of Financial Position [Abstract]
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock, shares authorized (in shares)	500,000,000	500,000,000
Common stock, shares issued (in shares)	188,339,482	186,250,719
Common stock, shares outstanding (in shares)	188,339,482	186,250,719

XML

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME - USD ($) shares in Thousands, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
REVENUES:
Total revenues	$ 581,329	$ 505,344	$ 1,773,019	$ 1,558,501
OPERATING EXPENSES:
Cost of sales	124,745	116,288	379,376	356,379
Research and development	191,314	157,829	540,523	476,855
Selling, general and administrative	223,928	216,816	662,267	608,270
Intangible asset amortization and contingent consideration	15,681	16,828	46,975	50,935
Gain on sale of nonfinancial assets, net	0	0	0	(108,000)
Total operating expenses	555,668	507,761	1,629,141	1,384,439
INCOME (LOSS) FROM OPERATIONS	25,661	(2,417)	143,878	174,062
Interest income	15,740	4,999	40,295	9,324
Interest expense	(3,779)	(4,679)	(11,237)	(12,344)
Other income (expense), net	4,047	193	(3,700)	(3,908)
INCOME (LOSS) BEFORE INCOME TAXES	41,669	(1,904)	169,236	167,134
Provision for income taxes	1,291	4,748	21,966	25,324
NET INCOME (LOSS)	$ 40,378	$ (6,652)	$ 147,270	$ 141,810
EARNINGS (LOSS) PER SHARE, BASIC (in dollars per share)	$ 0.21	$ (0.04)	$ 0.78	$ 0.77
EARNINGS (LOSS) PER SHARE, DILUTED (in dollars per share)	$ 0.21	$ (0.04)	$ 0.77	$ 0.75
Weighted average common shares outstanding, basic (in shares)	188,219	185,597	187,617	185,009
Weighted average common shares outstanding, diluted (in shares)	191,173	185,597	195,042	192,252
COMPREHENSIVE INCOME	$ 65,252	$ 5,625	$ 157,394	$ 158,127
Net product revenues
REVENUES:
Total revenues	568,266	493,348	1,739,390	1,516,533
Royalty and other revenues
REVENUES:
Total revenues	$ 13,063	$ 11,996	$ 33,629	$ 41,968

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CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands		Total		Common stock:		Additional paid-in capital:	Company common stock held by the NQDC:	Accumulated other comprehensive income (loss):	Accumulated Deficit:
Shares of common stock, beginning balances (in shares) at Dec. 31, 2021	[1]			183,913,000
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuances under equity incentive plans (in shares)				1,912,000
Shares of common stock, ending balances (in shares) at Sep. 30, 2022				185,825,000
Beginning balances at Dec. 31, 2021	[1]	$ 4,265,670		$ 184		$ 5,191,502	$ (9,689)	$ 14,432	$ (930,759)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuances under equity incentive plans, net of tax				2		(8,306)
Stock-based compensation						153,021
Common stock held by the NQDC						(364)	364
Other comprehensive income		16,317						16,317
Net income (loss)		141,810							141,810
Ending balances at Sep. 30, 2022		4,568,514		$ 186		5,335,853	(9,325)	30,749	(788,949)
Shares of common stock, beginning balances (in shares) at Jun. 30, 2022	[1]			185,453,000
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuances under equity incentive plans (in shares)				372,000
Shares of common stock, ending balances (in shares) at Sep. 30, 2022				185,825,000
Beginning balances at Jun. 30, 2022	[1]	4,499,737		$ 186		5,272,666	(9,290)	18,472	(782,297)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuances under equity incentive plans, net of tax						6,847
Stock-based compensation						56,305
Common stock held by the NQDC						35	(35)
Other comprehensive income		12,277						12,277
Net income (loss)		(6,652)							(6,652)
Ending balances at Sep. 30, 2022		$ 4,568,514		$ 186		5,335,853	(9,325)	30,749	(788,949)
Shares of common stock, beginning balances (in shares) at Dec. 31, 2022		186,250,719		186,251,000	[1]
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuances under equity incentive plans (in shares)				2,088,000
Shares of common stock, ending balances (in shares) at Sep. 30, 2023		188,339,482		188,339,000
Beginning balances at Dec. 31, 2022	[1]	$ 4,603,156	[2]	$ 186		5,404,895	(8,859)	(3,867)	(789,199)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuances under equity incentive plans, net of tax				2		(19,778)
Stock-based compensation						156,123
Common stock held by the NQDC						1,534	(1,534)
Other comprehensive income		10,124						10,124
Net income (loss)		147,270							147,270
Ending balances at Sep. 30, 2023		$ 4,896,897		$ 188		5,542,774	(10,393)	6,257	(641,929)
Shares of common stock, beginning balances (in shares) at Jun. 30, 2023	[1]			188,152,000
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuances under equity incentive plans (in shares)				187,000
Shares of common stock, ending balances (in shares) at Sep. 30, 2023		188,339,482		188,339,000
Beginning balances at Jun. 30, 2023	[1]	$ 4,782,827		$ 188		5,493,956	(10,393)	(18,617)	(682,307)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuances under equity incentive plans, net of tax						(1,827)
Stock-based compensation						50,645
Other comprehensive income		24,874						24,874
Net income (loss)		40,378							40,378
Ending balances at Sep. 30, 2023		$ 4,896,897		$ 188		$ 5,542,774	$ (10,393)	$ 6,257	$ (641,929)

[1] The beginning balances for the nine-month periods were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 filed with the SEC on February 27, 2023.

[2] (1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

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CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022
CASH FLOWS FROM OPERATING ACTIVITIES:
Net income	$ 147,270	$ 141,810
Adjustments to reconcile net income to net cash used in operating activities:
Depreciation and amortization	77,525	77,416
Non-cash interest expense	3,198	3,089
Amortization of premium (accretion of discount) on investments	(6,781)	3,741
Stock-based compensation	152,244	149,574
Gain on sale of nonfinancial assets, net	0	(108,000)
Loss on equity investment	12,650	0
Deferred income taxes	(20,137)	(743)
Unrealized foreign exchange loss (gain)	5,454	(16,075)
Non-cash changes in the fair value of contingent consideration	0	2,243
Other	(224)	(700)
Changes in operating assets and liabilities:
Accounts receivable, net	(131,940)	(53,752)
Inventory	(97,948)	(27,419)
Other current assets	(59,389)	(8,558)
Other assets	(20,812)	12,140
Accounts payable and other short-term liabilities	56,333	(2,398)
Other long-term liabilities	14,333	(3,252)
Net cash provided by operating activities	131,776	169,116
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property, plant and equipment	(67,774)	(85,271)
Maturities and sales of investments	751,677	477,244
Purchases of investments	(727,043)	(457,382)
Proceeds from sale of nonfinancial assets	0	110,000
Purchase of intangible assets	(3,141)	(9,910)
Net cash provided by (used in) investing activities	(46,281)	34,681
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from exercises of awards under equity incentive plans	54,548	43,866
Taxes paid related to net share settlement of equity awards	(72,399)	(50,696)
Payments of contingent consideration	(9,475)	(21,054)
Principal repayments of financing leases	(2,241)	(1,635)
Net cash used in financing activities	(29,567)	(29,519)
Effect of exchange rate changes on cash	4,955	(39)
NET INCREASE IN CASH AND CASH EQUIVALENTS	60,883	174,239
Cash and cash equivalents:
Beginning of period	724,531	587,276
End of period	785,414	761,515
SUPPLEMENTAL CASH FLOW DISCLOSURES:
Cash paid for interest	6,640	6,575
Cash paid for income taxes	51,687	13,425
SUPPLEMENTAL CASH FLOW DISCLOSURES FOR NON-CASH INVESTING AND FINANCING ACTIVITIES:
Increase (decrease) in accounts payable and accrued liabilities related to fixed assets	7,977	(5,240)
Increase (decrease) in accounts payable and accrued liabilities related to intangible assets	$ 6,449	$ (224)

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BUSINESS OVERVIEW AND SIGNIFICANT ACCOUNTING POLICIES

9 Months Ended

Sep. 30, 2023

Accounting Policies [Abstract]

BUSINESS OVERVIEW AND SIGNIFICANT ACCOUNTING POLICIES

BUSINESS OVERVIEW AND SIGNIFICANT ACCOUNTING POLICIES

Nature of Operations

Founded in 1997, BioMarin Pharmaceutical Inc. (the Company or BioMarin) is a global biotechnology company dedicated to transforming lives through genetic discovery. The Company develops and commercializes targeted therapies that address the root cause of genetic conditions. The Company's robust research and development (R&D) capabilities have resulted in multiple innovative commercial therapies for patients with rare genetic disorders. The Company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options.

Basis of Presentation

These Condensed Consolidated Financial Statements have been prepared pursuant to U.S. generally accepted accounting principles (U.S. GAAP) and the rules and regulations of the SEC for Quarterly Reports on Form 10-Q and do not include all of the information and note disclosures required by U.S. GAAP for complete financial statements, although management believes that the disclosures herein are adequate to ensure that the information presented is not misleading. The Condensed Consolidated Financial Statements should therefore be read in conjunction with the Consolidated Financial Statements and Notes thereto for the fiscal year ended December 31, 2022 included in the Company’s Annual Report on Form 10-K. The Condensed Consolidated Financial Statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany transactions have been eliminated. The results of operations for the three and nine months ended September 30, 2023 are not necessarily indicative of the results that may be expected for the fiscal year ending December 31, 2023 or any other period.

Use of Estimates

U.S. GAAP requires management to make estimates and assumptions that affect amounts reported in the Condensed Consolidated Financial Statements and accompanying disclosures. Although these estimates are based on management’s best knowledge of current events and actions that the Company may undertake in the future, actual results may be different from those estimates. The Condensed Consolidated Financial Statements reflect all adjustments of a normal, recurring nature that are, in the opinion of management, necessary for a fair presentation of results for these interim periods.

Management performed an evaluation of the Company’s activities through the date of filing of this Quarterly Report on Form 10-Q and has concluded that there were no subsequent events or transactions that occurred subsequent to the balance sheet date prior to filing this Quarterly Report on Form 10-Q that would require recognition or disclosure in the Condensed Consolidated Financial Statements.

Significant Accounting Policies

There have been no material changes to the Company’s significant accounting policies during the nine months ended September 30, 2023, as compared to the significant accounting policies disclosed in Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.

Recent Accounting Pronouncements

There have been no new accounting pronouncements adopted by the Company or new accounting pronouncements issued by the Financial Accounting Standards Board during the nine months ended September 30, 2023, as compared to the recent accounting pronouncements described in Note 1 to the Company’s Consolidated Financial Statements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, that the Company believes are of significance or potential significance to the Company.

