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 March 31, 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 001-33497

Amicus Therapeutics, Inc.

(Exact Name of Registrant as Specified in Its Charter)

Delaware				71-0869350
(State or Other Jurisdiction of				(I.R.S. Employer
Incorporation or Organization)				Identification Number)
3675 Market Street,	Philadelphia,	PA		19104
(Address of Principal Executive Offices)				(Zip Code)
(215)			921-7600
(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 $0.01 per share	FOLD	NASDAQ Global 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 ☒

The number of shares outstanding of the registrant's common stock, $0.01 par value per share, as of April 27, 2023 was 284,575,686 shares.

AMICUS THERAPEUTICS, INC.

Form 10-Q for the Quarterly Period Ended March 31, 2023

 

			Page
PART I.     FINANCIAL INFORMATION			3
	Item 1.	Consolidated Financial Statements and Notes (unaudited)	3
		Consolidated Balance Sheets as of March 31, 2023 and December 31, 2022	3
		Consolidated Statements of Operations for the Three Months Ended March 31, 2023 and 2022	4
		Consolidated Statements of Comprehensive Loss for the Three Months Ended March 31, 2023 and 2022	5
		Consolidated Statements of Changes in Stockholders' Equity for the Three Months Ended March 31, 2023 and 2022	6
		Consolidated Statements of Cash Flows for the Three Months Ended March 31, 2023 and 2022	7
		Notes to Consolidated Financial Statements	8
	Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	19
	Item 3.	Quantitative and Qualitative Disclosures about Market Risk	27
	Item 4.	Controls and Procedures	27
PART II.     OTHER INFORMATION			27
	Item 1.	Legal Proceedings	27
	Item 1A.	Risk Factors	27
	Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds	27
	Item 3.	Defaults Upon Senior Securities	28
	Item 4.	Mine Safety Disclosures	28
	Item 5.	Other Information	28
	Item 6.	Exhibits	29
SIGNATURES			30

i

SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q contains forward-looking statements that involve risks, uncertainties, and assumptions. Forward-looking statements are all statements, other than statements of historical facts, that discuss our current expectation and projections relating to our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans, and objectives of management. These statements may be preceded by, followed by or include the words "aim," "anticipate," "believe," "can," "could," "estimate," "expect," "forecast," "intend," "likely," "may," "might," "outlook," "plan," "potential," "predict," "project," "seek," "should," "will," "would," the negatives or plurals thereof, and other words and terms of similar meaning, although not all forward-looking statements contain these identifying words.

We have based these forward-looking statements on our current expectations and projections about future events. Although we believe that our assumptions made in connection with the forward-looking statements are reasonable, we cannot assure you that the assumptions and expectations will prove to be correct. You should understand that the following important factors could affect our future results and could cause those results or other outcomes to differ materially from those expressed or implied in our forward-looking statements:

•the scope, progress, results and costs of clinical trials for our drug candidates;

•the cost of manufacturing drug supply for our commercial, clinical and preclinical studies, including the cost of manufacturing Pompe Enzyme Replacement Therapy ("ERT" or "ATB200" or "cipaglucosidase alfa");

•the future results of preclinical research and subsequent clinical trials for pipeline candidates we may identify from time to time, including our ability to obtain regulatory approvals and commercialize these therapies and obtain market acceptance for such therapies;

•the costs, timing, and outcome of regulatory review of our product candidates, including AT-GAA;

•any changes in regulatory standards relating to the review of our product candidates, including AT-GAA;

•the number and development requirements of other product candidates that we pursue;

•the costs of commercialization activities, including product marketing, sales, and distribution;

•the emergence of competing technologies and other adverse market developments;

•the estimates regarding the potential market opportunity for our product and product candidates, including AT-GAA;

•our ability to successfully commercialize Galafold® (also referred to as "migalastat HCl") and, if our regulatory applications are approved, AT-GAA;

•our ability to manufacture or supply sufficient clinical or commercial products, including Galafold® and AT-GAA;

•our ability to obtain reimbursement for Galafold® and, if our regulatory applications are approved, AT-GAA;

•our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold®, and, if approved and applicable, AT-GAA;

•our ability to obtain market acceptance of Galafold® and, if our regulatory applications are approved, AT-GAA;

•the costs of preparing, filing, and prosecuting patent applications and maintaining, enforcing, and defending intellectual property-related claims, including Hatch-Waxman litigation;

•the impact of litigation that has been or may be brought against us or of litigation that we are pursuing or may pursue against others, including Hatch-Waxman litigation;

•the extent to which we acquire or invest in businesses, products, and technologies;

•our ability to successfully integrate our acquired products and technologies into our business, or successfully divest or license existing products and technologies from our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;

•our ability to establish licensing agreements, collaborations, partnerships or other similar arrangements and to obtain milestone, royalty, or other payments from any such collaborators;

•the extent to which our business could be adversely impacted by the effects of the novel coronavirus ("COVID-19") outbreak, including actions by us, governments, our customers, our suppliers, or other third parties to control the spread of COVID-19, or by other health epidemics or pandemics;

•the costs associated with, and our ability to comply with, emerging environmental, social and governance standards;

1

•our ability to accurately forecast revenue, operating expenditures, or other metrics impacting profitability;

•fluctuations in foreign currency exchange rates; and

•changes in accounting standards.

In light of these risks and uncertainties, we may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions, and expectations disclosed in the forward-looking statements we make. We have included important factors in the cautionary statements included in Part I Item 1A — Risk Factors of the Annual Report on Form 10-K for the fiscal year ended December 31, 2022, that we believe could cause actual results or events to differ materially from the forward-looking statements that we make. Those factors and the other risk factors described herein are not necessarily all of the important factors that could cause actual results or developments to differ materially from those expressed in any of our forward-looking statements. Other unknown or unpredictable factors also could harm our results. Our forward-looking statements do not reflect the potential impact of any future collaborations, alliances, business combinations, partnerships, strategic out-licensing of certain assets, the acquisition of preclinical-stage, clinical-stage, marketed products or platform technologies or other investments we may make. Consequently, there can be no assurance that actual results or developments anticipated by us will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, us. Given these uncertainties, investors are cautioned not to place undue reliance on such forward-looking statements.

You should read this Quarterly Report on Form 10-Q in conjunction with our Annual Report on Form 10-K for the fiscal year ended December 31, 2022 (including the documents incorporated by reference therein) completely and with the understanding that our actual future results may be materially different from what we expect. These forward-looking statements speak only as of the date of this report. We undertake no obligation, and specifically decline any obligation, to publicly update or revise any forward-looking statements, even if experience or future developments make it clear that projected results expressed or implied in such statements will not be realized, except as may be required by law. 

2

PART I.    FINANCIAL INFORMATION

ITEM 1.    CONSOLIDATED FINANCIAL STATEMENTS AND NOTES (UNAUDITED)

Amicus Therapeutics, Inc.

Consolidated Balance Sheets

(Unaudited)

(in thousands, except share and per share amounts)

	March 31, 2023		December 31, 2022
Assets
Current assets:
Cash and cash equivalents	$	160,602	$	148,813
Investments in marketable securities	106,507		144,782
Accounts receivable	68,178		66,196
Inventories	27,004		23,816
Prepaid expenses and other current assets	37,406		40,209
Total current assets	399,697		423,816
Operating lease right-of-use assets, net	28,483		29,534
Property and equipment, less accumulated depreciation of $22,901 and $22,281 at March 31, 2023 and December 31, 2022, respectively	31,406		30,778
Intangible assets, less accumulated amortization of $36 and $0 at March 31, 2023 and December 31, 2022, respectively	22,964		23,000
Goodwill	197,797		197,797
Other non-current assets	20,172		19,242
Total Assets	$	700,519	$	724,167
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable	$	24,965	$	15,413
Accrued expenses and other current liabilities	92,747		93,636
Contingent consideration payable	12,668		21,417
Operating lease liabilities	8,005		8,552
Total current liabilities	138,385		139,018
Long-term debt	392,658		391,990
Operating lease liabilities	51,349		51,578
Deferred reimbursements	5,906		4,656
Deferred income taxes	—		4,939
Other non-current liabilities	9,648		8,939
Total liabilities	597,946		601,120
Commitments and contingencies
Stockholders’ equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 283,300,585 and 281,108,273 shares issued and outstanding at March 31, 2023 and December 31, 2022, respectively	2,820		2,815
Additional paid-in capital	2,691,836		2,664,744
Accumulated other comprehensive loss:
Foreign currency translation adjustment	(6,543)		(11,989)
Unrealized loss on available-for-sale securities	(201)		(116)
Warrants	83		83
Accumulated deficit	(2,585,422)		(2,532,490)
Total stockholders’ equity	102,573		123,047
Total Liabilities and Stockholders’ Equity	$	700,519	$	724,167

See accompanying Notes to Consolidated Financial Statements

3

Amicus Therapeutics, Inc.

Consolidated Statements of Operations

(Unaudited)

(in thousands, except share and per share amounts)

	Three Months Ended March 31,
	2023		2022
Net product sales	$	86,270	$	78,715
Cost of goods sold	6,942		7,582
Gross profit	79,328		71,133
Operating expenses:
Research and development	41,499		81,517
Selling, general, and administrative	73,957		58,116
Changes in fair value of contingent consideration payable	251		(1,188)
Loss on impairment of assets	—		6,616
Depreciation and amortization	1,257		1,411
Total operating expenses	116,964		146,472
Loss from operations	(37,636)		(75,339)
Other (expense) income:
Interest income	2,199		133
Interest expense	(11,844)		(8,147)
Other (expense) income	(5,938)		1,902
Loss before income tax	(53,219)		(81,451)
Income tax benefit (expense)	287		(3,809)
Net loss attributable to common stockholders	$	(52,932)	$	(85,260)
Net loss attributable to common stockholders per common share — basic and diluted	$	(0.18)	$	(0.30)
Weighted-average common shares outstanding — basic and diluted	291,336,750		288,481,741

                                                                                    

See accompanying Notes to Consolidated Financial Statements

4

Amicus Therapeutics, Inc.

Consolidated Statements of Comprehensive Loss

(Unaudited)

(in thousands)

	Three Months Ended March 31,
	2023		2022
Net loss	$	(52,932)	$	(85,260)
Other comprehensive gain (loss):
Foreign currency translation adjustment gain (loss)	5,446		(5,671)
Unrealized loss on available-for-sale securities	(85)		(338)
Other comprehensive gain (loss)	5,361		(6,009)
Comprehensive loss	$	(47,571)	$	(91,269)

See accompanying Notes to Consolidated Financial Statements

5

Amicus Therapeutics, Inc.

Consolidated Statements of Changes in Stockholders' Equity

(Unaudited)

(in thousands, except share amounts)

Three Months Ended March 31, 2023
	Common Stock			Additional Paid-In Capital		Warrants		Other Comprehensive Gain (Loss)		Accumulated Deficit		Total Stockholders' Equity
	Shares	Amount
Balance at December 31, 2022	281,108,273	$	2,815	$	2,664,744	$	83	$	(12,105)	$	(2,532,490)	$	123,047
Stock options exercised, net	384,108	3		2,652		—		—		—		2,655
Vesting of restricted stock units, net of taxes	1,612,975	—		(12,806)		—		—		—		(12,806)
Stock-based compensation	—	—		34,894		—		—		—		34,894
Issuance of shares in connection with at-the-market offering, net of issuance costs	195,229	2		2,352		—		—		—		2,354
Unrealized loss on available-for-sale securities	—	—		—		—		(85)		—		(85)
Foreign currency translation adjustment	—	—		—		—		5,446		—		5,446
Net loss	—	—		—		—		—		(52,932)		(52,932)
Balance at March 31, 2023	283,300,585	$	2,820	$	2,691,836	$	83	$	(6,744)	$	(2,585,422)	$	102,573

Three Months Ended March 31, 2022
	Common Stock			Additional Paid-In Capital		Warrants		Other Comprehensive Gain (Loss)		Accumulated Deficit		Total Stockholders' Equity
	Shares	Amount
Balance at December 31, 2021	278,912,800	$	2,808	$	2,595,419	$	83	$	4,981	$	(2,295,922)	$	307,369
Stock options exercised, net	145,449	1		858		—		—		—		859
Vesting of restricted stock units, net of taxes	1,075,607	—		(8,993)		—		—		—		(8,993)
Stock-based compensation	—	—		30,651		—		—		—		30,651
Unrealized loss on available-for-sale securities	—	—		—		—		(338)		—		(338)
Foreign currency translation adjustment	—	—		—		—		(5,671)		—		(5,671)
Net loss	—	—		—		—		—		(85,260)		(85,260)
Balance at March 31, 2022	280,133,856	$	2,809	$	2,617,935	$	83	$	(1,028)	$	(2,381,182)	$	238,617

See accompanying Notes to Consolidated Financial Statements

6

Amicus Therapeutics, Inc.

