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SEGMENT INFORMATION
 6 Months Ended
Jun. 30, 2025
Segment Reporting [Abstract]  
SEGMENT INFORMATION SEGMENT INFORMATION
The Company operates and is managed as one business segment which derives revenue from activities related to the development and commercialization of innovative therapies for people with serious and life-threatening rare diseases and medical conditions.
The Company’s commercial organization is responsible for marketing our approved products worldwide. The Company’s research and development (R&D) organization is responsible for research and discovery of new product candidates and supporting the development and registration efforts for potential new products. The Company’s technical operations group is responsible for the development of manufacturing processes, supplying clinical drug product, and the manufacturing and distribution of the Company’s commercial products. The Company is also supported by corporate staff functions.
The Company’s Chief Executive Officer, as the Chief Operating Decision Maker (CODM), manages and allocates resources to the operations of the total company by assessing the overall level of resources available and how to best allocate them to support the Company’s long-term company-wide strategic goals. In making this decision, the CODM uses consolidated financial information for the purposes of evaluating performance, allocating resources, setting incentive compensation targets and planning and forecasting for future periods.
The key measure of segment profit or loss used by the CODM to allocate resources and assess the Company's performance is its Consolidated Net Income, as reported on the Condensed Consolidated Statements of Comprehensive Income. The CODM's analysis includes a comparison to budgeted results. Segment assets provided to the CODM are consistent with those reported on the Condensed Consolidated Balance Sheets with particular emphasis on the Company's available liquidity including cash, cash equivalents, investments, accounts receivable and inventory.
The following table includes information about segment revenue, significant segment expenses, and segment measure of profitability:
Three Months Ended
June 30,		  Six Months Ended
June 30,
20252024  20252024
Total revenues$825,410 $712,029 $1,570,555 $1,360,862 
Less:
Cost of sales150,090 130,459 301,648 255,639 
R&D expenses
Research and early pipeline95,968 106,063 186,438 226,455 
Later-stage clinical programs13,133 7,714 27,424 7,714 
Marketed products52,207 70,010 106,177 154,605 
SG&A expenses
S&M expenses122,100 122,798 224,631 242,158 
G&A expenses110,179 140,234 213,764 246,780 
Other segment expense, net (1)
41,201 27,577 84,255 31,675 
Net income$240,532 $107,174 $426,218 $195,836 
(1)Other segment expense, net during the three months ended June 30, 2025 and 2024 include intangible asset amortization, interest income and expense, other income (expense) and income tax expense.
The following table presents Total Revenues and disaggregates Net Product Revenues by product.
Three Months Ended
June 30,		Six Months Ended
June 30,
2025202420252024
VOXZOGO$221,391 $183,923 $435,145 $336,812 
VIMIZIM215,424 178,016 403,770 370,536 
NAGLAZYME128,867 132,049 243,157 237,678 
PALYNZIQ105,938 88,291 199,207 164,000 
ALDURAZYME56,437 38,558 105,414 73,820 
BRINEURA48,692 45,309 89,054 84,356 
KUVAN27,034 28,551 52,170 64,461 
ROCTAVIAN9,199 7,432 19,709 8,281 
Total net product revenues812,982 702,129 1,547,626 1,339,944 
Royalty and other revenues12,428 9,900 22,929 20,918 
Total revenues$825,410 $712,029 $1,570,555 $1,360,862 
The Company considers there to be revenue concentration risks for regions where Net Product Revenues exceed 10% of consolidated Net Product Revenues. The concentration of the Company’s Net Product Revenues within the regions below may have a material adverse effect on the Company’s revenues and results of operations if sales in the respective regions experience difficulties. The table below disaggregates total Net Product Revenues by geographic region, which is based on patient location for the Company's commercial products sold directly by the Company, except for ALDURAZYME, which is distributed, marketed and sold exclusively by Sanofi worldwide.
Three Months Ended
June 30,		Six Months Ended
June 30,
2025202420252024
United States$272,554 $227,449 $514,248 $420,448 
Europe243,138 220,990 472,716 416,738 
Latin America98,118 84,418 186,400 163,012 
Rest of world142,735 130,714 268,848 265,926 
Total net product revenues marketed by the Company$756,545 $663,571 $1,442,212 $1,266,124 
ALDURAZYME net product revenues marketed by Sanofi56,437 38,558 105,414 73,820 
Total net product revenues$812,982 $702,129 $1,547,626 $1,339,944 
The following table illustrates the percentage of the Company’s total Net Product Revenues attributed to the Company’s largest customers for the periods presented.
Three Months Ended
June 30,		Six Months Ended
June 30,
2025202420252024
Customer A15 %14 %15 %14 %
Customer B12 11 12 11 
Customer C11 10 11 9 
Total38 %35 %38 %34 %
Concentration Information
On a consolidated basis, three customers accounted for 19%, 15% and 11% of the Company’s June 30, 2025 accounts receivable balance, respectively, compared to December 31, 2024, when two customers accounted for 20% and 11% of the accounts receivable balance, respectively. As of June 30, 2025, and December 31, 2024, the accounts receivable balance for Sanofi included $128.4 million and $96.8 million, respectively, of unbilled accounts receivable, which becomes payable to the Company when the product is sold through by Sanofi. The Company does not require collateral from its customers, but does perform periodic credit evaluations of its customers’ financial condition and requires prepayments in certain circumstances.
The Company is mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, impact of new or increased tariffs and escalating trade tensions, and supply chain disruptions, could affect the Company’s ability to achieve its goals. In addition, the Company sells its products in countries that face economic volatility and weakness. Although the Company has historically collected receivables from customers in such countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to delay payment or be unable to pay for the Company’s products. The Company believes that the allowances for doubtful accounts related to these countries, if any, are adequate as of June 30, 2025 based on its analysis of the specific business circumstances and expectations of collection for each of the underlying accounts in these countries. The Company will continue to monitor these conditions and will attempt to adjust its business processes, as appropriate, to mitigate macroeconomic risks to its business.