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FINANCIAL INSTRUMENTS

9 Months Ended

Sep. 30, 2023

Investments, Debt and Equity Securities [Abstract]

FINANCIAL INSTRUMENTS

FINANCIAL INSTRUMENTS

All marketable securities were classified as available-for-sale as of September 30, 2023 and December 31, 2022.

The following tables show the Company’s cash, cash equivalents and available-for-sale securities by significant investment category for each period presented:

	September 30, 2023
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash	$	451,294	$	—	$	—	$	451,294	$	451,294	$	—	$	—
Level 2:
Money market instruments	291,425		—		—		291,425		291,425		—		—
Corporate debt securities	563,598		31		(6,135)		557,494		731		217,306		339,457
U.S. government agency securities	281,045		4		(3,023)		278,026		29,981		112,714		135,331
Commercial paper	21,864		—		(4)		21,860		11,983		9,877		—
Asset-backed securities	74,168		26		(446)		73,748		—		534		73,214
Subtotal	1,232,100		61		(9,608)		1,222,553		334,120		340,431		548,002
Total	$	1,683,394	$	61	$	(9,608)	$	1,673,847	$	785,414	$	340,431	$	548,002

	December 31, 2022
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash	$	463,248	$	—	$	—	$	463,248	$	463,248	$	—	$	—
Level 2:
Money market instruments	248,933		—		—		248,933		248,933		—		—
Corporate debt securities	504,984		34		(11,541)		493,477		1,881		299,153		192,443
U.S. government agency securities	312,720		45		(3,771)		308,994		—		229,846		79,148
Commercial paper	48,103		11		(22)		48,092		10,469		37,623		—
Asset-backed securities	63,151		69		(592)		62,628		—		384		62,244
Subtotal	1,177,891		159		(15,926)		1,162,124		261,283		567,006		333,835
Total	$	1,641,139	$	159	$	(15,926)	$	1,625,372	$	724,531	$	567,006	$	333,835

(1)    The Company’s short-term marketable securities mature in one year or less.

(2)    The Company’s long-term marketable securities mature between one and five years.

As of September 30, 2023, the Company had the ability and intent to hold all investments that were in an unrealized loss position until maturity. The Company considered its intent and ability to hold the securities until recovery of amortized cost basis, the extent to which fair value is less than amortized cost basis, conditions specifically related to the security’s industry and geography, payment structure and history and changes to the ratings (if any) in determining that the decline in fair value compared to carrying value is not related to a credit loss.

The Company has certain investments in non-marketable equity securities, measured using unobservable valuation inputs and remeasured on a nonrecurring basis, which are collectively considered strategic investments. As of September 30, 2023 and December 31, 2022, the fair value of the Company’s strategic investments was $11.2 million and $23.9 million, respectively. These investments were recorded to Other Assets in the Company’s Condensed Consolidated Balance Sheets. In the first quarter of 2023, based on new developments, the Company concluded that factors existed indicating it would no longer realize a $12.6 million equity investment in its non-marketable securities. The loss on the equity investment due to impairment was recorded to Other Income (Expense), Net on the Company’s Condensed Consolidated Statements of Comprehensive Income. See Note 1 - Business Overview and Significant Accounting Policies included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 for additional information related to the Company’s non-marketable securities policy.

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SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION

9 Months Ended

Sep. 30, 2023

Organization, Consolidation and Presentation of Financial Statements [Abstract]

SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION

SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION

Supplemental Balance Sheet Information

Inventory consisted of the following:

	September 30, 2023		December 31, 2022
Raw materials	$	142,133	$	131,071
Work-in-process	518,893		410,656
Finished goods	371,133		352,356
Total inventory	$	1,032,159	$	894,083

Property, Plant and Equipment, Net consisted of the following:

	September 30, 2023		December 31, 2022
Property, plant and equipment, gross	$	1,918,609	$	1,875,511
Accumulated depreciation	(851,453)		(802,145)
Total property, plant and equipment, net	$	1,067,156	$	1,073,366

Depreciation expense, net of amounts capitalized into inventory, for the three and nine months ended September 30, 2023 was $9.9 million and $29.6 million, respectively. Depreciation expense, net of amounts capitalized into inventory, for the three and nine months ended September 30, 2022 was $8.7 million and $29.9 million, respectively.

Intangible Assets, Net consisted of the following:

	September 30, 2023		December 31, 2022
Finite-lived intangible assets	$	701,793	$	690,871
Accumulated amortization	(399,317)		(352,302)
Net carrying value	$	302,476	$	338,569

    

Accounts Payable and Accrued Liabilities consisted of the following:

	September 30, 2023		December 31, 2022
Accounts payable and accrued operating expenses	$	301,369	$	231,238
Accrued compensation expense	175,183		207,573
Accrued rebates payable	103,379		72,654
Accrued litigation expense	39,000		—
Accrued royalties payable	12,293		13,306
Accrued income taxes	11,273		16,213
Foreign currency exchange forward contracts	10,584		12,601
Lease liability	9,324		10,375
Other	7,420		8,999
Total accounts payable and accrued liabilities	$	669,825	$	572,959

Reorganization Plan

On October 6, 2022, the Company announced a plan to simplify its organization that included a planned reduction in force of approximately 120 employees (representing approximately 4% of its global workforce), which was substantially completed as of December 31, 2022. The Company recorded costs of $23.0 million in 2022 and negligible adjustments in 2023 related to one-time

severance and employee termination benefits within Selling, General and Administrative in the Company’s Condensed Consolidated Statements of Comprehensive Income. As of December 31, 2022, $11.1 million was included in Accounts Payable and Accrued Liabilities on the Company’s Consolidated Balance Sheet. As of September 30, 2023, substantially all costs have been paid.

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FAIR VALUE MEASUREMENTS

9 Months Ended

Sep. 30, 2023

Fair Value Disclosures [Abstract]

FAIR VALUE MEASUREMENTS

FAIR VALUE MEASUREMENTS

The Company measures certain financial assets and liabilities at fair value in accordance with the policy described in Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.

The following tables present the classification within the fair value hierarchy of financial assets and liabilities not disclosed elsewhere in these Condensed Consolidated Financial Statements that are remeasured on a recurring basis as of September 30, 2023 and December 31, 2022. Other than the Company’s fixed-rate convertible debt disclosed in Note 6 – Debt, there were no financial assets or liabilities that were remeasured using a quoted price in active markets for identical assets (Level 1) as of September 30, 2023 or December 31, 2022.

	Fair Value Measurements as of September 30, 2023
		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Other current assets:
NQDC Plan assets		$	1,836	$	—	$	1,836
Other assets:
NQDC Plan assets		25,541		—		25,541
Restricted investments (1)		2,361		—		2,361
Total other assets		27,902		—		27,902
Total assets		$	29,738	$	—	$	29,738
Liabilities:
Current liabilities:
NQDC Plan liability		$	1,836	$	—	$	1,836
Other long-term liabilities:
NQDC Plan liability		25,541		—		25,541
Total liabilities		$	27,377	$	—	$	27,377

	Fair Value Measurements as of December 31, 2022
	Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Other current assets:
NQDC Plan assets	$	2,654	$	—	$	2,654
Other assets:
NQDC Plan assets	19,867		—		19,867
Restricted investments (1)	2,429		—		2,429
Total other assets	22,296		—		22,296
Total assets	$	24,950	$	—	$	24,950
Liabilities:
Current liabilities:
NQDC Plan liability	$	2,654	$	—	$	2,654
Contingent consideration	—		15,925		15,925
Total current liabilities	2,654		15,925		18,579
Other long-term liabilities:
NQDC Plan liability	19,867		—		19,867
Total liabilities	$	22,521	$	15,925	$	38,446

(1)    The restricted investments as of September 30, 2023 and December 31, 2022 secure the Company's irrevocable standby letters of credit obtained in connection with certain commercial agreements.

There were no transfers between levels during the three and nine months ended September 30, 2023.

Liabilities measured at fair value using Level 3 inputs consisted of contingent consideration. The following table represents a roll-forward of contingent consideration.

Contingent consideration as of December 31, 2022	$	15,925
Milestone payments to Ares Trading S.A. (Merck Serono)	(16,255)
Realized foreign exchange loss on settlement of contingent consideration	330
Contingent consideration as of September 30, 2023	$	—

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DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES

9 Months Ended

Sep. 30, 2023

Derivative Instruments and Hedging Activities Disclosure [Abstract]

DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES

DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIESThe Company uses foreign currency exchange forward contracts (forward contracts) to protect against the impact of changes in the value of forecasted foreign currency cash flows resulting from revenues and operating expenses denominated in currencies other than the U.S. Dollar (USD), primarily the Euro. Certain of these forward contracts are designated as cash flow hedges and have maturities of up to two years. The Company also enters into forward contracts to manage foreign exchange risk related to asset or liability positions denominated in currencies other than USD. Such forward contracts are considered to be economic hedges, are not designated as hedging instruments and have maturities of up to six months. The Company does not use derivative instruments for speculative trading purposes. The Company is exposed to counterparty credit risk on its derivatives. The Company has established and maintains strict counterparty credit guidelines and enters into hedging agreements with financial institutions that are investment grade or better to minimize the Company’s exposure to potential defaults. The Company is not required to pledge collateral under these agreements.

The following table summarizes the aggregate notional amounts for the Company’s derivatives outstanding as of the periods presented.

Forward Contracts	September 30, 2023		December 31, 2022
Derivatives designated as hedging instruments:
Sell	$	988,286	$	808,635
Purchase	$	180,560	$	177,393
Derivatives not designated as hedging instruments:
Sell	$	301,825	$	218,903
Purchase	$	6,517	$	6,785

The fair value carrying amounts of the Company’s derivatives, which are classified as Level 2 within the fair value hierarchy, were as follows:

Balance Sheet Location	September 30, 2023		December 31, 2022
Derivatives designated as hedging instruments:
Asset Derivatives
Other current assets	$	21,267	$	19,464
Other assets	7,620		2,059
Subtotal	$	28,887	$	21,523
Liability Derivatives
Accounts payable and accrued liabilities	$	9,918	$	12,130
Other long-term liabilities	3,623		1,074
Subtotal	$	13,541	$	13,204
Derivatives not designated as hedging instruments:
Asset Derivatives
Other current assets	$	5,210	$	1,472
Liability Derivatives
Accounts payable and accrued liabilities	$	666	$	471
Total Derivatives Assets	$	34,097	$	22,995
Total Derivatives Liabilities	$	14,207	$	13,675

For additional discussion of fair value measurements, see Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.

The following tables summarize the impact of gains and losses from the Company's derivatives on its Condensed Consolidated Statements of Comprehensive Income for the periods presented.