Consolidated Statements of Cash Flows

(Unaudited)

(in thousands)

	Three Months Ended March 31,
	2023		2022
Operating activities
Net loss	$	(52,932)	$	(85,260)
Adjustments to reconcile net loss to net cash used in operating activities:
Amortization of debt discount and deferred financing	667		637
Depreciation and amortization	1,257		1,411
Stock-based compensation	34,894		30,651
Non-cash changes in the fair value of contingent consideration payable	251		(1,188)
Foreign currency remeasurement loss	5,885		673
Deferred taxes	(4,939)		—
Asset impairment charges and other asset write-offs	—		6,616
Changes in operating assets and liabilities:
Accounts receivable	(1,367)		(1,000)
Inventories	(3,158)		1,812
Prepaid expenses and other current assets	1,839		3,332
Accounts payable, accrued expenses, and other current liabilities	(72)		(14,563)
Other non-current assets and liabilities	(394)		(1,436)
Net cash used in operating activities	$	(18,069)	$	(58,315)
Investing activities
Sale and redemption of marketable securities	54,944		108,328
Purchases of marketable securities	(16,747)		(49,244)
Capital expenditures	(1,942)		(871)
Net cash provided by investing activities	$	36,255	$	58,213
Financing activities
Payment of finance leases	(28)		(20)
Purchase of vested restricted stock units, net of taxes	(12,806)		(8,993)
Proceeds from stock options exercised, net	2,655		859
Proceeds from the issuance of shares in connection with at-the-market offering, net of issuance costs	2,354		—
Net cash used in financing activities	$	(7,825)	$	(8,154)
Effect of exchange rate changes on cash, cash equivalents, and restricted cash	$	1,507	$	(3,377)
Net increase (decrease) in cash, cash equivalents, and restricted cash at the end of the period	11,868		(11,633)
Cash, cash equivalents, and restricted cash at the beginning of period	153,115		249,456
Cash, cash equivalents, and restricted cash at the end of period	$	164,983	$	237,823
Supplemental disclosures of cash flow information
Cash paid during the period for interest	$	11,361	$	7,509
Cash paid for taxes	$	178	$	456
Capital expenditures unpaid at the end of period	$	1,260	$	72

See accompanying Notes to Consolidated Financial Statements

7

Amicus Therapeutics, Inc.

Notes to the Consolidated Financial Statements

(Unaudited)

1. Description of Business

Amicus Therapeutics, Inc. (the "Company") is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. The Company has a portfolio including the first, oral monotherapy for Fabry disease that has achieved widespread global approval and a differentiated biologic for Pompe disease, that is under regulatory review. The Company is committed to discovering and developing next generation therapies in Fabry and Pompe diseases.

The cornerstone of the Company's portfolio is Galafold® (also referred to as "migalastat"), the first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants. Migalastat is currently approved under the trade name Galafold® in the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan, with multiple additional approvals granted and applications pending in several geographies around the world.

The lead biologics program of the Company's pipeline is Amicus Therapeutics GAA ("AT-GAA", also known as ATB200/AT2221, or cipaglucosidase alfa/miglustat), a novel, two-component, potential best-in-class treatment for Pompe disease. AT-GAA is currently under review with regulatory authorities. In October 2022, the U.S. Food and Drug Administration ("FDA") deferred action on the BLA for cipaglucosidase alfa, citing the inability to complete the manufacturing site inspection prior to the PDUFA action date. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site. The Company believes the comments and observations received at the close of the FDA inspection are all addressable. In March 2023, the European Commission ("EC") granted approval for Pombiliti™ (cipaglucosidase alfa) to be used in combination with miglustat for adults with late-onset Pompe disease ("LOPD"). In April 2023, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion on Opfolda™ (miglustat), the enzyme stabilizer component of AT-GAA. The regulatory submission process for AT-GAA in the U.K. was initiated in December 2022.

The Company continues to monitor the novel coronavirus (“COVID-19”) pandemic. The Company's commercial operations have not been significantly impacted by the COVID-19 pandemic and the Company gradually continues to see an improvement in patient identification and Galafold® initiation. The Company has maintained operations in all geographies, secured its global supply chain for its commercial and clinical products, as well as maintained the operational integrity of its clinical trials, with minimum disruptions. The Company has been able to continue to meet required commercial demand for Galafold® as well as supply its ongoing Pompe disease clinical studies and access programs including the Early Access to Medicines Scheme ("EAMS") without interruption. In regard to the Company's regulatory operations, the FDA deferred action on the pending BLA for cipaglucosidase alfa, as a site inspection was necessary, however, could not be completed by the PDUFA action date due to COVID-19 pandemic related travel restrictions. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site. Per FDA guidance related to pre-approval inspections during the COVID-19 pandemic, receipt of a deferral action indicates no deficiencies have been identified and the application otherwise satisfies the requirements for approval.

The Company had an accumulated deficit of $2.6 billion as of March 31, 2023 and anticipates incurring losses through the fiscal year ending December 31, 2023. The Company has historically funded its operations through stock offerings, Galafold® revenues, debt issuances, collaborations, and other financing arrangements.

Based on its current operating model, the Company believes that the current cash position, which includes expected revenues, is sufficient to fund the Company's operations and ongoing research programs for at least the next 12 months. Potential impacts of the COVID-19 pandemic, business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact the Company's future capital requirements.

8

2. Summary of Significant Accounting Policies

Basis of Presentation

The Company has prepared the accompanying unaudited Consolidated Financial Statements in accordance with the U.S. generally accepted accounting principles ("U.S. GAAP") for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. In the opinion of management, the accompanying unaudited Consolidated Financial Statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company's interim financial information.

The accompanying unaudited Consolidated Financial Statements and related notes should be read in conjunction with the Company's financial statements and related notes as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2022. For a complete description of the Company's accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2022.

Consolidation

The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.

Foreign Currency Transactions

The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity.

Use of Estimates

The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.

Additionally, the Company assessed the impact the COVID-19 pandemic had on its operations and financial results as of March 31, 2023 and through the issuance of these financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and revenue and expenses.

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive loss in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs.

Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included in other current assets and other non-current assets on the Company's Consolidated Balance Sheets.

9

Concentration of Credit Risk

The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities.

The Company is subject to credit risk from its accounts receivable related to its product sales of Galafold®. The Company's accounts receivable at March 31, 2023 have arisen from product sales primarily in Europe, the U.S., and Japan. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis. As of March 31, 2023, the Company's allowance for doubtful accounts was $0.1 million.

Revenue Recognition

The Company's net product sales consist primarily of sales of Galafold® for the treatment of Fabry disease. Galafold® sales for the three months ended March 31, 2023 and 2022 were $86.1 million and $78.7 million, respectively. The Company has recorded revenue on sales where Galafold® is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold® are generally received from distributors and pharmacies, with the ultimate payor often a government authority.

The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of Galafold®. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale of Galafold® is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.

The following table summarizes the Company's net product sales disaggregated by geographic area:

	Three Months Ended March 31,
(in thousands)	2023		2022
U.S.	$	28,831	$	24,178
Ex-U.S.	57,439		54,537
Total net product sales	$	86,270	$	78,715

Inventories and Cost of Goods Sold

Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations.

Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable.

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Intangible Assets and Goodwill

The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Goodwill is assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill impairment test is conducted. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference.

Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and, if applicable, impairment charges. Amortization of finite-lived intangible assets is recorded over the assets’ estimated useful lives on a straight-line basis or based on the pattern in which economic benefits are consumed, if reliably determinable. The Company reviews finite-lived intangible assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the asset over its estimated fair value in the period at which such a determination is made.

No indicators of impairment were noted during the three months ended March 31, 2023.

Recent Accounting Developments

The Company has evaluated recent accounting pronouncements and believes that none of them will have a material effect on the Company's Consolidated Financial Statements or related disclosures.

3. Intangible Assets

As of March 31, 2023, the Company's intangible assets consisted of lead enzyme replacement therapy assets acquired with the Callidus Biopharma, Inc. acquisition in 2013, previously accounted for as in-process research and development. In March 2023, as a result of the EC's approval of Pombiliti™, the Company began amortizing the assets over the initial regulatory exclusivity period of 7 years. The Company completed an impairment assessment before changing the classification to definite-lived intangible asset noting no impairment. Amortization expense for the three months ended March 31, 2023 was nominal. Total estimated amortization for the finite-lived intangible assets is estimated to be $2.5 million for the year ended December 31, 2023 and $3.3 million for the next four years thereafter.

4. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

As of March 31, 2023, the Company held $160.6 million in cash and cash equivalents and $106.5 million of marketable securities which are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are generally reported within other comprehensive gain (loss) in the Company's Consolidated Statements of Comprehensive Loss. If a decline in the fair value of a marketable security below the Company's cost basis is determined to be other-than-temporary or if an available-for-sale debt security’s fair value is determined to be less than the amortized cost and the Company intends or is more than likely to sell the security before recovery and it is not considered a credit loss such security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in earnings as an impairment charge. If the unrealized loss of an available-for-sale debt security is determined to be a result of credit loss, the Company would recognize an allowance and the corresponding credit loss would be included in earnings.

The Company regularly invests excess operating cash in deposits with major financial institutions and money market funds, as well as fixed income investments which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as, in accordance with Company policy, securities are of high credit rating. Investments that have original maturities greater than three months but less than one year are classified as current.

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Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:

	As of March 31, 2023
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	160,602	$	—	$	—	$	160,602
Commercial paper	106,109		14		(17)		106,106
Money market	350		—		—		350
Certificates of deposit	51		—		—		51
	$	267,112	$	14	$	(17)	$	267,109
Included in cash and cash equivalents	$	160,602	$	—	$	—	$	160,602
Included in marketable securities	106,510		14		(17)		106,507
Total cash, cash equivalents, and marketable securities	$	267,112	$	14	$	(17)	$	267,109

	As of December 31, 2022
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Commercial paper	144,299		82		—		144,381
Money market	350		—		—		350
Certificate of deposit	51		—		—		51
	$	293,513	$	82	$	—	$	293,595
Included in cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Included in marketable securities	144,700		82		—		144,782
Total cash, cash equivalents, and marketable securities	$	293,513	$	82	$	—	$	293,595

For both the three months ended March 31, 2023 and the fiscal year ended December 31, 2022, there were no realized gains or losses. The cost of securities sold is based on the specific identification method.

Unrealized loss positions in the marketable securities as of March 31, 2023 reflect temporary impairments and are not a result of credit loss. Additionally, as these positions have been in a loss position for less than twelve months and the Company does not intend to sell these securities before recovery, the losses are recognized in other comprehensive gain (loss). The fair value of these marketable securities in unrealized loss positions was $37.6 million as of March 31, 2023. The Company had no securities in an unrealized loss position as of December 31, 2022.

The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Company's Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Company's Consolidated Statements of Cash Flows.

	As of March 31,
(in thousands)	2023		2022
Cash and cash equivalents	$	160,602	$	233,317
Restricted cash	4,381		4,506
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$	164,983	$	237,823

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5. Inventories

Inventories as of March 31, 2023 and December 31, 2022 consisted of the following:

(in thousands)	March 31, 2023		December 31, 2022
Raw materials	$	13,597	$	10,054
Work-in-process	5,823		9,615
Finished goods	7,584		4,147
Total inventories	$	27,004	$	23,816

The Company's reserve for inventory was $0.4 million as of both March 31, 2023 and December 31, 2022.

6. Debt

The Company's debt consists of the following:

(in thousands)	March 31, 2023		December 31, 2022
Senior Secured Term Loan due 2026:
Principal	$	400,000	$	400,000
Less: debt discount (1)	(4,190)		(4,571)
Less: deferred financing (1)	(3,152)		(3,439)
Net carrying value of Long-term debt	$	392,658	$	391,990

______________________________

(1) Included in the Company's Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan using the effective interest rate method.

Interest Expense

The following table sets forth interest expense recognized related to the Company's debt for the three months ended March 31, 2023 and 2022, respectively:

	Three Months Ended March 31,
(in thousands)	2023		2022
Contractual interest expense	$	11,230	$	7,500
Amortization of debt discount	$	381	$	364
Amortization of deferred financing	$	286	$	273

7. Stockholder's Equity

During the three months ended March 31, 2023, the Company issued and sold an aggregate of 195,229 shares through its at-the-market equity program ("ATM program") at a weighted-average public offering price of $12.71 per share and received aggregate net proceeds of $2.4 million. As of March 31, 2023, an aggregate of $247.5 million worth of shares remain available to be issued and sold under the ATM program.

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8. Stock-Based Compensation

The Company's Amended and Restated 2007 Equity Incentive Plan (the "Plan") provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Company's Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company's business. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option.

Stock Option Grants

The fair value of the stock options granted is estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:

	Three Months Ended March 31,
	2023			2022
Expected stock price volatility	59.3		%	62.3		%
Risk free interest rate	3.9		%	1.5		%
Expected life of options (years)	5.5			5.3
Expected annual dividend per share	$	—		$	—

 A summary of the Company's stock options for the three months ended March 31, 2023 were as follows:

	Number of Shares	Weighted Average Exercise  Price		Weighted Average Remaining Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Options outstanding, December 31, 2022	19,064	$	11.31
Granted	4,854	$	11.94
Exercised	(384)	$	6.91
Forfeited	(81)	$	10.97
Expired	(16)	$	15.51
Options outstanding, March 31, 2023	23,437	$	11.51	7.0	$	22.8
Vested and unvested expected to vest, March 31, 2023	21,395	$	11.45	6.9	$	22.1
Exercisable at March 31, 2023	12,395	$	10.94	5.3	$	19.4

As of March 31, 2023, the total unrecognized compensation cost related to non-vested stock options granted was $47.1 million and is expected to be recognized over a weighted average period of three years.

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Restricted Stock Units and Performance-Based Restricted Stock Units (collectively "RSUs")

RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee's continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse. A summary of non-vested RSU activity under the Plan for the three months ended March 31, 2023 is as follows:

	Number of Shares	Weighted Average Grant Date Fair Value		Weighted  Average Remaining  Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Non-vested units as of December 31, 2022	9,717	$	13.07
Granted	2,762	$	11.94
Vested	(2,532)	$	12.20
Forfeited	(517)	$	9.68
Non-vested units as of March 31, 2023	9,430	$	13.16	2.4	$	104.6

As of March 31, 2023, there was $63.8 million of total unrecognized compensation cost related to unvested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of two years.

Compensation Expense Related to Equity Awards

The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards:

	Three Months Ended March 31,
(in thousands)	2023		2022
Research and development expense	$	8,490	$	9,365
Selling, general, and administrative expense	26,404		21,286
Total equity compensation expense	$	34,894	$	30,651

9. Assets and Liabilities Measured at Fair Value

The Company's financial assets and liabilities are measured at fair value and classified within the fair value hierarchy, which is defined as follows:

Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date.

Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly.

