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SEGMENT INFORMATION
 9 Months Ended
Sep. 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 its 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
September 30,		Nine Months Ended
September 30,
2025202420252024
Total revenues$776,133 $745,740 $2,346,688 $2,106,602 
Less:
Cost of sales140,085 188,457 441,733 444,096 
R&D expenses
Research and early pipeline93,720 104,915 280,158 331,372 
Later-stage clinical programs251,650 10,078 279,074 17,792 
Marketed products64,108 69,908 170,285 224,511 
SG&A expenses
S&M expenses131,327 108,945 355,958 351,103 
G&A expenses137,088 144,535 350,852 391,315 
Other segment expense (income), net (1)
(11,101)12,822 73,154 44,497 
Net income (loss)
$(30,744)$106,080 $395,474 $301,916 
(1)Other segment expense, net during the three months ended September 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
September 30,		Nine Months Ended
September 30,
2025202420252024
VOXZOGO$218,387 $189,785 $653,532 $526,597 
VIMIZIM182,755 178,158 586,525 548,694 
NAGLAZYME122,025 131,906 365,182 369,584 
PALYNZIQ108,778 90,634 307,985 254,634 
ALDURAZYME53,709 71,029 159,123 144,849 
BRINEURA47,953 37,005 137,007 121,361 
KUVAN24,025 28,115 76,195 92,576 
ROCTAVIAN3,180 7,235 22,889 15,516 
Total net product revenues760,812 733,867 2,308,438 2,073,811 
Royalty and other revenues15,321 11,873 38,250 32,791 
Total revenues$776,133 $745,740 $2,346,688 $2,106,602 
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
September 30,		Nine Months Ended
September 30,
2025202420252024
United States$274,114 $235,515 $788,361 $655,963 
Europe195,184 191,017 667,900 607,755 
Latin America97,618 111,908 284,018 274,920 
Rest of world140,187 124,398 409,036 390,324 
Total net product revenues marketed by the Company707,103 662,838 2,149,315 1,928,962 
ALDURAZYME net product revenues marketed by Sanofi53,709 71,029 159,123 144,849 
Total net product revenues$760,812 $733,867 $2,308,438 $2,073,811 
The following table illustrates the percentage of the Company’s total Net Product Revenues attributed to the Company’s largest customers for the periods presented.
Three Months Ended
September 30,		Nine Months Ended
September 30,
2025202420252024
Customer A14 %11 %15 %13 %
Customer B12 10 11 10 
Customer C11 12 12 11 
Total37 %33 %38 %34 %
Concentration Information
On a consolidated basis, one customer accounted for 23% of the Company’s September 30, 2025 accounts receivable balance compared to December 31, 2024, when two customers accounted for 20% and 11% of the accounts receivable balance, respectively. As of September 30, 2025, and December 31, 2024, the accounts receivable balance for Sanofi included $135.7 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, regulatory uncertainty, 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 September 30, 2025 based on its analysis of the specific business circumstances and expectations of collection for each of the underlying accounts in these countries.