	Three Months Ended September 30,
	2023			2022
Derivatives Designated as Cash Flow Hedging Instruments		Cash Flow Hedging Gains (Losses) Reclassified into Earnings		Cash Flow Hedging Gains (Losses) Reclassified into Earnings
Net product revenues		$	(4,636)	$	15,280
Operating expenses		$	908	$	(3,936)
Derivatives Not Designated as Hedging Instruments		Gains (Losses) Recognized in Earnings		Gains (Losses) Recognized in Earnings
Operating expenses		$	6,559	$	6,572

	Nine Months Ended September 30,
	2023			2022
Derivatives Designated as Cash Flow Hedging Instruments		Cash Flow Hedging Gains (Losses) Reclassified into Earnings		Cash Flow Hedging Gains (Losses) Reclassified into Earnings
Net product revenues		$	(489)	$	26,779
Operating expenses		$	426	$	(7,115)
Derivatives Not Designated as Hedging Instruments		Gains (Losses) Recognized in Earnings		Gains (Losses) Recognized in Earnings
Operating expenses		$	3,165	$	12,812

As of September 30, 2023, the Company expects to reclassify unrealized gains of $9.6 million from Accumulated Other Comprehensive Income (AOCI) to earnings as the forecasted revenues and operating expense transactions occur over the next twelve months. For additional discussion of balances in AOCI see Note 7 – Accumulated Other Comprehensive Income.

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DEBT

9 Months Ended

Sep. 30, 2023

Debt Disclosure [Abstract]

DEBT

DEBT

Convertible Notes

As of September 30, 2023, the Company had outstanding fixed-rate notes with varying maturities for an undiscounted aggregate principal amount of $1.1 billion (collectively, the Notes). The Notes are senior subordinated convertible obligations, and interest is payable in arrears, semi-annually. The following table summarizes information regarding the Company’s convertible debt:

	September 30, 2023		December 31, 2022
1.25% senior subordinated convertible notes due in May 2027 (the 2027 Notes)	$	600,000	$	600,000
Unamortized discount net of deferred offering costs	(7,414)		(8,941)
2027 Notes, net	592,586		591,059
0.599% senior subordinated convertible notes due in August 2024 (the 2024 Notes)	495,000		495,000
Unamortized discount net of deferred offering costs	(1,602)		(3,040)
2024 Notes, net (1)	493,398		491,960
Total convertible debt, net	$	1,085,984	$	1,083,019
Fair value of fixed-rate convertible debt (2):
2027 Notes	$	595,278	$	647,370
2024 Notes	480,417		526,230
Total fair value of fixed-rate convertible debt	$	1,075,695	$	1,173,600

(1)    As the 2024 Notes mature in August 2024, the outstanding principal of the 2024 Notes is classified as a current liability as of September 30, 2023.

(2)     The fair value of the Company’s fixed-rate convertible debt is based on open-market trades and is classified as Level 1 in the fair value hierarchy. For additional discussion of fair value measurements, see Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.

Interest expense on the Company’s convertible debt consisted of the following: 

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Coupon interest expense	$	2,616	$	2,616	$	7,849	$	7,849
Accretion of discount on convertible notes	841		838		2,519		2,511
Amortization of debt issuance costs	148		148		445		445
Total interest expense on convertible debt	$	3,605	$	3,602	$	10,813	$	10,805

See Note 10 - Debt to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 for additional information related to the Company’s convertible debt.

Revolving Credit Facility

In October 2018, the Company entered into an unsecured revolving credit facility of up to $200.0 million which included a letter of credit subfacility and a swingline loan subfacility. The credit facility was intended to finance ongoing working capital needs and for other general corporate purposes. In May 2021, the credit facility was amended to extend the original maturity date from October 19, 2021 to May 28, 2024. The credit facility was terminated on August 4, 2023 and there were no amounts outstanding under the credit facility as of September 30, 2023.

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ACCUMULATED OTHER COMPREHENSIVE INCOME

9 Months Ended

Sep. 30, 2023

Equity [Abstract]

ACCUMULATED OTHER COMPREHENSIVE INCOME

ACCUMULATED OTHER COMPREHENSIVE INCOME

The following tables summarize changes in the accumulated balances for each component of AOCI, including current-period other comprehensive income and reclassifications out of AOCI, for the periods presented.

	Three Months Ended September 30, 2023
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of June 30, 2023	$	(10,035)	$	(8,582)	$	(18,617)
Other comprehensive income (loss) before      reclassifications	19,892		1,634		21,526
Less: gain (loss) reclassified from AOCI	(3,728)		—		(3,728)
Tax effect	—		(380)		(380)
Net current-period other comprehensive income (loss)	23,620		1,254		24,874
AOCI balance as of September 30, 2023	$	13,585	$	(7,328)	$	6,257

	Three Months Ended September 30, 2022
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of June 30, 2022	$	30,515	$	(12,043)	$	18,472
Other comprehensive income (loss) before      reclassifications	26,698		(4,011)		22,687
Less: gain (loss) reclassified from AOCI	11,344		—		11,344
Tax effect	—		934		934
Net current-period other comprehensive income (loss)	15,354		(3,077)		12,277
AOCI balance as of September 30, 2022	$	45,869	$	(15,120)	$	30,749

	Nine Months Ended September 30, 2023
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of December 31, 2022	$	8,226	$	(12,093)	$	(3,867)
Other comprehensive income (loss) before      reclassifications	5,296		6,220		11,516
Less: gain (loss) reclassified from AOCI	(63)		—		(63)
Tax effect	—		(1,455)		(1,455)
Net current-period other comprehensive income (loss)	5,359		4,765		10,124
AOCI balance as of September 30, 2023	$	13,585	$	(7,328)	$	6,257

	Nine Months Ended September 30, 2022
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of December 31, 2021	$	15,805	$	(1,373)	$	14,432
Other comprehensive income (loss) before      reclassifications	49,728		(17,905)		31,823
Less: gain (loss) reclassified from AOCI	19,664		—		19,664
Tax effect	—		4,158		4,158
Net current-period other comprehensive income (loss)	30,064		(13,747)		16,317
AOCI balance as of September 30, 2022	$	45,869	$	(15,120)	$	30,749

For additional discussion of reclassifications from AOCI see Note 5 – Derivative Instruments and Hedging Strategies.

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REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION

9 Months Ended

Sep. 30, 2023

Concentration Risk And Geographic Information [Abstract]

REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION

REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION

The Company operates in one business segment, which primarily focuses on the development and commercialization of innovative therapies for people with serious and life-threatening rare diseases and medical conditions.

The following table disaggregates total Net Product Revenues by product.

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Enzyme product revenues:
VIMIZIM	$	158,869	$	155,470	$	525,453	$	511,735
NAGLAZYME	108,916		99,506		322,040		343,320
PALYNZIQ	78,885		66,206		216,105		182,734
BRINEURA	41,042		37,793		118,244		111,691
ALDURAZYME	13,812		29,046		88,533		90,755
Total enzyme product revenues	401,524		388,021		1,270,375		1,240,235
VOXZOGO	122,973		48,289		324,146		102,321
KUVAN	42,947		57,038		144,047		173,977
ROCTAVIAN	822		—		822		—
Total net product revenues	568,266		493,348		1,739,390		1,516,533
Royalty and other revenues	13,063		11,996		33,629		41,968
Total revenues	$	581,329	$	505,344	$	1,773,019	$	1,558,501

The Company considers there to be revenue concentration risks for regions where Net Product Revenues exceed 10% of consolidated Net Product Revenues. The concentration of the Company’s Net Product Revenues within the regions below may have a material adverse effect on the Company’s revenues and results of operations if sales in the respective regions experience difficulties. The table below disaggregates total Net Product Revenues by geographic region, which is based on patient location for Company's commercial products sold directly by the Company, except for ALDURAZYME, which is marketed and sold exclusively by Sanofi worldwide.

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
United States	$	194,745	$	176,552	$	553,184	$	497,205
Europe	168,784		162,583		512,910		480,645
Latin America	81,524		60,791		224,716		213,246
Rest of world	109,401		64,376		360,047		234,682
Total net product revenues marketed by the Company	$	554,454	$	464,302	$	1,650,857	$	1,425,778
ALDURAZYME net product revenues marketed by Sanofi	13,812		29,046		88,533		90,755
Total net product revenues	$	568,266	$	493,348	$	1,739,390	$	1,516,533

The following table illustrates the percentage of the Company’s total Net Product Revenues attributed to the Company’s largest customers for the periods presented. 

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Customer A	15	%	15	%	15	%	16	%
Customer B	13	%	14	%	12	%	12	%
Customer C	10	%	10	%	10	%	9	%
Total	38	%	39	%	37	%	37	%

On a consolidated basis, two customers accounted for 16% and 11% of the Company’s September 30, 2023 accounts receivable balance, respectively, compared to December 31, 2022, when two customers accounted for 22% and 15% of the accounts receivable balance, respectively. As of September 30, 2023, and December 31, 2022, the accounts receivable balance for Sanofi included $54.8 million and $68.8 million, respectively, of unbilled accounts receivable, which becomes payable to the Company when the product is sold through by Sanofi. The Company does not require collateral from its customers, but does perform periodic credit evaluations of its customers’ financial condition and requires prepayments in certain circumstances.

The Company is mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, banking crises, natural disasters, the continuing effects of the COVID-19 pandemic, and supply chain disruptions, could affect the Company’s ability to achieve its goals. In addition, the Company sells its products in countries that face economic volatility and weakness. Although the Company has historically collected receivables from customers in certain countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to delay payment or be unable to pay for the Company’s products. The Company believes that the allowances for doubtful accounts related to these countries, if any, are adequate based on its analysis of the specific business circumstances and expectations of collection for each of the underlying accounts in these countries. The Company will continue to monitor these conditions and will attempt to adjust its business processes, as appropriate, to mitigate macroeconomic risks to its business.

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STOCK-BASED COMPENSATION

9 Months Ended

Sep. 30, 2023

Share-Based Payment Arrangement [Abstract]

STOCK-BASED COMPENSATION

STOCK-BASED COMPENSATION

The Company has stockholder-approved equity incentive plans that provide for the granting of restricted stock units (RSUs) and stock options as well as other forms of equity compensation to its employees, officers and non-employee directors. The Company also has an Employee Stock Purchase Plan (ESPP). Compensation expense included in the Company’s Condensed Consolidated Statements of Comprehensive Income for all stock-based compensation arrangements was as follows: 

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Cost of sales	$	4,042	$	4,361	$	13,094	$	13,449
Research and development	14,490		17,055		49,373		47,878
Selling, general and administrative	29,855		33,247		89,777		88,247
Total stock-based compensation expense	$	48,387	$	54,663	$	152,244	$	149,574

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EARNINGS (LOSS) PER COMMON SHARE

9 Months Ended

Sep. 30, 2023

Earnings Per Share [Abstract]

EARNINGS (LOSS) PER COMMON SHARE

EARNINGS (LOSS) PER COMMON SHAREPotentially issuable shares of common stock include shares issuable upon the exercise of outstanding employee stock option awards, common stock issuable under the ESPP, unvested RSUs and contingent issuances of common stock related to the Company’s convertible debt.