Level 3 — Inputs that are unobservable for the asset or liability.

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A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of March 31, 2023 are identified in the following tables:

(in thousands)	Level 2		Total
Assets:
Commercial paper	$	106,106	$	106,106
Money market	6,631		6,631
	$	112,737	$	112,737

(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	12,668	$	12,668
Deferred compensation plan liability	6,281		—		6,281
	$	6,281	$	12,668	$	18,949

A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2022 are identified in the following tables:

(in thousands)	Level 2		Total
Assets:
Commercial paper	$	144,381	$	144,381
Money market	5,808		5,808
	$	150,189	$	150,189

(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	21,417	$	21,417
Deferred compensation plan liability	5,458		—		5,458
	$	5,458	$	21,417	$	26,875

The Company's Senior Secured Term Loan due 2026 falls into the Level 2 category within the fair value level hierarchy and the fair value was determined using quoted prices for similar liabilities in active markets, as well as inputs that are observable for the liability (other than quoted prices), such as interest rates that are observable at commonly quoted intervals. The carrying value of the Senior Secured Term Loan due 2026 approximates the fair value.

The Company did not have any Level 3 assets as of March 31, 2023 or December 31, 2022.

Cash, Money Market Funds, and Marketable Securities

The Company classifies its cash within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in an active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available-for-sale and classifies these assets and the money market funds within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities.

Contingent Consideration Payable

The contingent consideration payable resulted from the acquisition of Callidus Biopharma, Inc. ("Callidus") in November 2013. The most recent valuation was determined using a probability weighted discounted cash flow valuation approach. Gains and losses are included in the Company's Consolidated Statements of Operations.

The contingent consideration payable for Callidus has been classified as a Level 3 recurring liability as its valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions

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were used for the various inputs to the valuation approach, the estimated fair value could be significantly higher or lower than the fair value the Company determined.

The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe disease program:

Contingent Consideration Liability	Fair Value as of March 31, 2023		Valuation Technique	Unobservable Input	Range
	(in thousands)
				Discount rate	11.2%
Clinical and regulatory milestones	$	12,668	Probability weighted discounted cash flow	Probability of achievement of milestones	88%
				Projected year of payments	2023

Contingent consideration liabilities are remeasured to fair value each reporting period using discount rates, probabilities of payment, and projected payment dates. Projected contingent payment amounts related to clinical and regulatory based milestones are discounted back to the current period using a discounted cash flow model. Increases in discount rates and the time to payment may result in lower fair value measurements. Increases or decreases in any of those inputs together, or in isolation, may result in a significantly lower or higher fair value measurement. There is no assurance that any of the conditions for the milestone payments will be met.

The Company reached a regulatory milestone in March 2023 associated with the EC granting approval for Pombiliti™, related to the contingent consideration of Callidus for the ATB200 Pompe disease program. The satisfaction of this milestone resulted in the milestone payment of $9.0 million being due, which is payable in cash and recorded as a component of accounts payable on the Company's Consolidated Balance Sheets as of March 31, 2023.

The following table shows the change in the balance of contingent consideration payable for the three months ended March 31, 2023 and 2022, respectively:

	Three Months Ended March 31,
(in thousands)	2023		2022
Balance, beginning of the period	$	21,417	$	20,339
Changes in fair value during the period, included in the Consolidated Statements of Operations	251		(1,188)
Milestone payment payable in cash	(9,000)		—
Balance, end of the period (1)	$	12,668	$	19,151

______________________________

(1) As certain milestones are expected to be reached within the next twelve months, the March 31, 2023 balance was recorded as a current liability in the Company's Consolidated Balance Sheets.

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10. Basic and Diluted Net Loss per Common Share

The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:

	Three Months Ended March 31,
(in thousands, except per share amounts)	2023		2022
Numerator:
Net loss attributable to common stockholders	$	(52,932)	$	(85,260)
Denominator:
Weighted average common shares outstanding — basic and diluted	291,336,750		288,481,741

Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options and unvested RSUs. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect. Weighted average common shares outstanding includes outstanding pre-funded warrants with an exercise price of $0.01.

The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:

	As of March 31,
(in thousands)	2023	2022
Options to purchase common stock	23,437	19,304
Unvested restricted stock units	9,430	10,351
Total number of potentially issuable shares	32,867	29,655

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ITEM 2. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS

The following discussion should be read in conjunction with the unaudited Consolidated Financial Statements and the notes thereto included in this Quarterly Report on Form 10-Q and the audited Consolidated Financial Statements and the notes thereto included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2022. Some of the statements we make in this section are forward-looking statements within the meaning of the federal securities laws. For a complete discussion of forward-looking statements, see the section in this Quarterly Report on Form 10-Q entitled “Special Note Regarding Forward-Looking Statements”. Certain risk factors may cause actual results, performance or achievements to differ materially from those expressed or implied by the following discussion. For a discussion of such risk factors, see the section in our Annual Report on Form 10-K for the fiscal year ended December 31, 2022 entitled “Risk Factors”.

Overview

We are a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. We have a portfolio including the first oral monotherapy for Fabry disease that has achieved widespread global approval and a differentiated biologic for Pompe disease that is under regulatory review. We are committed to discovering and developing next generation therapies in Fabry and Pompe diseases.

The cornerstone of our portfolio is Galafold® (also referred to as "migalastat"), the first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants. Migalastat is currently approved under the trade name Galafold® in the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan, with multiple additional approvals granted and applications pending in several geographies around the world.

The lead biologics program of our pipeline is Amicus Therapeutics GAA ("AT-GAA", also known as ATB200/AT2221, or cipaglucosidase alfa/miglustat), a novel, two-component, potential best-in-class treatment for Pompe disease. AT-GAA is currently under regulatory review. In October 2022, the U.S. Food and Drug Administration ("FDA") deferred action on the BLA for cipaglucosidase alfa, citing the inability to complete the manufacturing site inspection prior to the PDUFA action date. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site. We believe the comments and observations received at the close of the FDA inspection are all addressable. In March 2023, the European Commission ("EC") granted approval for Pombiliti™ (cipaglucosidase alfa) to be used in combination with miglustat for adults with late-onset Pompe disease ("LOPD"). In April 2023, the Committee for Medicinal Products for Human Use ("CHMP") of the European Medicines Agency ("EMA") adopted a positive opinion on Opfolda™ (miglustat), the enzyme stabilizer component of AT-GAA. The regulatory submission process for AT-GAA in the U.K. was initiated in December 2022.

Our Strategy

Our strategy is to create, manufacture, test, and deliver the highest quality medicines for people living with rare diseases through internally developed, jointly developed, acquired, or in-licensed products and product candidates that have the potential to obsolete current treatments, provide significant benefits to patients, and be first- or best-in-class. We are leveraging our global capabilities to develop and broaden our lead franchises in Fabry and Pompe disease, with focused discovery work on next generation therapies and novel platform technologies.

We continue to monitor the novel coronavirus ("COVID-19") pandemic. Our commercial operations have not been significantly impacted by the COVID-19 pandemic and we gradually continue to see an improvement in patient identification and Galafold® initiation. We have been able to continue to meet required commercial demand for Galafold® as well as supply our ongoing Pompe disease clinical studies and access programs including the Early Access to Medicines Scheme ("EAMS") without interruption. In regard to our regulatory operations, the FDA deferred action on the pending BLA for cipaglucosidase alfa, as a site inspection was necessary, however, could not be completed by the PDUFA action date due to COVID-19 related travel restrictions. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site. Per FDA guidance relating to pre-approval inspections during the COVID-19 pandemic, receipt of a deferral action indicates no deficiencies have been identified and the application otherwise satisfies the requirements for approval.

Highlights of our progress include:

•Commercial and regulatory success in Fabry disease. For the three months ended March 31, 2023, Galafold® revenue was $86.1 million of consolidated revenue, which represented an increase of $7.4 million compared to the same period in the prior year. We continue to see strong commercial momentum and expansion into additional geographies. In countries where we have been operating the longest, we see an increasing proportion of previously untreated patients

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come onto Galafold® as compared to treatment experienced patients. In the U.S., we continue to see a significant increase in patients from a growing and very wide prescriber base. Across all markets, we see a high rate of compliance and adherence to this oral treatment option.

•Pompe disease program milestones. AT-GAA is under regulatory reviews. In March 2023, the EC granted approval for Pombiliti™ (cipaglucosidase alfa), a long-term enzyme replacement therapy ("ERT") used in combination with miglustat for adults with LOPD. In April 2023, the CHMP adopted a positive opinion on Opfolda™ (miglustat), the enzyme stabilizer component of AT-GAA. Additionally, multiple expanded access mechanisms are in place around the globe, including in the U.S., U.K., Germany, France, Japan, and others.

•Pipeline advancement and growth. We are leveraging our global capabilities to develop and broaden our lead franchises in Fabry and Pompe disease, with focused discovery work on next generation therapies and novel platform technologies.

•Financial strength. Total cash, cash equivalents, and marketable securities as of March 31, 2023 was $267.1 million. Based on the current operating model, we believe that the current cash position, which includes expected revenues, is sufficient to fund our operations and ongoing research programs for at least the next 12 months. Potential impacts of the COVID-19 pandemic, business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our future capital requirements.

 Our Commercial Product and Product Candidates

Galafold® (migalastat HCl) for Fabry Disease

Our oral precision medicine Galafold® was granted accelerated approval by the FDA in August 2018 under the brand name Galafold® for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene ("GLA") variant based on in vitro assay data. The FDA has approved Galafold® for 350 amenable GLA variants. Galafold® was approved in the E.U. and U.K. in May 2016 as a first-line therapy for long-term treatment of adults and adolescents, aged 16 years and older, with a confirmed diagnosis of Fabry disease and who have an amenable mutation (variant). The approved E.U. and U.K. labels include 1,384 mutations amenable to Galafold® treatment, which represent up to half of all patients with Fabry disease. In countries where mutations are provided only on the amenability website, these 1,384 amenable mutations are now available. Marketing authorization approvals as well as approvals for adolescents aged 12 years and older weighing 45 kg or more have been granted in over 40 countries around the world, including 4 new approvals in 2023. We plan to continue to launch Galafold® in additional countries during 2023, including for adolescents aged 12 years and older.

As an orally administered monotherapy, Galafold® is designed to bind to and stabilize an endogenous alpha-galactosidase A ("alpha-Gal A") enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice ("GLP") cell-based amenability assay. Galafold® is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic variants, and at this time, it is not intended for concomitant use with ERT.

The Galafold® U.S. patent portfolio encompasses 49 Orange Book listed patents, including 8 composition-of-matter patents, of which 33 provide protection through at least 2038.

Next Generation for Fabry Disease

We are committed to continued innovation for all people living with Fabry disease. As part of our long-term commitment, we have an academic research collaboration agreement to explore next generation pharmacological chaperones for Fabry disease.

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Novel ERT for Pompe Disease

We are leveraging our biologics capabilities to develop AT-GAA, a novel treatment paradigm for Pompe disease. AT-GAA consists of a uniquely engineered rhGAA enzyme, ATB200, or cipaglucosidase alfa, with an optimized carbohydrate structure to enhance lysosomal uptake, administered in combination with AT2221, or miglustat, that functions as an enzyme stabilizer. Miglustat binds to and stabilizes ATB200 preventing inactivation of rhGAA in circulation to improve the uptake of active enzyme in key disease-relevant tissues, resulting in increased clearance of accumulated substrate, ("glycogen"). Miglustat is not an active ingredient that contributes directly to glycogen reduction.

In February 2021, we reported topline results from the Phase 3 PROPEL study. Of the Pompe disease patients enrolled, 77% were being treated with alglucosidase alfa (n=95) immediately prior to enrollment (“Switch”) and 23% had never been treated with any ERT (n=28) (“Naïve”). Nearly all patients from the PROPEL study continue to be treated with AT-GAA in the extension clinical study. The clinical data from the PROPEL study, the extension study as well as the Phase 1/2 study were included in the AT-GAA submissions to the FDA and the EMA.

In October 2022 and February 2023, we reported positive long-term data from our ongoing phase 1/2 clinical study and Phase 3 open-label extension study, respectively. Phase 1/2 and 3 study participants treated with AT-GAA for up to 48 months and up to 2 years, respectively, demonstrated persistent and durable effects on six-minute walk test distance and measures of motor function and muscle strength, stability, or increase in forced vital capacity, and reductions in biomarkers of muscle damage and disease substrate.

In March 2023, the EC granted approval for Pombiliti™ (cipaglucosidase alfa) to be used in combination with miglustat for adults with LOPD. In April 2023, the CHMP adopted a positive opinion on Opfolda™ (miglustat), the enzyme stabilizer component of AT-GAA. The regulatory submission process for AT-GAA in the U.K. was initiated in December 2022.

In addition, we are conducting ongoing clinical studies in pediatric patients for both LOPD and infantile-onset Pompe disease ("IOPD") populations.

Next Generation for Pompe Disease

We are committed to continued innovation for all people living with Pompe disease. As part of our long-term commitment, we are also continuing discovery for next-generation genetic medicines for Pompe disease.

Additional Development and Next Generation Programs

We are researching potential therapies for CDKL5 deficiency disorder ("CDD"). We are collaborating with the LouLou Foundation to assess the natural history of the disease to identify endpoints for potential use in future studies. We also have a number of additional gene therapies in clinical and preclinical development, including potential gene therapies in multiple forms of Batten disease.

Strategic Alliances and Arrangements

We will continue to evaluate business development opportunities as appropriate to build stockholder value and provide us with access to the financial, technical, clinical, and commercial resources necessary to develop and market technologies or products with a focus on rare and orphan diseases. We are exploring potential collaborations, alliances, and other business development opportunities on a regular basis. These opportunities may include business combinations, partnerships, the strategic out-licensing of certain assets, or the acquisition of preclinical-stage, clinical-stage, or marketed products or platform technologies consistent with our strategic plan to develop and provide therapies to patients living with rare and orphan diseases.

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Consolidated Results of Operations

Three Months Ended March 31, 2023 compared to March 31, 2022

The following table provides selected financial information for the Company:

	Three Months Ended March 31,
(in thousands)	2023			2022			Change
Net product sales	$	86,270		$	78,715		$	7,555
Cost of goods sold	6,942			7,582			(640)
Cost of goods sold as a percentage of net product sales	8.0		%	9.6		%	(1.6)		%
Operating expenses:
Research and development	41,499			81,517			(40,018)
Selling, general, and administrative	73,957			58,116			15,841
Changes in fair value of contingent consideration payable	251			(1,188)			1,439
Loss on impairment of assets	—			6,616			(6,616)
Depreciation and amortization	1,257			1,411			(154)
Other (expense) income:
Interest income	2,199			133			2,066
Interest expense	(11,844)			(8,147)			(3,697)
Other (expense) income	(5,938)			1,902			(7,840)
Income tax benefit (expense)	287			(3,809)			4,096
Net loss attributable to common stockholders	$	(52,932)		$	(85,260)		$	32,328

Net Product Sales. Net product sales increased $7.6 million during the three months ended March 31, 2023 compared to the same period in the prior year. The increase was primarily due to continued growth in the U.S., Europe and Japan markets, partially offset by the $3.8 million unfavorable impact of foreign currency exchange.

Cost of goods sold. Cost of goods sold includes manufacturing costs as well as royalties associated with net product sales. Cost of goods sold as a percentage of net product sales decreased 1.6% primarily due to the increased proportion of sales in countries not subject to royalties.

22

Research and Development Expense. The following table summarizes our principal development programs and the out-of-pocket, third-party expenses incurred:

(in thousands)	Three Months Ended March 31,
Projects	2023		2022
Third party direct project expenses
Galafold® (Fabry Disease)	$	2,644	$	3,620
AT-GAA (Pompe Disease)	15,776		26,978
Gene therapy programs	235		17,692
Pre-clinical and other programs	365		93
Total third-party direct project expenses	19,020		48,383
Other project costs
Personnel costs	18,252		25,675
Other costs	4,227		7,459
Total other project costs	22,479		33,134
Total research and development costs	$	41,499	$	81,517

The $40.0 million decrease in research and development costs was primarily driven by a decrease in gene therapy programs primarily due to contract exit costs in the prior year related to the strategic prioritization of our gene therapy portfolio and a decrease in Pompe disease program spend associated with timing of manufacturing costs. Personnel costs decreased in connection with the reallocation of resources to support our anticipated AT-GAA commercial launch and continued growth of Galafold®.

Selling, General, and Administrative Expense. Selling, general, and administrative expense increased $15.8 million, primarily driven by personnel costs in connection with the reallocation of resources to support our anticipated AT-GAA commercial launch and third-party professional fees.

Loss on Impairment of Assets. In connection with the strategic prioritization of our gene therapy portfolio in the prior year, the Company recognized a $6.6 million loss on impairment of assets for the three months ended March 31, 2022.

Interest Expense. The $3.7 million variance was due to a higher variable interest rate on debt period over period.

Other (Expense) Income. The $7.8 million variance was primarily related to foreign exchange losses caused by remeasurement of foreign-denominated balances.

Income Tax Benefit. We are subject to income taxes in various jurisdictions. The income tax benefit was primarily due to the recognition of a $4.9 million tax benefit in connection with a partial release of a valuation allowance on deferred tax assets resulting from the reclassification of in-process research and development to a definite-lived intangible asset.

Liquidity and Capital Resources

As a result of our significant research and development expenditures, as well as expenditures to build a commercial organization to support the launch of Galafold®, we have not been profitable and have generated operating losses since we were incorporated in 2002. We have historically funded our operations through stock offerings, Galafold® revenues, debt issuance, collaborations, and other financing arrangements.

Sources of Liquidity

In November 2022, we entered into a Sales Agreement with The Goldman Sachs & Co. LLC to create an at-the-market equity program ("ATM program") , pursuant to which we may offer to sell shares of our common stock having an aggregate offering gross proceeds of up to $250.0 million. During the three months ended March 31, 2023, we issued and sold an aggregate of 195,229 shares through our ATM program at a weighted-average public offering price of $12.71 per share and received net proceeds of $2.4 million. As of March 31, 2023, an aggregate of $247.5 million of shares remain available to be issued and sold under the ATM program.

23

Cash Flow Discussion

As of March 31, 2023, we had cash, cash equivalents, and marketable securities of $267.1 million. We invest cash in excess of our immediate requirements in regard to liquidity and capital preservation in a variety of interest-bearing instruments, including obligations of U.S. government agencies and money market accounts. Wherever possible, we seek to minimize the potential effects of concentration and degrees of risk. Although we maintain cash balances with financial institutions in excess of insured limits, we do not anticipate any losses with respect to such cash balances. For more details on the cash, cash equivalents, and marketable securities, refer to "— Note 4. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash," in our Notes to Consolidated Financial Statements.

Net Cash Used in Operating Activities

Net cash used in operations for the three months ended March 31, 2023 was $18.1 million. The components of net cash used in operations included the net loss for the three months ended March 31, 2023 of $52.9 million offset by $34.9 million of stock compensation, $3.1 million of other non-cash adjustments, and a net increase in changes in operating assets and liabilities of $3.2 million. The changes in operating assets and liabilities were primarily due to an increase in inventory of $3.2 million.

Net cash used in operations for the three months ended March 31, 2022 was $58.3 million. The components of net cash used in operations included the net loss for the three months ended March 31, 2022 of $85.3 million and an overall decrease in cash from changes in operating assets and liabilities of $11.9 million. The changes in operating assets and liabilities were primarily related to the payment of contract manufacturing and annual performance bonus. This was partially offset by $30.7 million of stock compensation and $8.1 million of other non-cash adjustments.

Net Cash Provided by Investing Activities

Net cash provided by investing activities for the three months ended March 31, 2023 was $36.3 million. Our investing activities have consisted primarily of purchases, sales, and maturities of investments and capital expenditures. Net cash provided by investing activities reflects $54.9 million for the sale and redemption of marketable securities, partially offset by $16.7 million for the purchase of marketable securities and $1.9 million for capital expenditures.

Net cash provided by investing activities for the three months ended March 31, 2022 was $58.2 million. Our investing activities have consisted primarily of purchases and sales and maturities of investments and capital expenditures. Net cash provided by investing activities reflects $108.3 million for the sale and redemption of marketable securities, partially offset by $49.2 million for the purchase of marketable securities and $0.9 million for capital expenditures.

Net Cash Used in Financing Activities

Net cash used in financing activities for the three months ended March 31, 2023 was $7.8 million. Net cash used in financing activities primarily reflects the purchase of vested restricted stock units of $12.8 million, partially offset by $2.7 million of proceeds from the exercise of stock options and $2.4 million of proceeds from the issuance of shares in connection with the ATM program offering, net of issuance costs.

Net cash used in financing activities for the three months ended March 31, 2022 was $8.2 million. Net cash used in financing activities primarily reflects the purchase of vested restricted stock units of $9.0 million, partially offset by $0.9 million of proceeds from the exercise of stock options.

Funding Requirements

We expect to continue to incur significant costs in the foreseeable future primarily due to research and development expenses, including expenses related to conducting clinical trials. Our future capital requirements will depend on a number of factors, including:

•the scope, progress, results and costs of clinical trials for our drug candidates;

•the cost of manufacturing drug supply for our commercial, clinical and preclinical studies, including the cost of manufacturing Pompe Enzyme Replacement Therapy ("ERT" or "ATB200" or "cipaglucosidase alfa");

24

•the future results of preclinical research and subsequent clinical trials for pipeline candidates we may identify from time to time, including our ability to obtain regulatory approvals and commercialize these therapies and obtain market acceptance for such therapies;

•the costs, timing, and outcome of regulatory review of our product candidates, including AT-GAA;

•any changes in regulatory standards relating to the review of our product candidates, including AT-GAA;

•the number and development requirements of other product candidates that we pursue;

•the costs of commercialization activities, including product marketing, sales, and distribution;

•the emergence of competing technologies and other adverse market developments;

•the estimates regarding the potential market opportunity for our product and product candidates, including AT-GAA;

•our ability to successfully commercialize Galafold® (also referred to as "migalastat HCl") and, if our regulatory applications are approved, AT-GAA;

•our ability to manufacture or supply sufficient clinical or commercial products, including Galafold® and AT-GAA;

•our ability to obtain reimbursement for Galafold® and, if our regulatory applications are approved, AT-GAA;

•our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold®, and, if approved and applicable, AT-GAA;

•our ability to obtain market acceptance of Galafold® and, if our regulatory applications are approved, AT-GAA;

•the costs of preparing, filing, and prosecuting patent applications and maintaining, enforcing, and defending intellectual property-related claims, including Hatch-Waxman litigation;

•the impact of litigation that has been or may be brought against us or of litigation that we are pursuing or may pursue against others, including Hatch-Waxman litigation;

•the extent to which we acquire or invest in businesses, products, and technologies;

•our ability to successfully integrate our acquired products and technologies into our business, or successfully divest or license existing products and technologies from our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;

•our ability to establish licensing agreements, collaborations, partnerships or other similar arrangements and to obtain milestone, royalty, or other payments from any such collaborators;

•the extent to which our business could be adversely impacted by the effects of the novel coronavirus ("COVID-19") outbreak, including actions by us, governments, our customers, our suppliers, or other third parties to control the spread of COVID-19, or by other health epidemics or pandemics;

•the costs associated with, and our ability to comply with, emerging environmental, social and governance standards;

•our ability to accurately forecast revenue, operating expenditures, or other metrics impacting profitability;

•fluctuations in foreign currency exchange rates; and

•changes in accounting standards.

We may seek additional funding through public or private financings of debt or equity. Based on our current operating model, we believe that the current cash position, which includes expected revenues, is sufficient to fund our operations and ongoing research programs for at least the next 12 months. Potential impacts of the COVID-19 pandemic, business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our future capital requirements.

Financial Uncertainties Related to Potential Future Payments

Milestone Payments / Royalties

25

Callidus - In connection with our acquisition of Callidus Biopharma, Inc. ("Callidus"), we may be obligated to make additional payments to the former stockholders of Callidus upon the achievement of certain clinical milestones of up to $35 million and regulatory milestones of up to $80 million set forth in the merger agreement, provided that the aggregate merger consideration shall not exceed $130 million. As of March 31, 2023, $20 million and $68 million remain outstanding, respectively. Refer to “— Note 9. Assets and Liabilities Measured at Fair Value,” to the Consolidated Financial Statements.

Celenex - In connection with our acquisition of Celenex, Inc. ("Celenex"), we may be obligated to pay up to an additional $10 million in connection with the achievement of certain development milestones, $220 million in connection with the achievement of certain regulatory approval milestones across multiple programs and up to $75 million in tiered sales milestone payments. Celenex has an exclusive license agreement with Nationwide Children’s Hospital ("Nationwide Children’s"). Under this license agreement, Nationwide Children’s is eligible to receive development and sales-based milestones of up to $7.8 million for each product.

University of Pennsylvania - In connection with our license agreement with the University of Pennsylvania ("Penn"), Penn is eligible to receive up to an aggregate of $86.5 million for the achievement of certain milestones and royalty payments with respect to licensed products for each indication. Royalty payments are based on net sales of licensed products on a licensed product-by-licensed product and country-by-country basis.

GlaxoSmithKline - In connection with our collaboration agreement with GlaxoSmithKline ("GSK"), pursuant to which we obtained global rights to develop and commercialize Galafold® as a monotherapy and in combination with ERT for Fabry disease, GSK is eligible to receive post-approval and sales-based milestones up to $40 million, as well as tiered royalties in the mid-teens in eight major markets outside the U.S.

Critical Accounting Policies and Significant Judgments 

The discussion and analysis of our financial condition and results of operations are based on our financial statements, which we have prepared in accordance with U.S. GAAP. The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported revenues and expenses during the reporting periods. On an ongoing basis, we evaluate our estimates and judgments, including those described in greater detail below. We base our estimates on historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions.

There were no significant changes during the three months ended March 31, 2023 to the items that we disclosed as our significant accounting policies and estimates described in "—Note 2. Summary of Significant Accounting Policies" to the Company's financial statements as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2022.

Recent Accounting Pronouncements

Please refer to "—Note 2. Summary of Significant Accounting Policies" in our Notes to Consolidated Financial Statements.

26

ITEM 3. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK

Our market risks, and the way we manage them, are summarized in Part II, Item 7A, "Quantitative and Qualitative Disclosures About Market Risk" of our Annual Report on Form 10-K for the fiscal year ended December 31, 2022. As of March 31, 2023, there have been no material changes to our market risks or to our management of such risks since December 31, 2022.

ITEM 4. CONTROLS AND PROCEDURES

Evaluation of Disclosure Controls and Procedures

As of the end of the period covered by this Quarterly Report on Form 10-Q, an evaluation of the effectiveness of our disclosure controls and procedures (pursuant to Rule 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the "Exchange Act") was carried out under the supervision of our Principal Executive Officer and Principal Financial Officer, with the participation of our management. Based on that evaluation, the Principal Executive Officer and the Principal Financial Officer concluded that, as of the end of such period, our disclosure controls and procedures are effective in recording, processing, summarizing and reporting, on a timely basis, information required to be disclosed by us in the reports that we file or submit under the Exchange Act and are effective in ensuring that information required to be disclosed by us in the reports that we file or submit under the Exchange Act is accumulated and communicated to our management, including our Principal Executive Officer and Principal Financial Officer, as appropriate, to allow timely decisions regarding required disclosure.