The following table sets forth the computation of basic and diluted earnings (loss) per common share (common shares in thousands):

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Numerator:
Net Income (loss), basic	$	40,378	$	(6,652)	$	147,270	$	141,810
Add: Interest expense, net of tax, on the Company's convertible debt	—		—		2,810		2,806
Net Income (loss), diluted	$	40,378	$	(6,652)	$	150,080	$	144,616
Denominator:
Weighted-average common shares outstanding, basic	188,219		185,597		187,617		185,009
Effect of dilutive securities:
Issuances under equity incentive plans	2,954		—		3,455		3,273
Common stock issuable under the Company’s convertible debt(1)	—		—		3,970		3,970
Weighted-average common shares outstanding, diluted	191,173		185,597		195,042		192,252
Earnings (loss) per common share, basic	$	0.21	$	(0.04)	$	0.78	$	0.77
Earnings (loss) per common share, diluted	$	0.21	$	(0.04)	$	0.77	$	0.75

In addition to the equity instruments included in the table above, the table below presents potential shares of common stock that were excluded from the computation of diluted earnings per common share as they were anti-dilutive (in thousands):

	Three Months Ended September 30,		Nine Months Ended September 30,
	2023	2022	2023	2022
Issuances under equity incentive plans	8,547	12,221	8,045	8,948
Common stock issuable under the Company’s convertible debt (1)	8,335	8,335	4,365	4,365
Total number of potentially issuable shares	16,882	20,556	12,410	13,313

(1)    If converted, the Company would issue 4.0 million shares under the 2024 Notes and 4.4 million shares under the 2027 Notes. For additional discussion of our convertible debt, see Note 6 - Debt.

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COMMITMENTS AND CONTINGENCIES

9 Months Ended

Sep. 30, 2023

Commitments and Contingencies Disclosure [Abstract]

COMMITMENTS AND CONTINGENCIES

COMMITMENTS AND CONTINGENCIES

Contingencies

From time to time the Company is involved in legal actions arising in the normal course of its business. The process of resolving matters through litigation or other means is inherently uncertain and it is possible that an unfavorable resolution of these matters could adversely affect the Company, its results of operations, financial condition or cash flows. The Company’s general practice is to expense legal fees as services are rendered in connection with legal matters, and to accrue for liabilities when losses are probable and reasonably estimable based on existing information. The Company accrues for the best estimate of a loss within a range; however, if no estimate in the range is better than any other, then the minimum amount in the range is accrued. Liabilities are evaluated and refined each reporting period as additional information is known. Any receivables for insurance recoveries for these liability claims are recorded as assets when it is probable that a recovery will be realized.

The Company is involved in a purported shareholder class action lawsuit filed against the Company and certain officers and directors alleging violations under Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 as amended (the Exchange Act) for making materially false or misleading statements regarding the clinical trials and Biologics License Application (BLA) for ROCTAVIAN (formerly known as valoctocogene roxaparvovec) by purportedly failing to disclose that differences between the Company’s Phase 1/2 and Phase 3 clinical studies limited the ability of the Phase 1/2 study to support ROCTAVIAN’s durability of effect and, as a result, that it was foreseeable that the FDA would not approve the BLA without additional data. On March 21, 2023, the Court entered an order staying all proceedings and vacating all deadlines because the parties agreed to settle the case through a binding term sheet. The Court preliminarily approved the settlement on June 8, 2023. On October 4, 2023, plaintiff filed a motion for final approval of settlement. A final approval hearing is scheduled for November 8, 2023. The Company maintains directors and officers liability insurance that covers exposure related to this class action lawsuit. As of September 30, 2023, based on these terms the Company has recorded an estimated loss contingency of $39.0 million to Accounts Payable and Accrued Liabilities and the same amount recorded for expected insurance recoveries in Other Current Assets on the Company’s Condensed Consolidated Balance Sheets. As such, there was a net zero impact on the Company’s Condensed Consolidated Statement of Comprehensive Income in the three and nine months ended September 30, 2023.

Contingent Payments

As of September 30, 2023, the Company was subject to contingent payments considered reasonably possible of $788.3 million, of which $591.5 million related to early-stage development programs licensed from two third parties.

Other Commitments

The Company uses experts and laboratories at universities and other institutions to perform certain R&D activities. These amounts are recorded as R&D expense as services are provided. In the normal course of business, the Company enters into various firm purchase commitments primarily to procure active pharmaceutical ingredients, certain inventory-related items and certain third-party R&D services, production services and facility construction services. As of September 30, 2023, such commitments were estimated at $246.0 million, of which $134.4 million is expected to be paid in 2023 as underlying goods and services are received. The Company has also licensed technology from third parties, for which it is required to pay royalties upon future sales, subject to certain annual minimums.

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Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Pay vs Performance Disclosure
Net income	$ 40,378	$ (6,652)	$ 147,270	$ 141,810

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Insider Trading Arrangements

3 Months Ended

9 Months Ended

Sep. 30, 2023

shares

Sep. 30, 2023

shares

Trading Arrangements, by Individual

Material Terms of Trading Arrangement

During the three months ended September 30, 2023, our directors and officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted or terminated the contracts, instructions or written plans for the purchase or sale of BioMarin securities set forth in the table below.

Type of Trading Arrangement
Name	Position	Action	Adoption/Termination Date	Rule 10b5-1*	Non- Rule 10b5-1**	Total Shares of Common Stock to be Sold***	Expiration Date
Jean-Jacques Bienaimé	Chairman and Chief Executive Officer	Adoption	August 7, 2023	X		up to 191,000	June 3, 2024
G. Eric Davis	Executive Vice President, Chief Legal Officer and Secretary	Adoption	September 13, 2023	X		up to 164,429****	August 1, 2024
Henry J. Fuchs, M.D.	President of Worldwide Research & Development	Adoption	August 17, 2023	X		up to 64,659	November 15, 2024
Brian R. Mueller	Executive Vice President and Chief Financial Officer	Adoption	August 23, 2023	X		up to 12,495	July 30, 2024

*    Contract, instruction or written plan intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act.

**     “Non-Rule 10b5-1 trading arrangement” as defined in Item 408(c) of Regulation S-K under the Exchange Act.

***    Represents the maximum number of shares that may be sold pursuant to the 10b5-1 arrangement. The number of shares sold will be dependent on the satisfaction of certain conditions as set forth in the written plan.

**** Includes up to 41,680 shares subject to performance-based restricted stock units previously granted to Mr. Davis that may vest and be released to Mr. Davis on or before August 1, 2024 upon the satisfaction of the applicable vesting conditions. The actual number of shares that will be released to Mr. Davis pursuant to the performance-based restricted stock units and sold under the Rule 10b5-1 trading arrangement will be net of the number of shares withheld to satisfy tax withholding obligations arising from the vesting of such shares and is not yet determinable.

Non-Rule 10b5-1 Arrangement Adopted

false

Rule 10b5-1 Arrangement Terminated

false

Non-Rule 10b5-1 Arrangement Terminated

false

Jean-Jacques Bienaimé [Member]

Trading Arrangements, by Individual

Name

Jean-Jacques Bienaimé

Title

Chairman and Chief Executive Officer

Rule 10b5-1 Arrangement Adopted

true

Adoption Date

August 7, 2023

Arrangement Duration

301 days

Aggregate Available

191,000

191,000

G. Eric Davis [Member]

Trading Arrangements, by Individual

Name

G. Eric Davis

Title

Executive Vice President, Chief Legal Officer and Secretary

Rule 10b5-1 Arrangement Adopted

true

Adoption Date

September 13, 2023

Arrangement Duration

323 days

Aggregate Available

164,429

164,429

Henry J. Fuchs, M.D. [Member]

Trading Arrangements, by Individual

Name

Henry J. Fuchs, M.D.

Title

President of Worldwide Research & Development

Rule 10b5-1 Arrangement Adopted

true

Adoption Date

August 17, 2023

Arrangement Duration

456 days

Aggregate Available

64,659

64,659

Brian R. Mueller [Member]

Trading Arrangements, by Individual

Name

Brian R. Mueller

Title

Executive Vice President and Chief Financial Officer

Rule 10b5-1 Arrangement Adopted

true

Adoption Date

August 23, 2023

Arrangement Duration

342 days

Aggregate Available

12,495

12,495

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BUSINESS OVERVIEW AND SIGNIFICANT ACCOUNTING POLICIES (Policies)

9 Months Ended

Sep. 30, 2023

Accounting Policies [Abstract]

Basis of Presentation

Basis of Presentation

These Condensed Consolidated Financial Statements have been prepared pursuant to U.S. generally accepted accounting principles (U.S. GAAP) and the rules and regulations of the SEC for Quarterly Reports on Form 10-Q and do not include all of the information and note disclosures required by U.S. GAAP for complete financial statements, although management believes that the disclosures herein are adequate to ensure that the information presented is not misleading. The Condensed Consolidated Financial Statements should therefore be read in conjunction with the Consolidated Financial Statements and Notes thereto for the fiscal year ended December 31, 2022 included in the Company’s Annual Report on Form 10-K. The Condensed Consolidated Financial Statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany transactions have been eliminated. The results of operations for the three and nine months ended September 30, 2023 are not necessarily indicative of the results that may be expected for the fiscal year ending December 31, 2023 or any other period.

Use of Estimates

Use of Estimates

U.S. GAAP requires management to make estimates and assumptions that affect amounts reported in the Condensed Consolidated Financial Statements and accompanying disclosures. Although these estimates are based on management’s best knowledge of current events and actions that the Company may undertake in the future, actual results may be different from those estimates. The Condensed Consolidated Financial Statements reflect all adjustments of a normal, recurring nature that are, in the opinion of management, necessary for a fair presentation of results for these interim periods.

Management performed an evaluation of the Company’s activities through the date of filing of this Quarterly Report on Form 10-Q and has concluded that there were no subsequent events or transactions that occurred subsequent to the balance sheet date prior to filing this Quarterly Report on Form 10-Q that would require recognition or disclosure in the Condensed Consolidated Financial Statements.

Recent Accounting Pronouncements

Recent Accounting Pronouncements

There have been no new accounting pronouncements adopted by the Company or new accounting pronouncements issued by the Financial Accounting Standards Board during the nine months ended September 30, 2023, as compared to the recent accounting pronouncements described in Note 1 to the Company’s Consolidated Financial Statements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, that the Company believes are of significance or potential significance to the Company.