Changes in Internal Control Over Financial Reporting

In the first quarter of 2023, we implemented a new enterprise resource planning ("ERP") system for financial reporting. In connection with the implementation, there were material changes to our internal control over financial reporting as defined in Rule 13a-15(f) and 15d-15(f) under the Exchange Act. We believe our new ERP system will facilitate better transactional processing, enhanced reporting and oversight, and function as an important component of our transactional and reporting controls and procedures.

PART II. OTHER INFORMATION

ITEM 1. LEGAL PROCEEDINGS

In the fourth quarter of 2022, the Company received Paragraph IV Certification Notice Letters from Teva Pharmaceuticals USA, Inc. ("Teva"), Aurobindo Pharma Limited ("Aurobindo"), and Lupin Limited ("Lupin") in connection with Abbreviated New Drug Applications (“ANDA”) filed with the FDA requesting approval to market generic Galafold®. In November 2022, the Company filed four lawsuits against Teva, Lupin, and Aurobindo in the U.S. District Court for the District of Delaware for infringement of its Orange Book-listed patents and will vigorously enforce its Galafold® intellectual property rights.

ITEM 1A. RISK FACTORS

There have been no material changes to the risk factors previously disclosed in our Annual Report on Form 10-K for the fiscal year ended December 31, 2022.

ITEM 2. UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS

Recent Sales of Unregistered Securities

None.

27

Issuer Purchases of Equity Securities

The following table provides certain information with respect to purchase of our common stock during the three months ended March 31, 2023:

Period	Total Number of Shares Purchased (1)	Average Price Paid per Share		Total Number of Shares Purchased as Part of Publicly Announced Plans or Programs	Maximum Number (or Approximate Dollar Value) of Shares That May Yet Be Purchased Under the Plans or Programs
January 1, 2023 through January 31, 2023	938,481	$	12.07	—	—
February 1, 2023 through February 28, 2023	15,148	$	13.21	—	—
March 1, 2023 through March 31, 2023	170,987	$	11.45	—	—
Total	1,124,616	$	11.99	—	—

______________________________

(1) Represents shares of common stock withheld to satisfy taxes associated with the vesting of restricted stock units

ITEM 3. DEFAULTS UPON SENIOR SECURITIES

None.

ITEM 4. MINE SAFETY DISCLOSURES

None.

ITEM 5. OTHER INFORMATION

None.

28

ITEM 6. EXHIBITS

Exhibit Number	Description
31.1	Certification of Principal Executive Officer pursuant to Rules 13a-14 and 15d-14 promulgated pursuant to the Securities Exchange Act of 1934, as amended
31.2	Certification of Principal Financial Officer pursuant to Rules 13a-14 and 15d-14 promulgated pursuant to the Securities Exchange Act of 1934, as amended
32.1	Certification of Principal Executive Officer and Principal Financial Officer pursuant to Section 906 of the Sarbanes-Oxley Act of 2002
101.INS	Inline 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 Linkbase Document
101.LAB	Inline XBRL Taxonomy Extension Label Linkbase Document
101.PRE	Inline XBRL Taxonomy Extension Presentation Linkbase Document
101.DEF	Inline XBRL Taxonomy Extension Definition Linkbase Document
104	Cover Page Interactive Data File (formatted in Inline XBRL and included in Exhibit 101)

29

SIGNATURES

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

		AMICUS THERAPEUTICS, INC.
Date:	May 10, 2023	By:	/s/ Bradley L. Campbell
			Bradley L. Campbell
			President and Chief Executive Officer
			(Principal Executive Officer)
Date:	May 10, 2023	By:	/s/ Daphne Quimi
			Daphne Quimi
			Chief Financial Officer
			(Principal Financial Officer)
			(Principal Accounting Officer)

30

EX-31.1

Exhibit 31.1

CERTIFICATIONS PURSUANT TO SECTION 302 OF

THE SARBANES-OXLEY ACT OF 2002

CERTIFICATION BY PRINCIPAL EXECUTIVE OFFICER

I, Bradley L. Campbell, certify that:

1.I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, 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 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 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 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: May 10, 2023	/s/ Bradley L. Campbell
	Bradley L. Campbell
	President and Chief Executive Officer

EX-31.2

Exhibit 31.2

CERTIFICATIONS PURSUANT TO SECTION 302 OF

THE SARBANES-OXLEY ACT OF 2002

CERTIFICATION BY PRINCIPAL FINANCIAL OFFICER

I, Daphne Quimi, certify that:

1.I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, 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 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 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 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: May 10, 2023	/s/ Daphne Quimi
	Daphne Quimi
	Chief Financial Officer

EX-32.1

Exhibit 32.1

CERTIFICATION OF PRINCIPAL EXECUTIVE OFFICER AND

PRINCIPAL FINANCIAL OFFICER PURSUANT TO

18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TO

SECTION 906 OF THE SARBANES-OXLEY ACT OF 2002

    Each of the undersigned hereby certifies, pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, in his or her capacity as an officer of Amicus Therapeutics, Inc. (the “Company”), that, to his or her knowledge, the Quarterly Report of the Company on Form 10-Q for the period ended March 31, 2023, fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934 (15 U.S.C. 78m or 78o(d)) and that the information contained in such report fairly presents, in all material respects, the financial condition and results of operations of the Company. This written statement is being furnished to the Securities and Exchange Commission as an exhibit to such Form 10-Q. A signed original of this statement has been provided to the Company and will be retained by the Company and furnished to the Securities and Exchange Commission or its staff upon request.

Date: May 10, 2023	By:	/s/ Bradley L. Campbell
		Bradley L. Campbell
		President and Chief Executive Officer
Date: May 10, 2023	By:	/s/ Daphne Quimi
		Daphne Quimi
		Chief Financial Officer

XML

Cover - shares	3 Months Ended
	Mar. 31, 2023	Apr. 27, 2023
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Mar. 31, 2023
Document Transition Report	false
Entity File Number	001-33497
Entity Registrant Name	Amicus Therapeutics, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	71-0869350
Entity Address, Address Line One	3675 Market Street,
Entity Address, City or Town	Philadelphia,
Entity Address, State or Province	PA
Entity Address, Postal Zip Code	19104
City Area Code	(215)
Local Phone Number	921-7600
Title of 12(b) Security	Common Stock, par value $0.01 per share
Trading Symbol	FOLD
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		284,575,686
Entity Central Index Key	0001178879
Amendment Flag	false
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q1

XML

Consolidated Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Current assets:
Cash and cash equivalents	$ 160,602	$ 148,813
Investments in marketable securities	106,507	144,782
Accounts receivable	68,178	66,196
Inventories	27,004	23,816
Prepaid expenses and other current assets	37,406	40,209
Total current assets	399,697	423,816
Operating lease right-of-use assets, net	28,483	29,534
Property and equipment, less accumulated depreciation of $22,901 and $22,281 at March 31, 2023 and December 31, 2022, respectively	31,406	30,778
Intangible assets, less accumulated amortization of $36 and $0 at March 31, 2023 and December 31, 2022, respectively	22,964	23,000
Goodwill	197,797	197,797
Other non-current assets	20,172	19,242
Total Assets	700,519	724,167
Current liabilities:
Accounts payable	24,965	15,413
Accrued expenses and other current liabilities	92,747	93,636
Contingent consideration payable	12,668	21,417
Operating lease liabilities	8,005	8,552
Total current liabilities	138,385	139,018
Long-term debt	392,658	391,990
Operating lease liabilities	51,349	51,578
Deferred reimbursements	5,906	4,656
Deferred income taxes	0	4,939
Other non-current liabilities	9,648	8,939
Total liabilities	597,946	601,120
Commitments and contingencies
Stockholders’ equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 283,300,585 and 281,108,273 shares issued and outstanding at March 31, 2023 and December 31, 2022, respectively	2,820	2,815
Additional paid-in capital	2,691,836	2,664,744
Accumulated other comprehensive loss:
Foreign currency translation adjustment	(6,543)	(11,989)
Unrealized loss on available-for-sale securities	(201)	(116)
Warrants	83	83
Accumulated deficit	(2,585,422)	(2,532,490)
Total stockholders’ equity	102,573	123,047
Total Liabilities and Stockholders’ Equity	$ 700,519	$ 724,167

XML

Consolidated Balance Sheets (Parenthetical) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Statement of Financial Position [Abstract]
Accumulated depreciation of property and equipment	$ 22,901	$ 22,281
Accumulated amortization of intangible assets	$ 36	$ 0
Common stock, par value (in dollars per share)	$ 0.01	$ 0.01
Common stock, shares authorized (in shares)	500,000,000	500,000,000
Common stock, shares issued (in shares)	283,300,585	281,108,273
Common stock, shares outstanding (in shares)	283,300,585	281,108,273

XML

Consolidated Statements of Operations - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Revenue:
Net product sales	$ 86,270	$ 78,715
Cost of goods sold	6,942	7,582
Gross profit	79,328	71,133
Operating expenses:
Research and development	41,499	81,517
Selling, general, and administrative	73,957	58,116
Changes in fair value of contingent consideration payable	251	(1,188)
Loss on impairment of assets	0	6,616
Depreciation and amortization	1,257	1,411
Total operating expenses	116,964	146,472
Loss from operations	(37,636)	(75,339)
Other (expense) income:
Interest income	2,199	133
Interest expense	(11,844)	(8,147)
Other (expense) income	(5,938)	1,902
Loss before income tax	(53,219)	(81,451)
Income tax benefit (expense)	287	(3,809)
Net loss attributable to common stockholders	$ (52,932)	$ (85,260)
Net loss attributable to common stockholders per common share - basic (in dollars per share)	$ (0.18)	$ (0.30)
Net loss attributable to common stockholders per common share — diluted (in dollars per share)	$ (0.18)	$ (0.30)
Weighted average common shares outstanding - basic (in shares)	291,336,750	288,481,741
Weighted average common shares outstanding - diluted (in shares)	291,336,750	288,481,741

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Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Statement of Comprehensive Income [Abstract]
Net loss	$ (52,932)	$ (85,260)
Other comprehensive gain (loss):
Foreign currency translation adjustment gain (loss)	5,446	(5,671)
Unrealized loss on available-for-sale securities	(85)	(338)
Other comprehensive gain (loss)	5,361	(6,009)
Comprehensive loss	$ (47,571)	$ (91,269)

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Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands	Total	ATM	Common Stock	Common Stock ATM	Additional Paid-In Capital	Additional Paid-In Capital ATM	Warrants	Other Comprehensive Gain (Loss)	Accumulated Deficit
Beginning balance (in shares) at Dec. 31, 2021			278,912,800
Beginning balance at Dec. 31, 2021	$ 307,369		$ 2,808		$ 2,595,419		$ 83	$ 4,981	$ (2,295,922)
Increase (Decrease) in Stockholders' Equity
Stock options exercised, net (in shares)			145,449
Stock options exercised, net	859		$ 1		858
Vesting of restricted stock units, net of taxes (in shares)			1,075,607
Vesting of restricted stock units, net of taxes	(8,993)				(8,993)
Stock-based compensation	30,651				30,651
Unrealized loss on available-for-sale securities	(338)							(338)
Foreign currency translation adjustment	(5,671)							(5,671)
Net loss	(85,260)								(85,260)
Ending balance (in shares) at Mar. 31, 2022			280,133,856
Ending balance at Mar. 31, 2022	$ 238,617		$ 2,809		2,617,935		83	(1,028)	(2,381,182)
Beginning balance (in shares) at Dec. 31, 2022	281,108,273		281,108,273
Beginning balance at Dec. 31, 2022	$ 123,047		$ 2,815		2,664,744		83	(12,105)	(2,532,490)
Increase (Decrease) in Stockholders' Equity
Stock options exercised, net (in shares)	384,000		384,108
Stock options exercised, net	$ 2,655		$ 3		2,652
Vesting of restricted stock units, net of taxes (in shares)			1,612,975
Vesting of restricted stock units, net of taxes	(12,806)				(12,806)
Stock-based compensation	34,894				34,894
Issuance of shares in connection with at-the-market offering, net of issuance costs (in shares)				195,229
Issuance of shares in connection with at-the-market offering, net of issuance costs		$ 2,354		$ 2		$ 2,352
Unrealized loss on available-for-sale securities	(85)							(85)
Foreign currency translation adjustment	5,446							5,446
Net loss	$ (52,932)								(52,932)
Ending balance (in shares) at Mar. 31, 2023	283,300,585		283,300,585
Ending balance at Mar. 31, 2023	$ 102,573		$ 2,820		$ 2,691,836		$ 83	$ (6,744)	$ (2,585,422)

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Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Operating activities
Net loss	$ (52,932)	$ (85,260)
Adjustments to reconcile net loss to net cash used in operating activities:
Amortization of debt discount and deferred financing	667	637
Depreciation and amortization	1,257	1,411
Stock-based compensation	34,894	30,651
Non-cash changes in the fair value of contingent consideration payable	251	(1,188)
Foreign currency remeasurement loss	5,885	673
Deferred taxes	(4,939)	0
Asset impairment charges and other asset write-offs	0	6,616
Changes in operating assets and liabilities:
Accounts receivable	(1,367)	(1,000)
Inventories	(3,158)	1,812
Prepaid expenses and other current assets	1,839	3,332
Accounts payable, accrued expenses, and other current liabilities	(72)	(14,563)
Other non-current assets and liabilities	(394)	(1,436)
Net cash used in operating activities	(18,069)	(58,315)
Investing activities
Sale and redemption of marketable securities	54,944	108,328
Purchases of marketable securities	(16,747)	(49,244)
Capital expenditures	(1,942)	(871)
Net cash provided by investing activities	36,255	58,213
Financing activities
Payment of finance leases	(28)	(20)
Purchase of vested restricted stock units, net of taxes	(12,806)	(8,993)
Proceeds from stock options exercised, net	2,655	859
Proceeds from the issuance of shares in connection with at-the-market offering, net of issuance costs	2,354	0
Net cash used in financing activities	(7,825)	(8,154)
Effect of exchange rate changes on cash, cash equivalents, and restricted cash	1,507	(3,377)
Net increase (decrease) in cash, cash equivalents, and restricted cash at the end of the period	11,868	(11,633)
Cash, cash equivalents, and restricted cash at the beginning of period	153,115	249,456
Cash, cash equivalents, and restricted cash at the end of period	164,983	237,823
Supplemental disclosures of cash flow information
Cash paid during the period for interest	11,361	7,509
Cash paid for taxes	178	456
Capital expenditures unpaid at the end of period	$ 1,260	$ 72

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Description of Business

3 Months Ended

Mar. 31, 2023

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Description of Business

Description of Business

Amicus Therapeutics, Inc. (the "Company") is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. The Company has a portfolio including the first, oral monotherapy for Fabry disease that has achieved widespread global approval and a differentiated biologic for Pompe disease, that is under regulatory review. The Company is committed to discovering and developing next generation therapies in Fabry and Pompe diseases.

The cornerstone of the Company's portfolio is Galafold® (also referred to as "migalastat"), the first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants. Migalastat is currently approved under the trade name Galafold® in the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan, with multiple additional approvals granted and applications pending in several geographies around the world.

The lead biologics program of the Company's pipeline is Amicus Therapeutics GAA ("AT-GAA", also known as ATB200/AT2221, or cipaglucosidase alfa/miglustat), a novel, two-component, potential best-in-class treatment for Pompe disease. AT-GAA is currently under review with regulatory authorities. In October 2022, the U.S. Food and Drug Administration ("FDA") deferred action on the BLA for cipaglucosidase alfa, citing the inability to complete the manufacturing site inspection prior to the PDUFA action date. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site. The Company believes the comments and observations received at the close of the FDA inspection are all addressable. In March 2023, the European Commission ("EC") granted approval for Pombiliti™ (cipaglucosidase alfa) to be used in combination with miglustat for adults with late-onset Pompe disease ("LOPD"). In April 2023, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion on Opfolda™ (miglustat), the enzyme stabilizer component of AT-GAA. The regulatory submission process for AT-GAA in the U.K. was initiated in December 2022.

The Company continues to monitor the novel coronavirus (“COVID-19”) pandemic. The Company's commercial operations have not been significantly impacted by the COVID-19 pandemic and the Company gradually continues to see an improvement in patient identification and Galafold® initiation. The Company has maintained operations in all geographies, secured its global supply chain for its commercial and clinical products, as well as maintained the operational integrity of its clinical trials, with minimum disruptions. The Company has been able to continue to meet required commercial demand for Galafold® as well as supply its ongoing Pompe disease clinical studies and access programs including the Early Access to Medicines Scheme ("EAMS") without interruption. In regard to the Company's regulatory operations, the FDA deferred action on the pending BLA for cipaglucosidase alfa, as a site inspection was necessary, however, could not be completed by the PDUFA action date due to COVID-19 pandemic related travel restrictions. In the second quarter of 2023, the FDA completed the required pre-approval inspection of the manufacturing site. Per FDA guidance related to pre-approval inspections during the COVID-19 pandemic, receipt of a deferral action indicates no deficiencies have been identified and the application otherwise satisfies the requirements for approval.

The Company had an accumulated deficit of $2.6 billion as of March 31, 2023 and anticipates incurring losses through the fiscal year ending December 31, 2023. The Company has historically funded its operations through stock offerings, Galafold® revenues, debt issuances, collaborations, and other financing arrangements.

Based on its current operating model, the Company believes that the current cash position, which includes expected revenues, is sufficient to fund the Company's operations and ongoing research programs for at least the next 12 months. Potential impacts of the COVID-19 pandemic, business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact the Company's future capital requirements.

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Summary of Significant Accounting Policies

3 Months Ended

Mar. 31, 2023

Accounting Policies [Abstract]

Summary of Significant Accounting Policies

Summary of Significant Accounting Policies

Basis of Presentation

The Company has prepared the accompanying unaudited Consolidated Financial Statements in accordance with the U.S. generally accepted accounting principles ("U.S. GAAP") for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. In the opinion of management, the accompanying unaudited Consolidated Financial Statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company's interim financial information.

The accompanying unaudited Consolidated Financial Statements and related notes should be read in conjunction with the Company's financial statements and related notes as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2022. For a complete description of the Company's accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2022.

Consolidation

The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.

Foreign Currency Transactions

The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity.

Use of Estimates

The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.

Additionally, the Company assessed the impact the COVID-19 pandemic had on its operations and financial results as of March 31, 2023 and through the issuance of these financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and revenue and expenses.

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive loss in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs.

Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included in other current assets and other non-current assets on the Company's Consolidated Balance Sheets.

Concentration of Credit Risk

The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities.

The Company is subject to credit risk from its accounts receivable related to its product sales of Galafold®. The Company's accounts receivable at March 31, 2023 have arisen from product sales primarily in Europe, the U.S., and Japan. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis. As of March 31, 2023, the Company's allowance for doubtful accounts was $0.1 million.

Revenue Recognition

The Company's net product sales consist primarily of sales of Galafold® for the treatment of Fabry disease. Galafold® sales for the three months ended March 31, 2023 and 2022 were $86.1 million and $78.7 million, respectively. The Company has recorded revenue on sales where Galafold® is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold® are generally received from distributors and pharmacies, with the ultimate payor often a government authority.

The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of Galafold®. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale of Galafold® is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.

The following table summarizes the Company's net product sales disaggregated by geographic area:

	Three Months Ended March 31,
(in thousands)	2023		2022
U.S.	$	28,831	$	24,178
Ex-U.S.	57,439		54,537
Total net product sales	$	86,270	$	78,715

Inventories and Cost of Goods Sold

Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations.

Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable.

Intangible Assets and Goodwill

The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Goodwill is assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill impairment test is conducted. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference.

Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and, if applicable, impairment charges. Amortization of finite-lived intangible assets is recorded over the assets’ estimated useful lives on a straight-line basis or based on the pattern in which economic benefits are consumed, if reliably determinable. The Company reviews finite-lived intangible assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the asset over its estimated fair value in the period at which such a determination is made.

No indicators of impairment were noted during the three months ended March 31, 2023.

Recent Accounting Developments

The Company has evaluated recent accounting pronouncements and believes that none of them will have a material effect on the Company's Consolidated Financial Statements or related disclosures.

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Intangible Assets	3 Months Ended
	Mar. 31, 2023
Goodwill and Intangible Assets Disclosure [Abstract]
Intangible Assets	Intangible AssetsAs of March 31, 2023, the Company's intangible assets consisted of lead enzyme replacement therapy assets acquired with the Callidus Biopharma, Inc. acquisition in 2013, previously accounted for as in-process research and development. In March 2023, as a result of the EC's approval of Pombiliti™, the Company began amortizing the assets over the initial regulatory exclusivity period of 7 years. The Company completed an impairment assessment before changing the classification to definite-lived intangible asset noting no impairment. Amortization expense for the three months ended March 31, 2023 was nominal. Total estimated amortization for the finite-lived intangible assets is estimated to be $2.5 million for the year ended December 31, 2023 and $3.3 million for the next four years thereafter.

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Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

3 Months Ended

Mar. 31, 2023

Cash, Cash Equivalents, and Short-Term Investments [Abstract]

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

As of March 31, 2023, the Company held $160.6 million in cash and cash equivalents and $106.5 million of marketable securities which are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are generally reported within other comprehensive gain (loss) in the Company's Consolidated Statements of Comprehensive Loss. If a decline in the fair value of a marketable security below the Company's cost basis is determined to be other-than-temporary or if an available-for-sale debt security’s fair value is determined to be less than the amortized cost and the Company intends or is more than likely to sell the security before recovery and it is not considered a credit loss such security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in earnings as an impairment charge. If the unrealized loss of an available-for-sale debt security is determined to be a result of credit loss, the Company would recognize an allowance and the corresponding credit loss would be included in earnings.

The Company regularly invests excess operating cash in deposits with major financial institutions and money market funds, as well as fixed income investments which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as, in accordance with Company policy, securities are of high credit rating. Investments that have original maturities greater than three months but less than one year are classified as current.

Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:

	As of March 31, 2023
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	160,602	$	—	$	—	$	160,602
Commercial paper	106,109		14		(17)		106,106
Money market	350		—		—		350
Certificates of deposit	51		—		—		51
	$	267,112	$	14	$	(17)	$	267,109
Included in cash and cash equivalents	$	160,602	$	—	$	—	$	160,602
Included in marketable securities	106,510		14		(17)		106,507
Total cash, cash equivalents, and marketable securities	$	267,112	$	14	$	(17)	$	267,109

	As of December 31, 2022
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Commercial paper	144,299		82		—		144,381
Money market	350		—		—		350
Certificate of deposit	51		—		—		51
	$	293,513	$	82	$	—	$	293,595
Included in cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Included in marketable securities	144,700		82		—		144,782
Total cash, cash equivalents, and marketable securities	$	293,513	$	82	$	—	$	293,595

For both the three months ended March 31, 2023 and the fiscal year ended December 31, 2022, there were no realized gains or losses. The cost of securities sold is based on the specific identification method.

Unrealized loss positions in the marketable securities as of March 31, 2023 reflect temporary impairments and are not a result of credit loss. Additionally, as these positions have been in a loss position for less than twelve months and the Company does not intend to sell these securities before recovery, the losses are recognized in other comprehensive gain (loss). The fair value of these marketable securities in unrealized loss positions was $37.6 million as of March 31, 2023. The Company had no securities in an unrealized loss position as of December 31, 2022.

The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Company's Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Company's Consolidated Statements of Cash Flows.

	As of March 31,
(in thousands)	2023		2022
Cash and cash equivalents	$	160,602	$	233,317
Restricted cash	4,381		4,506
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$	164,983	$	237,823

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Inventories

3 Months Ended

Mar. 31, 2023

Inventory Disclosure [Abstract]

Inventories

Inventories

Inventories as of March 31, 2023 and December 31, 2022 consisted of the following:

(in thousands)	March 31, 2023		December 31, 2022
Raw materials	$	13,597	$	10,054
Work-in-process	5,823		9,615
Finished goods	7,584		4,147
Total inventories	$	27,004	$	23,816

The Company's reserve for inventory was $0.4 million as of both March 31, 2023 and December 31, 2022.

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Debt

3 Months Ended

Mar. 31, 2023

Debt Disclosure [Abstract]

Debt

Debt

The Company's debt consists of the following:

(in thousands)	March 31, 2023		December 31, 2022
Senior Secured Term Loan due 2026:
Principal	$	400,000	$	400,000
Less: debt discount (1)	(4,190)		(4,571)
Less: deferred financing (1)	(3,152)		(3,439)
Net carrying value of Long-term debt	$	392,658	$	391,990

______________________________

(1) Included in the Company's Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan using the effective interest rate method.

Interest Expense

The following table sets forth interest expense recognized related to the Company's debt for the three months ended March 31, 2023 and 2022, respectively:

	Three Months Ended March 31,
(in thousands)	2023		2022
Contractual interest expense	$	11,230	$	7,500
Amortization of debt discount	$	381	$	364
Amortization of deferred financing	$	286	$	273

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Stockholders' Equity	3 Months Ended
	Mar. 31, 2023
Equity [Abstract]
Stockholders' Equity	Stockholder's EquityDuring the three months ended March 31, 2023, the Company issued and sold an aggregate of 195,229 shares through its at-the-market equity program ("ATM program") at a weighted-average public offering price of $12.71 per share and received aggregate net proceeds of $2.4 million. As of March 31, 2023, an aggregate of $247.5 million worth of shares remain available to be issued and sold under the ATM program.

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Stock-Based Compensation

3 Months Ended

Mar. 31, 2023

Share-Based Payment Arrangement [Abstract]

Stock-Based Compensation

Stock-Based Compensation

The Company's Amended and Restated 2007 Equity Incentive Plan (the "Plan") provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Company's Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company's business. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option.

Stock Option Grants

The fair value of the stock options granted is estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:

	Three Months Ended March 31,
	2023			2022
Expected stock price volatility	59.3		%	62.3		%
Risk free interest rate	3.9		%	1.5		%
Expected life of options (years)	5.5			5.3
Expected annual dividend per share	$	—		$	—

 A summary of the Company's stock options for the three months ended March 31, 2023 were as follows:

	Number of Shares	Weighted Average Exercise  Price		Weighted Average Remaining Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Options outstanding, December 31, 2022	19,064	$	11.31
Granted	4,854	$	11.94
Exercised	(384)	$	6.91
Forfeited	(81)	$	10.97
Expired	(16)	$	15.51
Options outstanding, March 31, 2023	23,437	$	11.51	7.0	$	22.8
Vested and unvested expected to vest, March 31, 2023	21,395	$	11.45	6.9	$	22.1
Exercisable at March 31, 2023	12,395	$	10.94	5.3	$	19.4

As of March 31, 2023, the total unrecognized compensation cost related to non-vested stock options granted was $47.1 million and is expected to be recognized over a weighted average period of three years.

Restricted Stock Units and Performance-Based Restricted Stock Units (collectively "RSUs")

RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee's continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse. A summary of non-vested RSU activity under the Plan for the three months ended March 31, 2023 is as follows:

	Number of Shares	Weighted Average Grant Date Fair Value		Weighted  Average Remaining  Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Non-vested units as of December 31, 2022	9,717	$	13.07
Granted	2,762	$	11.94
Vested	(2,532)	$	12.20
Forfeited	(517)	$	9.68
Non-vested units as of March 31, 2023	9,430	$	13.16	2.4	$	104.6

As of March 31, 2023, there was $63.8 million of total unrecognized compensation cost related to unvested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of two years.