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FINANCIAL INSTRUMENTS (Tables)

9 Months Ended

Sep. 30, 2023

Investments, Debt and Equity Securities [Abstract]

Schedule of Cash, Cash Equivalents and Available-for-Sale Securities by Significant Investment Category

The following tables show the Company’s cash, cash equivalents and available-for-sale securities by significant investment category for each period presented:

	September 30, 2023
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash	$	451,294	$	—	$	—	$	451,294	$	451,294	$	—	$	—
Level 2:
Money market instruments	291,425		—		—		291,425		291,425		—		—
Corporate debt securities	563,598		31		(6,135)		557,494		731		217,306		339,457
U.S. government agency securities	281,045		4		(3,023)		278,026		29,981		112,714		135,331
Commercial paper	21,864		—		(4)		21,860		11,983		9,877		—
Asset-backed securities	74,168		26		(446)		73,748		—		534		73,214
Subtotal	1,232,100		61		(9,608)		1,222,553		334,120		340,431		548,002
Total	$	1,683,394	$	61	$	(9,608)	$	1,673,847	$	785,414	$	340,431	$	548,002

	December 31, 2022
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Aggregate Fair Value		Cash and Cash Equivalents		Short-term Marketable Securities (1)		Long-term Marketable Securities (2)
Level 1:
Cash	$	463,248	$	—	$	—	$	463,248	$	463,248	$	—	$	—
Level 2:
Money market instruments	248,933		—		—		248,933		248,933		—		—
Corporate debt securities	504,984		34		(11,541)		493,477		1,881		299,153		192,443
U.S. government agency securities	312,720		45		(3,771)		308,994		—		229,846		79,148
Commercial paper	48,103		11		(22)		48,092		10,469		37,623		—
Asset-backed securities	63,151		69		(592)		62,628		—		384		62,244
Subtotal	1,177,891		159		(15,926)		1,162,124		261,283		567,006		333,835
Total	$	1,641,139	$	159	$	(15,926)	$	1,625,372	$	724,531	$	567,006	$	333,835

(1)    The Company’s short-term marketable securities mature in one year or less.

(2)    The Company’s long-term marketable securities mature between one and five years.

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SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION (Tables)

9 Months Ended

Sep. 30, 2023

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Schedule of Inventory

Inventory consisted of the following:

	September 30, 2023		December 31, 2022
Raw materials	$	142,133	$	131,071
Work-in-process	518,893		410,656
Finished goods	371,133		352,356
Total inventory	$	1,032,159	$	894,083

Schedule of Property, Plant and Equipment, Net

Property, Plant and Equipment, Net consisted of the following:

	September 30, 2023		December 31, 2022
Property, plant and equipment, gross	$	1,918,609	$	1,875,511
Accumulated depreciation	(851,453)		(802,145)
Total property, plant and equipment, net	$	1,067,156	$	1,073,366

Schedule of Intangible Assets

Intangible Assets, Net consisted of the following:

	September 30, 2023		December 31, 2022
Finite-lived intangible assets	$	701,793	$	690,871
Accumulated amortization	(399,317)		(352,302)
Net carrying value	$	302,476	$	338,569

Schedule of Accounts Payable and Accrued Liabilities

Accounts Payable and Accrued Liabilities consisted of the following:

	September 30, 2023		December 31, 2022
Accounts payable and accrued operating expenses	$	301,369	$	231,238
Accrued compensation expense	175,183		207,573
Accrued rebates payable	103,379		72,654
Accrued litigation expense	39,000		—
Accrued royalties payable	12,293		13,306
Accrued income taxes	11,273		16,213
Foreign currency exchange forward contracts	10,584		12,601
Lease liability	9,324		10,375
Other	7,420		8,999
Total accounts payable and accrued liabilities	$	669,825	$	572,959

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FAIR VALUE MEASUREMENTS (Tables)

9 Months Ended

Sep. 30, 2023

Fair Value Disclosures [Abstract]

Schedule of Fair Value of Financial Assets and Liabilities

The following tables present the classification within the fair value hierarchy of financial assets and liabilities not disclosed elsewhere in these Condensed Consolidated Financial Statements that are remeasured on a recurring basis as of September 30, 2023 and December 31, 2022. Other than the Company’s fixed-rate convertible debt disclosed in Note 6 – Debt, there were no financial assets or liabilities that were remeasured using a quoted price in active markets for identical assets (Level 1) as of September 30, 2023 or December 31, 2022.

	Fair Value Measurements as of September 30, 2023
		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Other current assets:
NQDC Plan assets		$	1,836	$	—	$	1,836
Other assets:
NQDC Plan assets		25,541		—		25,541
Restricted investments (1)		2,361		—		2,361
Total other assets		27,902		—		27,902
Total assets		$	29,738	$	—	$	29,738
Liabilities:
Current liabilities:
NQDC Plan liability		$	1,836	$	—	$	1,836
Other long-term liabilities:
NQDC Plan liability		25,541		—		25,541
Total liabilities		$	27,377	$	—	$	27,377

	Fair Value Measurements as of December 31, 2022
	Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Other current assets:
NQDC Plan assets	$	2,654	$	—	$	2,654
Other assets:
NQDC Plan assets	19,867		—		19,867
Restricted investments (1)	2,429		—		2,429
Total other assets	22,296		—		22,296
Total assets	$	24,950	$	—	$	24,950
Liabilities:
Current liabilities:
NQDC Plan liability	$	2,654	$	—	$	2,654
Contingent consideration	—		15,925		15,925
Total current liabilities	2,654		15,925		18,579
Other long-term liabilities:
NQDC Plan liability	19,867		—		19,867
Total liabilities	$	22,521	$	15,925	$	38,446

(1)    The restricted investments as of September 30, 2023 and December 31, 2022 secure the Company's irrevocable standby letters of credit obtained in connection with certain commercial agreements.

Schedule of Liabilities Measured at Fair Value on Recurring Basis Using Level 3 Inputs

Liabilities measured at fair value using Level 3 inputs consisted of contingent consideration. The following table represents a roll-forward of contingent consideration.

Contingent consideration as of December 31, 2022	$	15,925
Milestone payments to Ares Trading S.A. (Merck Serono)	(16,255)
Realized foreign exchange loss on settlement of contingent consideration	330
Contingent consideration as of September 30, 2023	$	—

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DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES (Tables)

9 Months Ended

Sep. 30, 2023

Derivative Instruments and Hedging Activities Disclosure [Abstract]

Schedule of Aggregate Notional Amounts for the Derivatives Outstanding

The following table summarizes the aggregate notional amounts for the Company’s derivatives outstanding as of the periods presented.

Forward Contracts	September 30, 2023		December 31, 2022
Derivatives designated as hedging instruments:
Sell	$	988,286	$	808,635
Purchase	$	180,560	$	177,393
Derivatives not designated as hedging instruments:
Sell	$	301,825	$	218,903
Purchase	$	6,517	$	6,785

Schedule of Fair Value Carrying Amounts of Derivatives

The fair value carrying amounts of the Company’s derivatives, which are classified as Level 2 within the fair value hierarchy, were as follows:

Balance Sheet Location	September 30, 2023		December 31, 2022
Derivatives designated as hedging instruments:
Asset Derivatives
Other current assets	$	21,267	$	19,464
Other assets	7,620		2,059
Subtotal	$	28,887	$	21,523
Liability Derivatives
Accounts payable and accrued liabilities	$	9,918	$	12,130
Other long-term liabilities	3,623		1,074
Subtotal	$	13,541	$	13,204
Derivatives not designated as hedging instruments:
Asset Derivatives
Other current assets	$	5,210	$	1,472
Liability Derivatives
Accounts payable and accrued liabilities	$	666	$	471
Total Derivatives Assets	$	34,097	$	22,995
Total Derivatives Liabilities	$	14,207	$	13,675

Schedule of Impact of Gains and Losses from Derivatives Designated as Hedging Instruments

The following tables summarize the impact of gains and losses from the Company's derivatives on its Condensed Consolidated Statements of Comprehensive Income for the periods presented.

	Three Months Ended September 30,
	2023			2022
Derivatives Designated as Cash Flow Hedging Instruments		Cash Flow Hedging Gains (Losses) Reclassified into Earnings		Cash Flow Hedging Gains (Losses) Reclassified into Earnings
Net product revenues		$	(4,636)	$	15,280
Operating expenses		$	908	$	(3,936)
Derivatives Not Designated as Hedging Instruments		Gains (Losses) Recognized in Earnings		Gains (Losses) Recognized in Earnings
Operating expenses		$	6,559	$	6,572

	Nine Months Ended September 30,
	2023			2022
Derivatives Designated as Cash Flow Hedging Instruments		Cash Flow Hedging Gains (Losses) Reclassified into Earnings		Cash Flow Hedging Gains (Losses) Reclassified into Earnings
Net product revenues		$	(489)	$	26,779
Operating expenses		$	426	$	(7,115)
Derivatives Not Designated as Hedging Instruments		Gains (Losses) Recognized in Earnings		Gains (Losses) Recognized in Earnings
Operating expenses		$	3,165	$	12,812

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DEBT (Tables)

9 Months Ended

Sep. 30, 2023

Debt Disclosure [Abstract]

Schedule of Convertible Debt

The following table summarizes information regarding the Company’s convertible debt:

	September 30, 2023		December 31, 2022
1.25% senior subordinated convertible notes due in May 2027 (the 2027 Notes)	$	600,000	$	600,000
Unamortized discount net of deferred offering costs	(7,414)		(8,941)
2027 Notes, net	592,586		591,059
0.599% senior subordinated convertible notes due in August 2024 (the 2024 Notes)	495,000		495,000
Unamortized discount net of deferred offering costs	(1,602)		(3,040)
2024 Notes, net (1)	493,398		491,960
Total convertible debt, net	$	1,085,984	$	1,083,019
Fair value of fixed-rate convertible debt (2):
2027 Notes	$	595,278	$	647,370
2024 Notes	480,417		526,230
Total fair value of fixed-rate convertible debt	$	1,075,695	$	1,173,600

(1)    As the 2024 Notes mature in August 2024, the outstanding principal of the 2024 Notes is classified as a current liability as of September 30, 2023.

(2)     The fair value of the Company’s fixed-rate convertible debt is based on open-market trades and is classified as Level 1 in the fair value hierarchy. For additional discussion of fair value measurements, see Note 1 – Business Overview and Significant Accounting Policies to the Company’s Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.

Schedule of Interest Expense on Convertible Debt

Interest expense on the Company’s convertible debt consisted of the following: 

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Coupon interest expense	$	2,616	$	2,616	$	7,849	$	7,849
Accretion of discount on convertible notes	841		838		2,519		2,511
Amortization of debt issuance costs	148		148		445		445
Total interest expense on convertible debt	$	3,605	$	3,602	$	10,813	$	10,805

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ACCUMULATED OTHER COMPREHENSIVE INCOME (Tables)

9 Months Ended

Sep. 30, 2023

Equity [Abstract]

Schedule of Changes in Accumulated Balances of AOCI Including Current Period Other Comprehensive Income and Reclassifications Out of AOCI

The following tables summarize changes in the accumulated balances for each component of AOCI, including current-period other comprehensive income and reclassifications out of AOCI, for the periods presented.