Compensation Expense Related to Equity Awards

The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards:

	Three Months Ended March 31,
(in thousands)	2023		2022
Research and development expense	$	8,490	$	9,365
Selling, general, and administrative expense	26,404		21,286
Total equity compensation expense	$	34,894	$	30,651

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Assets and Liabilities Measured at Fair Value

3 Months Ended

Mar. 31, 2023

Fair Value Disclosures [Abstract]

Assets and Liabilities Measured at Fair Value

Assets and Liabilities Measured at Fair Value

The Company's financial assets and liabilities are measured at fair value and classified within the fair value hierarchy, which is defined as follows:

Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date.

Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly.

Level 3 — Inputs that are unobservable for the asset or liability.

A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of March 31, 2023 are identified in the following tables:

(in thousands)	Level 2		Total
Assets:
Commercial paper	$	106,106	$	106,106
Money market	6,631		6,631
	$	112,737	$	112,737

(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	12,668	$	12,668
Deferred compensation plan liability	6,281		—		6,281
	$	6,281	$	12,668	$	18,949

A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2022 are identified in the following tables:

(in thousands)	Level 2		Total
Assets:
Commercial paper	$	144,381	$	144,381
Money market	5,808		5,808
	$	150,189	$	150,189

(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	21,417	$	21,417
Deferred compensation plan liability	5,458		—		5,458
	$	5,458	$	21,417	$	26,875

The Company's Senior Secured Term Loan due 2026 falls into the Level 2 category within the fair value level hierarchy and the fair value was determined using quoted prices for similar liabilities in active markets, as well as inputs that are observable for the liability (other than quoted prices), such as interest rates that are observable at commonly quoted intervals. The carrying value of the Senior Secured Term Loan due 2026 approximates the fair value.

The Company did not have any Level 3 assets as of March 31, 2023 or December 31, 2022.

Cash, Money Market Funds, and Marketable Securities

The Company classifies its cash within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in an active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available-for-sale and classifies these assets and the money market funds within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities.

Contingent Consideration Payable

The contingent consideration payable resulted from the acquisition of Callidus Biopharma, Inc. ("Callidus") in November 2013. The most recent valuation was determined using a probability weighted discounted cash flow valuation approach. Gains and losses are included in the Company's Consolidated Statements of Operations.

The contingent consideration payable for Callidus has been classified as a Level 3 recurring liability as its valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions

were used for the various inputs to the valuation approach, the estimated fair value could be significantly higher or lower than the fair value the Company determined.

The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe disease program:

Contingent Consideration Liability	Fair Value as of March 31, 2023		Valuation Technique	Unobservable Input	Range
	(in thousands)
				Discount rate	11.2%
Clinical and regulatory milestones	$	12,668	Probability weighted discounted cash flow	Probability of achievement of milestones	88%
				Projected year of payments	2023

Contingent consideration liabilities are remeasured to fair value each reporting period using discount rates, probabilities of payment, and projected payment dates. Projected contingent payment amounts related to clinical and regulatory based milestones are discounted back to the current period using a discounted cash flow model. Increases in discount rates and the time to payment may result in lower fair value measurements. Increases or decreases in any of those inputs together, or in isolation, may result in a significantly lower or higher fair value measurement. There is no assurance that any of the conditions for the milestone payments will be met.

The Company reached a regulatory milestone in March 2023 associated with the EC granting approval for Pombiliti™, related to the contingent consideration of Callidus for the ATB200 Pompe disease program. The satisfaction of this milestone resulted in the milestone payment of $9.0 million being due, which is payable in cash and recorded as a component of accounts payable on the Company's Consolidated Balance Sheets as of March 31, 2023.

The following table shows the change in the balance of contingent consideration payable for the three months ended March 31, 2023 and 2022, respectively:

	Three Months Ended March 31,
(in thousands)	2023		2022
Balance, beginning of the period	$	21,417	$	20,339
Changes in fair value during the period, included in the Consolidated Statements of Operations	251		(1,188)
Milestone payment payable in cash	(9,000)		—
Balance, end of the period (1)	$	12,668	$	19,151

______________________________

(1) As certain milestones are expected to be reached within the next twelve months, the March 31, 2023 balance was recorded as a current liability in the Company's Consolidated Balance Sheets.

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Basic and Diluted Net Loss per Common Share

3 Months Ended

Mar. 31, 2023

Earnings Per Share [Abstract]

Basic and Diluted Net Loss per Common Share

Basic and Diluted Net Loss per Common Share

The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:

	Three Months Ended March 31,
(in thousands, except per share amounts)	2023		2022
Numerator:
Net loss attributable to common stockholders	$	(52,932)	$	(85,260)
Denominator:
Weighted average common shares outstanding — basic and diluted	291,336,750		288,481,741

Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options and unvested RSUs. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect. Weighted average common shares outstanding includes outstanding pre-funded warrants with an exercise price of $0.01.

The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:

	As of March 31,
(in thousands)	2023	2022
Options to purchase common stock	23,437	19,304
Unvested restricted stock units	9,430	10,351
Total number of potentially issuable shares	32,867	29,655

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Summary of Significant Accounting Policies (Policies)

3 Months Ended

Mar. 31, 2023

Accounting Policies [Abstract]

Basis of Presentation

Basis of Presentation

The Company has prepared the accompanying unaudited Consolidated Financial Statements in accordance with the U.S. generally accepted accounting principles ("U.S. GAAP") for interim financial information and with the instructions to Form 10-Q and Article 10-01 of Regulation S-X. Accordingly, they do not include all of the information and disclosures required by U.S. GAAP for complete financial statements. In the opinion of management, the accompanying unaudited Consolidated Financial Statements reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company's interim financial information.

The accompanying unaudited Consolidated Financial Statements and related notes should be read in conjunction with the Company's financial statements and related notes as contained in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2022. For a complete description of the Company's accounting policies, please refer to the Annual Report on Form 10-K for the fiscal year ended December 31, 2022.

Consolidation

Consolidation

The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.

Foreign Currency Transactions

Foreign Currency Transactions

The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity.

Use of Estimates

Use of Estimates

The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.

Additionally, the Company assessed the impact the COVID-19 pandemic had on its operations and financial results as of March 31, 2023 and through the issuance of these financial statements. The Company’s analysis was informed by the facts and circumstances as they were known to the Company. This assessment considered the impact COVID-19 may have on financial estimates and assumptions that affect the reported amounts of assets and liabilities and revenue and expenses.

Cash, Cash Equivalents, Marketable Securities and Restricted Cash

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash

The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive loss in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs.

Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included in other current assets and other non-current assets on the Company's Consolidated Balance Sheets.

Concentration of Credit Risk

Concentration of Credit Risk

The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities.

The Company is subject to credit risk from its accounts receivable related to its product sales of Galafold®. The Company's accounts receivable at March 31, 2023 have arisen from product sales primarily in Europe, the U.S., and Japan. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis.

Revenue Recognition

Revenue Recognition

The Company's net product sales consist primarily of sales of Galafold® for the treatment of Fabry disease. Galafold® sales for the three months ended March 31, 2023 and 2022 were $86.1 million and $78.7 million, respectively. The Company has recorded revenue on sales where Galafold® is available either on a commercial basis or through a reimbursed early access program. Orders for Galafold® are generally received from distributors and pharmacies, with the ultimate payor often a government authority.

The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of Galafold®. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which are third party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale of Galafold® is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.

Inventories and Cost of Goods Sold

Inventories and Cost of Goods Sold

Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations.

Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable.

Intangible Assets and Goodwill

Intangible Assets and Goodwill

The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Goodwill is assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill impairment test is conducted. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference.

Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and, if applicable, impairment charges. Amortization of finite-lived intangible assets is recorded over the assets’ estimated useful lives on a straight-line basis or based on the pattern in which economic benefits are consumed, if reliably determinable. The Company reviews finite-lived intangible assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the asset over its estimated fair value in the period at which such a determination is made.

No indicators of impairment were noted during the three months ended March 31, 2023.

Recent Accounting Developments

Recent Accounting Developments

The Company has evaluated recent accounting pronouncements and believes that none of them will have a material effect on the Company's Consolidated Financial Statements or related disclosures.

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Summary of Significant Accounting Policies (Tables)

3 Months Ended

Mar. 31, 2023

Accounting Policies [Abstract]

Schedule of Revenue Disaggregated by Geographical Area

The following table summarizes the Company's net product sales disaggregated by geographic area:

	Three Months Ended March 31,
(in thousands)	2023		2022
U.S.	$	28,831	$	24,178
Ex-U.S.	57,439		54,537
Total net product sales	$	86,270	$	78,715

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Cash, Cash Equivalents, Marketable Securities, and Restricted Cash (Tables)

3 Months Ended

Mar. 31, 2023

Cash, Cash Equivalents, and Short-Term Investments [Abstract]

Schedule of Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities are classified as current unless mentioned otherwise below and consisted of the following:

	As of March 31, 2023
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	160,602	$	—	$	—	$	160,602
Commercial paper	106,109		14		(17)		106,106
Money market	350		—		—		350
Certificates of deposit	51		—		—		51
	$	267,112	$	14	$	(17)	$	267,109
Included in cash and cash equivalents	$	160,602	$	—	$	—	$	160,602
Included in marketable securities	106,510		14		(17)		106,507
Total cash, cash equivalents, and marketable securities	$	267,112	$	14	$	(17)	$	267,109

	As of December 31, 2022
(in thousands)	Cost		Gross Unrealized Gain		Gross Unrealized Loss		Fair Value
Cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Commercial paper	144,299		82		—		144,381
Money market	350		—		—		350
Certificate of deposit	51		—		—		51
	$	293,513	$	82	$	—	$	293,595
Included in cash and cash equivalents	$	148,813	$	—	$	—	$	148,813
Included in marketable securities	144,700		82		—		144,782
Total cash, cash equivalents, and marketable securities	$	293,513	$	82	$	—	$	293,595

Schedule of Cash, Cash Equivalents, and Restricted Cash

The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Company's Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Company's Consolidated Statements of Cash Flows.

	As of March 31,
(in thousands)	2023		2022
Cash and cash equivalents	$	160,602	$	233,317
Restricted cash	4,381		4,506
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$	164,983	$	237,823

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Inventories (Tables)

3 Months Ended

Mar. 31, 2023

Inventory Disclosure [Abstract]

Schedule of Inventories for the Period

Inventories as of March 31, 2023 and December 31, 2022 consisted of the following:

(in thousands)	March 31, 2023		December 31, 2022
Raw materials	$	13,597	$	10,054
Work-in-process	5,823		9,615
Finished goods	7,584		4,147
Total inventories	$	27,004	$	23,816

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Debt (Tables)

3 Months Ended

Mar. 31, 2023

Debt Disclosure [Abstract]

Schedule of Liability Components of Long-Term Debt

The Company's debt consists of the following:

(in thousands)	March 31, 2023		December 31, 2022
Senior Secured Term Loan due 2026:
Principal	$	400,000	$	400,000
Less: debt discount (1)	(4,190)		(4,571)
Less: deferred financing (1)	(3,152)		(3,439)
Net carrying value of Long-term debt	$	392,658	$	391,990

______________________________

(1) Included in the Company's Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the Senior Secured Term Loan using the effective interest rate method.

Schedule of Components of Total Interest Expense

The following table sets forth interest expense recognized related to the Company's debt for the three months ended March 31, 2023 and 2022, respectively:

	Three Months Ended March 31,
(in thousands)	2023		2022
Contractual interest expense	$	11,230	$	7,500
Amortization of debt discount	$	381	$	364
Amortization of deferred financing	$	286	$	273

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Stock-Based Compensation (Tables)

3 Months Ended

Mar. 31, 2023

Share-Based Payment Arrangement [Abstract]

Schedule of Fair Value of Options

The fair value of the stock options granted is estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions:

	Three Months Ended March 31,
	2023			2022
Expected stock price volatility	59.3		%	62.3		%
Risk free interest rate	3.9		%	1.5		%
Expected life of options (years)	5.5			5.3
Expected annual dividend per share	$	—		$	—

Schedule of Stock Options Activity

A summary of the Company's stock options for the three months ended March 31, 2023 were as follows:

	Number of Shares	Weighted Average Exercise  Price		Weighted Average Remaining Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Options outstanding, December 31, 2022	19,064	$	11.31
Granted	4,854	$	11.94
Exercised	(384)	$	6.91
Forfeited	(81)	$	10.97
Expired	(16)	$	15.51
Options outstanding, March 31, 2023	23,437	$	11.51	7.0	$	22.8
Vested and unvested expected to vest, March 31, 2023	21,395	$	11.45	6.9	$	22.1
Exercisable at March 31, 2023	12,395	$	10.94	5.3	$	19.4

Schedule of Non-Vested RSU Activity under the Plan

A summary of non-vested RSU activity under the Plan for the three months ended March 31, 2023 is as follows:

	Number of Shares	Weighted Average Grant Date Fair Value		Weighted  Average Remaining  Years	Aggregate Intrinsic Value
	(in thousands)				(in millions)
Non-vested units as of December 31, 2022	9,717	$	13.07
Granted	2,762	$	11.94
Vested	(2,532)	$	12.20
Forfeited	(517)	$	9.68
Non-vested units as of March 31, 2023	9,430	$	13.16	2.4	$	104.6

Schedule of Equity Compensation Expenses

The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards:

	Three Months Ended March 31,
(in thousands)	2023		2022
Research and development expense	$	8,490	$	9,365
Selling, general, and administrative expense	26,404		21,286
Total equity compensation expense	$	34,894	$	30,651

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Assets and Liabilities Measured at Fair Value (Tables)

3 Months Ended

Mar. 31, 2023

Fair Value Disclosures [Abstract]

Summary of Assets and Liabilities Subject to Fair Value Measurements

A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of March 31, 2023 are identified in the following tables:

(in thousands)	Level 2		Total
Assets:
Commercial paper	$	106,106	$	106,106
Money market	6,631		6,631
	$	112,737	$	112,737

(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	12,668	$	12,668
Deferred compensation plan liability	6,281		—		6,281
	$	6,281	$	12,668	$	18,949

A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2022 are identified in the following tables:

(in thousands)	Level 2		Total
Assets:
Commercial paper	$	144,381	$	144,381
Money market	5,808		5,808
	$	150,189	$	150,189

(in thousands)	Level 2		Level 3		Total
Liabilities:
Contingent consideration payable	$	—	$	21,417	$	21,417
Deferred compensation plan liability	5,458		—		5,458
	$	5,458	$	21,417	$	26,875

Schedule of Significant Unobservable Inputs Used in the Valuation of the Contingent Consideration Payable

The following significant unobservable inputs were used in the valuation of the contingent consideration payable of Callidus for the ATB200 Pompe disease program:

Contingent Consideration Liability	Fair Value as of March 31, 2023		Valuation Technique	Unobservable Input	Range
	(in thousands)
				Discount rate	11.2%
Clinical and regulatory milestones	$	12,668	Probability weighted discounted cash flow	Probability of achievement of milestones	88%
				Projected year of payments	2023

Schedule of Changes in Continent Consideration Payable

The following table shows the change in the balance of contingent consideration payable for the three months ended March 31, 2023 and 2022, respectively:

	Three Months Ended March 31,
(in thousands)	2023		2022
Balance, beginning of the period	$	21,417	$	20,339
Changes in fair value during the period, included in the Consolidated Statements of Operations	251		(1,188)
Milestone payment payable in cash	(9,000)		—
Balance, end of the period (1)	$	12,668	$	19,151

______________________________

(1) As certain milestones are expected to be reached within the next twelve months, the March 31, 2023 balance was recorded as a current liability in the Company's Consolidated Balance Sheets.

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Basic and Diluted Net Loss per Common Share (Tables)

3 Months Ended

Mar. 31, 2023

Earnings Per Share [Abstract]

Schedule of Reconciliation of the Numerator and Denominator Used in Computing Basic and Diluted Net Loss per Common Share

The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share:

	Three Months Ended March 31,
(in thousands, except per share amounts)	2023		2022
Numerator:
Net loss attributable to common stockholders	$	(52,932)	$	(85,260)
Denominator:
Weighted average common shares outstanding — basic and diluted	291,336,750		288,481,741

Schedule of Potential Shares of Common Stock that were Excluded from the Computation as they were Anti-Dilutive Using the Treasury Stock Method

The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method:

	As of March 31,
(in thousands)	2023	2022
Options to purchase common stock	23,437	19,304
Unvested restricted stock units	9,430	10,351
Total number of potentially issuable shares	32,867	29,655

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Description of Business (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Accumulated deficit	$ (2,585,422)	$ (2,532,490)

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Summary of Significant Accounting Policies - Concentration of Credit Risk and Revenue Recognition (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Concentration Risk [Line Items]
Allowance for doubtful accounts receivable	$ 100
Net product sales	86,270	$ 78,715
Galafold
Concentration Risk [Line Items]
Net product sales	$ 86,100	$ 78,700

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Summary of Significant Accounting Policies - Revenue by Geographical Area (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Net product sales	$ 86,270	$ 78,715
U.S.
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Net product sales	28,831	24,178
Ex-U.S.
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Net product sales	$ 57,439	$ 54,537

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Intangible Assets - Narrative (Details) $ in Millions	3 Months Ended
	Mar. 31, 2023 USD ($)
Goodwill and Intangible Assets Disclosure [Abstract]
Initial regulatory exclusivity period	7 years
Remainder of fiscal year 2023	$ 2.5
2024	$ 3.3

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Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Narrative (Details) - USD ($)	3 Months Ended	12 Months Ended
	Mar. 31, 2023	Dec. 31, 2022	Mar. 31, 2022
Cash, Cash Equivalents, and Short-Term Investments [Abstract]
Cash and cash equivalents	$ 160,602,000	$ 148,813,000	$ 233,317,000
Available-for-sale debt securities	106,507,000	144,782,000
Debt securities, realized gain (loss)	0	0
Fair value of available-for-sale debt securities in unrealized loss positions	$ 37,600,000	$ 0

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Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Components (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022	Mar. 31, 2022
Cash, Money Market Funds, and Marketable Securities
Cash and cash equivalents	$ 160,602	$ 148,813	$ 233,317
Fair value, cash balances	160,602	148,813
Cost, available-for-sale securities	106,510	144,700
Gross unrealized gain, available-for-sale securities	14	82
Gross unrealized loss, available-for-sale securities	(17)	0
Fair value, available-for-sale debt securities	106,507	144,782
Cost, cash balances and available-for-sale securities	267,112	293,513
Gross unrealized gain, cash balances and available-for-sale securities	14	82
Gross unrealized loss, cash balances and available-for-sale securities	(17)	0
Fair value, cash balances and available-for-sale securities	267,109	293,595
Commercial paper
Cash, Money Market Funds, and Marketable Securities
Cost, available-for-sale securities	106,109	144,299
Gross unrealized gain, available-for-sale securities	14	82
Gross unrealized loss, available-for-sale securities	(17)	0
Fair value, available-for-sale debt securities	106,106	144,381
Money market
Cash, Money Market Funds, and Marketable Securities
Cost, available-for-sale securities	350	350
Fair value, available-for-sale debt securities	350	350
Certificates of deposit
Cash, Money Market Funds, and Marketable Securities
Cost, available-for-sale securities	51	51
Fair value, available-for-sale debt securities	$ 51	$ 51

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Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Cash, Cash Equivalents, And Restricted Cash (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022	Mar. 31, 2022	Dec. 31, 2021
Cash, Cash Equivalents, and Short-Term Investments [Abstract]
Cash and cash equivalents	$ 160,602	$ 148,813	$ 233,317
Restricted cash	4,381		4,506
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$ 164,983	$ 153,115	$ 237,823	$ 249,456

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Inventories (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Inventory Disclosure [Abstract]
Raw materials	$ 13,597	$ 10,054
Work-in-process	5,823	9,615
Finished goods	7,584	4,147
Total inventories	$ 27,004	$ 23,816

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Inventories - Narrative (Details) - USD ($) $ in Millions	Mar. 31, 2023	Dec. 31, 2022
Inventory Disclosure [Abstract]
Reserve for inventory	$ 0.4	$ 0.4

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Debt - Summary of Long Term Debt (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Debt Instrument [Line Items]
Net carrying value of Long-term debt	$ 392,658	$ 391,990
Senior Loans | Senior Secured Term Loan due 2026
Debt Instrument [Line Items]
Principal	400,000	400,000
Less: debt discount	(4,190)	(4,571)
Less: deferred financing	$ (3,152)	$ (3,439)

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Debt - Interest Expense (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Debt Disclosure [Abstract]
Contractual interest expense	$ 11,230	$ 7,500
Amortization of debt discount	381	364
Amortization of deferred financing	$ 286	$ 273

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Stockholders' Equity - Common Stock and Warrants (Details) - ATM $ / shares in Units, $ in Millions	3 Months Ended
	Mar. 31, 2023 USD ($) $ / shares shares
Subsidiary, Sale of Stock [Line Items]
Number of shares issued, private placement (in shares)	195,229
Offering price per share (in dollars per share) | $ / shares	$ 12.71
Aggregate net proceeds from stock offering | $	$ 2.4
Common Stock, Capital Shares Reserved for Future Issuance	247,500,000

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Stock-Based Compensation - Weighted-average Assumptions (Details) - Options to purchase common stock - $ / shares	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expected stock price volatility	59.30%	62.30%
Risk free interest rate	3.90%	1.50%
Expected life of options (years)	5 years 6 months	5 years 3 months 18 days
Expected annual dividend per share (in dollars per share)	$ 0	$ 0

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Stock-Based Compensation - Stock Option Activity (Details) $ / shares in Units, shares in Thousands, $ in Millions	3 Months Ended
	Mar. 31, 2023 USD ($) $ / shares shares
Number of Shares
Balance at the beginning of the period (in shares) | shares	19,064
Options, granted (in shares) | shares	4,854
Options, exercised (in shares) | shares	(384)
Options, forfeited (in shares) | shares	(81)
Options, expired (in shares) | shares	(16)
Balance at the end of the period (in shares) | shares	23,437
Vested and unvested expected to vest as of the end of the period (in shares) | shares	21,395
Exercisable at the end of the period (in shares) | shares	12,395
Weighted Average Exercise  Price
Balance at the beginning of the period (in dollars per share) | $ / shares	$ 11.31
Options, granted (in dollars per share) | $ / shares	11.94
Options, exercised (in dollars per share) | $ / shares	6.91
Options, forfeited (in dollars per share) | $ / shares	10.97
Options, expired (in dollars per share) | $ / shares	15.51
Balance at the end of the period (in dollars per share) | $ / shares	11.51
Vested and unvested expected to vest as of the end of the period (in dollars per share) | $ / shares	11.45
Exercisable at the end of the period (in dollars per share) | $ / shares	$ 10.94
Additional Disclosures
Balance at the end of the period	7 years
Vested and unvested expected to vest at the end of the period	6 years 10 months 24 days
Exercisable at the end of the period	5 years 3 months 18 days
Aggregate intrinsic value, options outstanding | $	$ 22.8
Aggregate intrinsic value, vested and unvested expected to vest | $	22.1
Aggregate intrinsic value of options exercisable | $	$ 19.4

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Stock-Based Compensation - Narrative (Details) $ in Millions	3 Months Ended
	Mar. 31, 2023 USD ($)
Options to purchase common stock
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total unrecognized compensation costs	$ 47.1
Unrecognized compensation costs, period for recognition (in years)	3 years
Unvested restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total unrecognized compensation costs	$ 63.8
Unrecognized compensation costs, period for recognition (in years)	2 years

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Stock-Based Compensation - RSUs and PBRSUs Summary (Details) - Unvested restricted stock units $ / shares in Units, shares in Thousands, $ in Millions	3 Months Ended
	Mar. 31, 2023 USD ($) $ / shares shares
Number of Shares
Non-vested units as of the beginning of the period (in shares) | shares	9,717
Granted (in shares) | shares	2,762
Vested (in shares) | shares	(2,532)
Forfeited (in shares) | shares	(517)
Non-vested units as of the end of the period (in shares) | shares	9,430
Weighted Average Grant Date Fair Value
Non-vested units as of the beginning of the period (in dollars per share) | $ / shares	$ 13.07
Granted (in dollars per share) | $ / shares	11.94
Vested (in dollars per share) | $ / shares	12.20
Forfeited (in dollars per share) | $ / shares	9.68
Non-vested units as of the end of the period (in dollars per share) | $ / shares	$ 13.16
Share-based Compensation Arrangement by Share-based Payment Award, Equity Instruments Other than Options, Additional Disclosures [Abstract]
Non-vested units, weighted average remaining years	2 years 4 months 24 days
Non-vested units, aggregate intrinsic value | $	$ 104.6

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Stock-Based Compensation - Expense Summary (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total equity compensation expense	$ 34,894	$ 30,651
Research and development expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total equity compensation expense	8,490	9,365
Selling, general, and administrative expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total equity compensation expense	$ 26,404	$ 21,286

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Assets and Liabilities Measured at Fair Value - Summary of Fair Value of Assets and Liabilities (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Assets:
Fair value of assets	$ 112,737	$ 150,189
Liabilities:
Contingent consideration payable	12,668	21,417
Deferred compensation plan liability	6,281	5,458
Fair value of liabilities	18,949	26,875
Commercial paper
Assets:
Fair value of assets	106,106	144,381
Money market
Assets:
Fair value of assets	6,631	5,808
Level 2
Assets:
Fair value of assets	112,737	150,189
Liabilities:
Contingent consideration payable	0	0
Deferred compensation plan liability	6,281	5,458
Fair value of liabilities	6,281	5,458
Level 2 | Commercial paper
Assets:
Fair value of assets	106,106
Level 2 | Money market
Assets:
Fair value of assets	6,631	5,808
Level 3
Liabilities:
Contingent consideration payable	12,668	21,417
Deferred compensation plan liability	0	0
Fair value of liabilities	$ 12,668	$ 21,417

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Assets and Liabilities Measured at Fair Value - Significant Unobservable Inputs (Details) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Contingent consideration payable	$ 12,668	$ 21,417
Discount rate
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Contingent consideration payable	12,668	$ 21,417
Callidus Biopharma Inc | Clinical and regulatory milestones | ATB200 Pompe Program
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Contingent consideration payable	$ 12,668
Callidus Biopharma Inc | Clinical and regulatory milestones | Probability of achievement of milestones | ATB200 Pompe Program | Minimum
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Contingent consideration, measurement input	88.00%
Callidus Biopharma Inc | Clinical and regulatory milestones | Discount rate | Discount rate | ATB200 Pompe Program | Probability weighted discounted cash flow
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Contingent consideration, measurement input	11.20%

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Assets and Liabilities Measured at Fair Value - Level 3 Roll Forward (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward]
Balance, beginning of the period	$ 21,417	$ 20,339
Changes in fair value during the period, included in the Consolidated Statements of Operations	251	(1,188)
Fair Value, Measurement with Unobservable Inputs Reconciliation, Recurring Basis, Liability, Settlements	9,000	0
Balance, end of the period	$ 12,668	$ 19,151

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Basic and Diluted Net Loss per Common Share (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Numerator:
Net loss attributable to common stockholders	$ (52,932)	$ (85,260)
Denominator:
Weighted average common shares outstanding - basic (in shares)	291,336,750	288,481,741
Weighted average common shares outstanding - diluted (in shares)	291,336,750	288,481,741
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	32,867,000	29,655,000
Options to purchase common stock
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	23,437,000	19,304,000
Unvested restricted stock units
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	9,430,000	10,351,000

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Basic and Diluted Net Loss per Common Share - Narrative (Details)	Mar. 31, 2023 $ / shares
Private Placement
Antidilutive securities
Warrants exercise price (in dollars per share)	$ 0.01