	Three Months Ended September 30, 2023
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of June 30, 2023	$	(10,035)	$	(8,582)	$	(18,617)
Other comprehensive income (loss) before      reclassifications	19,892		1,634		21,526
Less: gain (loss) reclassified from AOCI	(3,728)		—		(3,728)
Tax effect	—		(380)		(380)
Net current-period other comprehensive income (loss)	23,620		1,254		24,874
AOCI balance as of September 30, 2023	$	13,585	$	(7,328)	$	6,257

	Three Months Ended September 30, 2022
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of June 30, 2022	$	30,515	$	(12,043)	$	18,472
Other comprehensive income (loss) before      reclassifications	26,698		(4,011)		22,687
Less: gain (loss) reclassified from AOCI	11,344		—		11,344
Tax effect	—		934		934
Net current-period other comprehensive income (loss)	15,354		(3,077)		12,277
AOCI balance as of September 30, 2022	$	45,869	$	(15,120)	$	30,749

	Nine Months Ended September 30, 2023
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of December 31, 2022	$	8,226	$	(12,093)	$	(3,867)
Other comprehensive income (loss) before      reclassifications	5,296		6,220		11,516
Less: gain (loss) reclassified from AOCI	(63)		—		(63)
Tax effect	—		(1,455)		(1,455)
Net current-period other comprehensive income (loss)	5,359		4,765		10,124
AOCI balance as of September 30, 2023	$	13,585	$	(7,328)	$	6,257

	Nine Months Ended September 30, 2022
	Unrealized Gains (Losses) on Cash Flow Hedges		Unrealized Gains (Losses) on Available-for-Sale Debt Securities		Total
AOCI balance as of December 31, 2021	$	15,805	$	(1,373)	$	14,432
Other comprehensive income (loss) before      reclassifications	49,728		(17,905)		31,823
Less: gain (loss) reclassified from AOCI	19,664		—		19,664
Tax effect	—		4,158		4,158
Net current-period other comprehensive income (loss)	30,064		(13,747)		16,317
AOCI balance as of September 30, 2022	$	45,869	$	(15,120)	$	30,749

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REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION (Tables)

9 Months Ended

Sep. 30, 2023

Concentration Risk And Geographic Information [Abstract]

Schedule of Net Product Revenues by Product

The following table disaggregates total Net Product Revenues by product.

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Enzyme product revenues:
VIMIZIM	$	158,869	$	155,470	$	525,453	$	511,735
NAGLAZYME	108,916		99,506		322,040		343,320
PALYNZIQ	78,885		66,206		216,105		182,734
BRINEURA	41,042		37,793		118,244		111,691
ALDURAZYME	13,812		29,046		88,533		90,755
Total enzyme product revenues	401,524		388,021		1,270,375		1,240,235
VOXZOGO	122,973		48,289		324,146		102,321
KUVAN	42,947		57,038		144,047		173,977
ROCTAVIAN	822		—		822		—
Total net product revenues	568,266		493,348		1,739,390		1,516,533
Royalty and other revenues	13,063		11,996		33,629		41,968
Total revenues	$	581,329	$	505,344	$	1,773,019	$	1,558,501

Schedule of Total Net Product Revenues Based on Patient Location

The table below disaggregates total Net Product Revenues by geographic region, which is based on patient location for Company's commercial products sold directly by the Company, except for ALDURAZYME, which is marketed and sold exclusively by Sanofi worldwide.

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
United States	$	194,745	$	176,552	$	553,184	$	497,205
Europe	168,784		162,583		512,910		480,645
Latin America	81,524		60,791		224,716		213,246
Rest of world	109,401		64,376		360,047		234,682
Total net product revenues marketed by the Company	$	554,454	$	464,302	$	1,650,857	$	1,425,778
ALDURAZYME net product revenues marketed by Sanofi	13,812		29,046		88,533		90,755
Total net product revenues	$	568,266	$	493,348	$	1,739,390	$	1,516,533

Schedule of Net Product Revenue Concentrations Attributed to Largest Customers

The following table illustrates the percentage of the Company’s total Net Product Revenues attributed to the Company’s largest customers for the periods presented. 

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Customer A	15	%	15	%	15	%	16	%
Customer B	13	%	14	%	12	%	12	%
Customer C	10	%	10	%	10	%	9	%
Total	38	%	39	%	37	%	37	%

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STOCK-BASED COMPENSATION (Tables)

9 Months Ended

Sep. 30, 2023

Share-Based Payment Arrangement [Abstract]

Schedule of Stock-Based Compensation Expense

Compensation expense included in the Company’s Condensed Consolidated Statements of Comprehensive Income for all stock-based compensation arrangements was as follows: 

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Cost of sales	$	4,042	$	4,361	$	13,094	$	13,449
Research and development	14,490		17,055		49,373		47,878
Selling, general and administrative	29,855		33,247		89,777		88,247
Total stock-based compensation expense	$	48,387	$	54,663	$	152,244	$	149,574

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EARNINGS (LOSS) PER COMMON SHARE (Tables)

9 Months Ended

Sep. 30, 2023

Earnings Per Share [Abstract]

Schedule of Computation of Basic and Diluted Earnings (Loss) Per Common Share

The following table sets forth the computation of basic and diluted earnings (loss) per common share (common shares in thousands):

	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Numerator:
Net Income (loss), basic	$	40,378	$	(6,652)	$	147,270	$	141,810
Add: Interest expense, net of tax, on the Company's convertible debt	—		—		2,810		2,806
Net Income (loss), diluted	$	40,378	$	(6,652)	$	150,080	$	144,616
Denominator:
Weighted-average common shares outstanding, basic	188,219		185,597		187,617		185,009
Effect of dilutive securities:
Issuances under equity incentive plans	2,954		—		3,455		3,273
Common stock issuable under the Company’s convertible debt(1)	—		—		3,970		3,970
Weighted-average common shares outstanding, diluted	191,173		185,597		195,042		192,252
Earnings (loss) per common share, basic	$	0.21	$	(0.04)	$	0.78	$	0.77
Earnings (loss) per common share, diluted	$	0.21	$	(0.04)	$	0.77	$	0.75

Schedule of Anti-Dilutive Common Stock Excluded from Computation of Diluted Earnings Per Share

In addition to the equity instruments included in the table above, the table below presents potential shares of common stock that were excluded from the computation of diluted earnings per common share as they were anti-dilutive (in thousands):

	Three Months Ended September 30,		Nine Months Ended September 30,
	2023	2022	2023	2022
Issuances under equity incentive plans	8,547	12,221	8,045	8,948
Common stock issuable under the Company’s convertible debt (1)	8,335	8,335	4,365	4,365
Total number of potentially issuable shares	16,882	20,556	12,410	13,313

(1)    If converted, the Company would issue 4.0 million shares under the 2024 Notes and 4.4 million shares under the 2027 Notes. For additional discussion of our convertible debt, see Note 6 - Debt.

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FINANCIAL INSTRUMENTS - Schedule of Cash, Cash Equivalents and Available-for-Sale Securities by Significant Investment Category (Details) - USD ($) $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2023	Dec. 31, 2022
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	$ 1,683,394	$ 1,641,139
Gross Unrealized Gains	61	159
Gross Unrealized Losses	(9,608)	(15,926)
Aggregate Fair Value	1,673,847	1,625,372
Cash and Cash Equivalents	785,414	724,531	[1]
Short-term Marketable Securities	340,431	567,006	[1]
Long-term Marketable Securities	$ 548,002	$ 333,835	[1]
Maximum
Schedule of Available-for-sale Securities [Line Items]
Short term marketable securities maturity period	1 year	1 year
Long term marketable securities maturity period	5 years	5 years
Minimum
Schedule of Available-for-sale Securities [Line Items]
Long term marketable securities maturity period	1 year	1 year
Level 1:
Schedule of Available-for-sale Securities [Line Items]
Cash	$ 451,294	$ 463,248
Cash, Aggregate Fair Value	451,294	463,248
Level 2:
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	1,232,100	1,177,891
Gross Unrealized Gains	61	159
Gross Unrealized Losses	(9,608)	(15,926)
Aggregate Fair Value	1,222,553	1,162,124
Cash and Cash Equivalents	334,120	261,283
Short-term Marketable Securities	340,431	567,006
Long-term Marketable Securities	548,002	333,835
Level 2: | Money market instruments
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	291,425	248,933
Gross Unrealized Gains	0	0
Gross Unrealized Losses	0	0
Aggregate Fair Value	291,425	248,933
Cash and Cash Equivalents	291,425	248,933
Short-term Marketable Securities	0	0
Long-term Marketable Securities	0	0
Level 2: | Corporate debt securities
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	563,598	504,984
Gross Unrealized Gains	31	34
Gross Unrealized Losses	(6,135)	(11,541)
Aggregate Fair Value	557,494	493,477
Cash and Cash Equivalents	731	1,881
Short-term Marketable Securities	217,306	299,153
Long-term Marketable Securities	339,457	192,443
Level 2: | U.S. government agency securities
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	281,045	312,720
Gross Unrealized Gains	4	45
Gross Unrealized Losses	(3,023)	(3,771)
Aggregate Fair Value	278,026	308,994
Cash and Cash Equivalents	29,981	0
Short-term Marketable Securities	112,714	229,846
Long-term Marketable Securities	135,331	79,148
Level 2: | Commercial paper
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	21,864	48,103
Gross Unrealized Gains	0	11
Gross Unrealized Losses	(4)	(22)
Aggregate Fair Value	21,860	48,092
Cash and Cash Equivalents	11,983	10,469
Short-term Marketable Securities	9,877	37,623
Long-term Marketable Securities	0	0
Level 2: | Asset-backed securities
Schedule of Available-for-sale Securities [Line Items]
Amortized Cost	74,168	63,151
Gross Unrealized Gains	26	69
Gross Unrealized Losses	(446)	(592)
Aggregate Fair Value	73,748	62,628
Cash and Cash Equivalents	0	0
Short-term Marketable Securities	534	384
Long-term Marketable Securities	$ 73,214	$ 62,244

[1] (1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

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FINANCIAL INSTRUMENTS - Narrative (Details) - USD ($) $ in Thousands	3 Months Ended	9 Months Ended
	Mar. 31, 2023	Sep. 30, 2023	Sep. 30, 2022	Dec. 31, 2022
Schedule of Available-for-sale Securities [Line Items]
Loss on equity investment	$ 12,600	$ 12,650	$ 0
Strategic Investment | Fair Value, Measurements, Recurring
Schedule of Available-for-sale Securities [Line Items]
Strategic investments fair value		$ 11,200		$ 23,900

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SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION - Schedule of Inventory (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Raw materials	$ 142,133	$ 131,071
Work-in-process	518,893	410,656
Finished goods	371,133	352,356
Total inventory	$ 1,032,159	$ 894,083	[1]

[1] (1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

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SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION - Schedule of Property, Plant and Equipment, Net (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Property, plant and equipment, gross	$ 1,918,609	$ 1,875,511
Accumulated depreciation	(851,453)	(802,145)
Property, plant and equipment, net	$ 1,067,156	$ 1,073,366	[1]

[1] (1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

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SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION - Narrative (Details) $ in Millions		3 Months Ended		9 Months Ended		12 Months Ended
	Oct. 06, 2022 employee	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Dec. 31, 2022 USD ($)
Supplemental Balance Sheet Information [Line Items]
Depreciation expense, net of amounts capitalized into inventory		$ 9.9	$ 8.7	$ 29.6	$ 29.9
2022 Restructuring Plan
Supplemental Balance Sheet Information [Line Items]
Reduction in force | employee	120
Percentage of global workforce	4.00%
Restructuring costs						$ 23.0
2022 Restructuring Plan | Accounts payable and accrued liabilities
Supplemental Balance Sheet Information [Line Items]
Restructuring reserve						$ 11.1

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SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION - Schedule of Intangible Assets (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Finite-lived intangible assets	$ 701,793	$ 690,871
Accumulated amortization	(399,317)	(352,302)
Net carrying value	$ 302,476	$ 338,569

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SUPPLEMENTAL FINANCIAL STATEMENTS INFORMATION - Schedule of Accounts Payable and Accrued Liabilities (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Accounts payable and accrued operating expenses	$ 301,369	$ 231,238
Accrued compensation expense	175,183	207,573
Accrued rebates payable	103,379	72,654
Accrued litigation expense	39,000	0
Accrued royalties payable	12,293	13,306
Accrued income taxes	11,273	16,213
Foreign currency exchange forward contracts	10,584	12,601
Lease liability	9,324	10,375
Other	7,420	8,999
Accounts payable and accrued liabilities	$ 669,825	$ 572,959	[1]

[1] (1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

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FAIR VALUE MEASUREMENTS - Schedule of Fair Value of Financial Assets and Liabilities (Details) - USD ($)	Sep. 30, 2023	Dec. 31, 2022
Fair Value, Measurements, Recurring
Assets:
Fair value of other non-current assets	$ 27,902,000	$ 22,296,000
Fair value of financial assets, Total	29,738,000	24,950,000
Liabilities:
Fair value of other current liabilities		18,579,000
Fair value of financial liabilities, Total	27,377,000	38,446,000
Fair Value, Measurements, Recurring | NQDC Plan liability
Liabilities:
Fair value of other current liabilities	1,836,000	2,654,000
Fair value of other non-current liabilities	25,541,000	19,867,000
Fair Value, Measurements, Recurring | Contingent consideration
Liabilities:
Fair value of other current liabilities		15,925,000
Fair Value, Measurements, Recurring | NQDC Plan assets
Assets:
Fair value of other current assets	1,836,000	2,654,000
Fair value of other non-current assets	25,541,000	19,867,000
Fair Value, Measurements, Recurring | Restricted Investments
Assets:
Fair value of other non-current assets	2,361,000	2,429,000
Quoted Price in Active Markets For Identical Assets (Level 1)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Financial assets remeasured	0	0
Liabilities remeasured	0	0
Significant Other Observable Inputs (Level 2) | Fair Value, Measurements, Recurring
Assets:
Fair value of other non-current assets	27,902,000	22,296,000
Fair value of financial assets, Total	29,738,000	24,950,000
Liabilities:
Fair value of other current liabilities		2,654,000
Fair value of financial liabilities, Total	27,377,000	22,521,000
Significant Other Observable Inputs (Level 2) | Fair Value, Measurements, Recurring | NQDC Plan liability
Liabilities:
Fair value of other current liabilities	1,836,000	2,654,000
Fair value of other non-current liabilities	25,541,000	19,867,000
Significant Other Observable Inputs (Level 2) | Fair Value, Measurements, Recurring | Contingent consideration
Liabilities:
Fair value of other current liabilities		0
Significant Other Observable Inputs (Level 2) | Fair Value, Measurements, Recurring | NQDC Plan assets
Assets:
Fair value of other current assets	1,836,000	2,654,000
Fair value of other non-current assets	25,541,000	19,867,000
Significant Other Observable Inputs (Level 2) | Fair Value, Measurements, Recurring | Restricted Investments
Assets:
Fair value of other non-current assets	2,361,000	2,429,000
Significant Unobservable Inputs (Level 3) | Fair Value, Measurements, Recurring
Assets:
Fair value of other non-current assets	0	0
Fair value of financial assets, Total	0	0
Liabilities:
Fair value of other current liabilities		15,925,000
Fair value of financial liabilities, Total	0	15,925,000
Significant Unobservable Inputs (Level 3) | Fair Value, Measurements, Recurring | NQDC Plan liability
Liabilities:
Fair value of other current liabilities	0	0
Fair value of other non-current liabilities	0	0
Significant Unobservable Inputs (Level 3) | Fair Value, Measurements, Recurring | Contingent consideration
Liabilities:
Fair value of other current liabilities		15,925,000
Significant Unobservable Inputs (Level 3) | Fair Value, Measurements, Recurring | NQDC Plan assets
Assets:
Fair value of other current assets	0	0
Fair value of other non-current assets	0	0
Significant Unobservable Inputs (Level 3) | Fair Value, Measurements, Recurring | Restricted Investments
Assets:
Fair value of other non-current assets	$ 0	$ 0

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FAIR VALUE MEASUREMENTS - Schedule of Liabilities Measured at Fair Value on Recurring Basis Using Level 3 Inputs (Details) - Contingent Payment $ in Thousands	9 Months Ended
	Sep. 30, 2023 USD ($)
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward]
Contingent consideration, beginning balance	$ 15,925
Milestone payments to Ares Trading S.A. (Merck Serono)	(16,255)
Realized foreign exchange loss on settlement of contingent consideration	330
Contingent consideration, ending balance	$ 0

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DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES - Narrative (Details) $ in Millions	9 Months Ended
	Sep. 30, 2023 USD ($)
Derivative [Line Items]
Accumulated other comprehensive income (AOCI) to earnings	$ 9.6
Derivatives designated as hedging instruments:
Derivative [Line Items]
Maturity of derivatives	2 years
Derivatives Not Designated as Hedging Instruments
Derivative [Line Items]
Maturity of derivatives	6 months

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DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES - Schedule of Aggregate Notional Amounts for the Derivatives Outstanding (Details) - Forward Foreign Exchange Contracts - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Derivatives designated as hedging instruments: | Sell
Derivative [Line Items]
Notional amount	$ 988,286	$ 808,635
Derivatives designated as hedging instruments: | Purchase
Derivative [Line Items]
Notional amount	180,560	177,393
Derivatives Not Designated as Hedging Instruments | Sell
Derivative [Line Items]
Notional amount	301,825	218,903
Derivatives Not Designated as Hedging Instruments | Purchase
Derivative [Line Items]
Notional amount	$ 6,517	$ 6,785

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DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES - Schedule of Fair Value Carrying Amounts of Derivatives (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Derivative [Line Items]
Derivative asset, fair value	$ 34,097	$ 22,995
Derivative liability, fair value	14,207	13,675
Level 2: | Derivatives designated as hedging instruments:
Derivative [Line Items]
Derivative asset, fair value	28,887	21,523
Derivative liability, fair value	13,541	13,204
Level 2: | Derivatives designated as hedging instruments: | Other current assets
Derivative [Line Items]
Derivative asset, fair value	21,267	19,464
Level 2: | Derivatives designated as hedging instruments: | Other assets
Derivative [Line Items]
Derivative asset, fair value	7,620	2,059
Level 2: | Derivatives designated as hedging instruments: | Accounts payable and accrued liabilities
Derivative [Line Items]
Derivative liability, fair value	9,918	12,130
Level 2: | Derivatives designated as hedging instruments: | Other long-term liabilities
Derivative [Line Items]
Derivative liability, fair value	3,623	1,074
Level 2: | Derivatives Not Designated as Hedging Instruments | Other current assets
Derivative [Line Items]
Derivative asset, fair value	5,210	1,472
Level 2: | Derivatives Not Designated as Hedging Instruments | Accounts payable and accrued liabilities
Derivative [Line Items]
Derivative liability, fair value	$ 666	$ 471

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DERIVATIVE INSTRUMENTS AND HEDGING STRATEGIES - Schedule of Impact of Gains and Losses from Derivatives Designated as Hedging Instruments (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Derivatives designated as hedging instruments: | Accumulated Gain (Loss), Net, Cash Flow Hedge, Parent | Operating expenses
Derivative Instruments, Gain (Loss) [Line Items]
Derivatives Designated as Cash Flow Hedging Instruments	$ 908	$ (3,936)	$ 426	$ (7,115)
Derivatives designated as hedging instruments: | Accumulated Gain (Loss), Net, Cash Flow Hedge, Parent | Net product revenues
Derivative Instruments, Gain (Loss) [Line Items]
Derivatives Designated as Cash Flow Hedging Instruments	(4,636)	15,280	(489)	26,779
Derivatives Not Designated as Hedging Instruments
Derivative Instruments, Gain (Loss) [Line Items]
Derivatives Not Designated as Hedging Instruments	$ 6,559	$ 6,572	$ 3,165	$ 12,812

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DEBT - Narrative (Details) - USD ($)	Sep. 30, 2023	Oct. 31, 2018
Debt Instrument [Line Items]
Carrying value of equity component	$ 1,100,000,000
The 2018 Credit Facility
Debt Instrument [Line Items]
Maximum borrowing capacity		$ 200,000,000
Outstanding amount	$ 0

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DEBT - Schedule of Convertible Debt (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Debt Instrument [Line Items]
Convertible notes	$ 1,100,000
Convertible debt, noncurrent	592,586	$ 1,083,019	[1]
Convertible debt, current	493,398	0	[1]
Total convertible debt, net	1,085,984	1,083,019
Total fair value of fixed-rate convertible debt	$ 1,075,695	$ 1,173,600
1.25% senior subordinated convertible notes due in May 2027 (the 2027 Notes)
Debt Instrument [Line Items]
Convertible notes stated rate	1.25%	1.25%
Convertible notes	$ 600,000	$ 600,000
Unamortized discount net of deferred offering costs	(7,414)	(8,941)
Convertible debt, noncurrent	592,586	591,059
Total fair value of fixed-rate convertible debt	$ 595,278	$ 647,370
0.599% senior subordinated convertible notes due in August 2024 (the 2024 Notes)
Debt Instrument [Line Items]
Convertible notes stated rate	0.599%	0.599%
Convertible notes	$ 495,000	$ 495,000
Unamortized discount net of deferred offering costs	(1,602)	(3,040)
Convertible debt, noncurrent		491,960
Convertible debt, current	493,398
Total fair value of fixed-rate convertible debt	$ 480,417	$ 526,230

[1] (1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

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DEBT - Schedule of Interest Expense on Convertible Debt (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Schedule Of Interest Expenses [Line Items]
Total interest expense on convertible debt	$ 3,779	$ 4,679	$ 11,237	$ 12,344
Convertible Senior Notes
Schedule Of Interest Expenses [Line Items]
Coupon interest expense	2,616	2,616	7,849	7,849
Accretion of discount on convertible notes	841	838	2,519	2,511
Amortization of debt issuance costs	148	148	445	445
Total interest expense on convertible debt	$ 3,605	$ 3,602	$ 10,813	$ 10,805

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ACCUMULATED OTHER COMPREHENSIVE INCOME - Schedule of Changes in Accumulated Balances of AOCI Including Current Period Other Comprehensive Income and Reclassifications Out of AOCI (Details) - USD ($) $ in Thousands		3 Months Ended		9 Months Ended
		Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023		Sep. 30, 2022
AOCI Attributable to Parent, Net of Tax [Roll Forward]
Beginning balances	[1]	$ 4,782,827	$ 4,499,737	$ 4,603,156	[2]	$ 4,265,670
Other comprehensive income (loss) before      reclassifications		21,526	22,687	11,516		31,823
Less: gain (loss) reclassified from AOCI		(3,728)	11,344	(63)		19,664
Tax effect		(380)	934	(1,455)		4,158
Net current-period other comprehensive income (loss)		24,874	12,277	10,124		16,317
Ending balances		4,896,897	4,568,514	4,896,897		4,568,514
AOCI attributable to parent
AOCI Attributable to Parent, Net of Tax [Roll Forward]
Beginning balances	[1]	(18,617)	18,472	(3,867)		14,432
Net current-period other comprehensive income (loss)		24,874	12,277	10,124		16,317
Ending balances		6,257	30,749	6,257		30,749
Unrealized Gains (Losses) on Cash Flow Hedges
AOCI Attributable to Parent, Net of Tax [Roll Forward]
Beginning balances		(10,035)	30,515	8,226		15,805
Other comprehensive income (loss) before      reclassifications		19,892	26,698	5,296		49,728
Less: gain (loss) reclassified from AOCI		(3,728)	11,344	(63)		19,664
Tax effect		0	0	0		0
Net current-period other comprehensive income (loss)		23,620	15,354	5,359		30,064
Ending balances		13,585	45,869	13,585		45,869
Unrealized Gains (Losses) on Available-for-Sale Debt Securities
AOCI Attributable to Parent, Net of Tax [Roll Forward]
Beginning balances		(8,582)	(12,043)	(12,093)		(1,373)
Other comprehensive income (loss) before      reclassifications		1,634	(4,011)	6,220		(17,905)
Less: gain (loss) reclassified from AOCI		0	0	0		0
Tax effect		(380)	934	(1,455)		4,158
Net current-period other comprehensive income (loss)		1,254	(3,077)	4,765		(13,747)
Ending balances		$ (7,328)	$ (15,120)	$ (7,328)		$ (15,120)

[1] The beginning balances for the nine-month periods were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 filed with the SEC on February 27, 2023.

[2] (1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

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REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION - Narrative (Details) $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2023 USD ($) segment	Dec. 31, 2022 USD ($)
Concentration Risk And Geographic Information [Line Items]
Number of operating business segment | segment	1
Accounts receivable, net	$ 572,498	$ 461,316	[1]
Sanofi
Concentration Risk And Geographic Information [Line Items]
Accounts receivable, net	$ 54,800	$ 68,800
Credit Concentration Risk | Accounts Receivable | Customer One
Concentration Risk And Geographic Information [Line Items]
Concentration risk, percentage	16.00%	22.00%
Credit Concentration Risk | Accounts Receivable | Customer Two
Concentration Risk And Geographic Information [Line Items]
Concentration risk, percentage	11.00%	15.00%

[1] (1)December 31, 2022 balances were derived from the audited Consolidated Financial Statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

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REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION - Schedule of Net Product Revenues by Product (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Revenue from External Customer [Line Items]
Revenue	$ 581,329	$ 505,344	$ 1,773,019	$ 1,558,501
Product
Revenue from External Customer [Line Items]
Revenue	568,266	493,348	1,739,390	1,516,533
Total enzyme product revenues
Revenue from External Customer [Line Items]
Revenue	401,524	388,021	1,270,375	1,240,235
VIMIZIM
Revenue from External Customer [Line Items]
Revenue	158,869	155,470	525,453	511,735
NAGLAZYME
Revenue from External Customer [Line Items]
Revenue	108,916	99,506	322,040	343,320
PALYNZIQ
Revenue from External Customer [Line Items]
Revenue	78,885	66,206	216,105	182,734
BRINEURA
Revenue from External Customer [Line Items]
Revenue	41,042	37,793	118,244	111,691
ALDURAZYME
Revenue from External Customer [Line Items]
Revenue	13,812	29,046	88,533	90,755
VOXZOGO
Revenue from External Customer [Line Items]
Revenue	122,973	48,289	324,146	102,321
KUVAN
Revenue from External Customer [Line Items]
Revenue	42,947	57,038	144,047	173,977
ROCTAVIAN
Revenue from External Customer [Line Items]
Revenue	822	0	822	0
Royalty and other revenues
Revenue from External Customer [Line Items]
Revenue	$ 13,063	$ 11,996	$ 33,629	$ 41,968

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REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION - Schedule of Total Net Product Revenues Based on Patient Location (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Disaggregation of Revenue [Line Items]
Revenue	$ 581,329	$ 505,344	$ 1,773,019	$ 1,558,501
ALDURAZYME
Disaggregation of Revenue [Line Items]
Revenue	13,812	29,046	88,533	90,755
Product
Disaggregation of Revenue [Line Items]
Revenue	568,266	493,348	1,739,390	1,516,533
Marketed by Company | Products excluding ALDURAZYME
Disaggregation of Revenue [Line Items]
Revenue	554,454	464,302	1,650,857	1,425,778
Marketed by Company | Products excluding ALDURAZYME | United States
Disaggregation of Revenue [Line Items]
Revenue	194,745	176,552	553,184	497,205
Marketed by Company | Products excluding ALDURAZYME | Europe
Disaggregation of Revenue [Line Items]
Revenue	168,784	162,583	512,910	480,645
Marketed by Company | Products excluding ALDURAZYME | Latin America
Disaggregation of Revenue [Line Items]
Revenue	81,524	60,791	224,716	213,246
Marketed by Company | Products excluding ALDURAZYME | Rest of world
Disaggregation of Revenue [Line Items]
Revenue	109,401	64,376	360,047	234,682
Marketed by Sanofi | ALDURAZYME
Disaggregation of Revenue [Line Items]
Revenue	$ 13,812	$ 29,046	$ 88,533	$ 90,755

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REVENUE, CREDIT CONCENTRATIONS AND GEOGRAPHIC INFORMATION - Schedule of Net Product Revenue Concentrations Attributed to Largest Customers (Details) - Customer Concentration Risk - Net Product Revenue	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Customer A, B & C
Concentration Risk [Line Items]
Concentration risk, percentage	38.00%	39.00%	37.00%	37.00%
Customer A
Concentration Risk [Line Items]
Concentration risk, percentage	15.00%	15.00%	15.00%	16.00%
Customer B
Concentration Risk [Line Items]
Concentration risk, percentage	13.00%	14.00%	12.00%	12.00%
Customer C
Concentration Risk [Line Items]
Concentration risk, percentage	10.00%	10.00%	10.00%	9.00%

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STOCK-BASED COMPENSATION (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 48,387	$ 54,663	$ 152,244	$ 149,574
Cost of sales
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	4,042	4,361	13,094	13,449
Research and development
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	14,490	17,055	49,373	47,878
Selling, general and administrative
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total stock-based compensation expense	$ 29,855	$ 33,247	$ 89,777	$ 88,247

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EARNINGS (LOSS) PER COMMON SHARE - Schedule of Computation of Basic and Diluted Earnings (Loss) Per Common Share (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Numerator:
Net Income (loss), basic	$ 40,378	$ (6,652)	$ 147,270	$ 141,810
Add: Interest expense, net of tax, on the Company's convertible debt	0	0	2,810	2,806
Net Income (loss), diluted	$ 40,378	$ (6,652)	$ 150,080	$ 144,616
Denominator:
Weighted-average common shares outstanding, basic (in shares)	188,219	185,597	187,617	185,009
Effect of dilutive securities:
Weighted-average common shares outstanding, diluted (in shares)	191,173	185,597	195,042	192,252
Earnings (loss) per common share, basic (in dollars per share)	$ 0.21	$ (0.04)	$ 0.78	$ 0.77
Earnings (loss) per common share, diluted (in dollars per share)	$ 0.21	$ (0.04)	$ 0.77	$ 0.75
Issuances under equity incentive plans
Effect of dilutive securities:
Effect of dilutive securities (in shares)	2,954	0	3,455	3,273
Common stock issuable under the Company’s convertible debt
Effect of dilutive securities:
Effect of dilutive securities (in shares)	0	0	3,970	3,970

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EARNINGS (LOSS) PER COMMON SHARE - Schedule of Anti-Dilutive Common Stock Excluded from Computation of Diluted Net Loss Per Share (Details) - shares shares in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total number of potentially issuable shares (in shares)	16,882	20,556	12,410	13,313
0.599% senior subordinated convertible notes due in August 2024 (the 2024 Notes) | Convertible Senior Notes
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential shares issued if converted (in shares)			4,000
1.25% senior subordinated convertible notes due in May 2027 (the 2027 Notes) | Convertible Senior Notes
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential shares issued if converted (in shares)			4,400
Issuances under equity incentive plans
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total number of potentially issuable shares (in shares)	8,547	12,221	8,045	8,948
Common stock issuable under the Company’s convertible debt
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Total number of potentially issuable shares (in shares)	8,335	8,335	4,365	4,365

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COMMITMENTS AND CONTINGENCIES (Details) $ in Millions	9 Months Ended
	Sep. 30, 2023 USD ($) third_party
Commitments And Contingencies [Line Items]
Contingent payments upon achievement of certain development and regulatory activities and commercial sales and licensing milestones	$ 788.3
Purchase commitment	246.0
Purchase commitments expected to be paid in 2023	134.4
Early Stage Development Program | Third Party
Commitments And Contingencies [Line Items]
Contingent payments upon achievement of certain development and regulatory activities and commercial sales and licensing milestones	$ 591.5
Number of third parties | third_party	2
Shareholder vs BioMarin Pharmaceutical Inc
Commitments And Contingencies [Line Items]
Loss contingency	$ 39.0