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SEGMENT INFORMATION
3 Months Ended
Mar. 31, 2026
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
March 31,
20262025
Total revenues$766,208 $745,145 
Less:
Cost of sales194,999 151,558 
R&D expenses
Research and early pipeline87,291 90,470 
Later-stage clinical programs36,177 14,291 
Marketed products55,328 53,970 
SG&A expenses
S&M expenses124,927 102,531 
G&A expenses133,363 103,585 
Other segment expense, net (1)
28,596 43,054 
Net income$105,527 $185,686 
(1)Other segment expense, net during the three months ended March 31, 2026 and 2025 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
March 31,
20262025
VOXZOGO$219,880 $213,754 
Enzyme Therapies:
VIMIZIM210,204 188,346 
NAGLAZYME130,074 114,290 
PALYNZIQ89,558 93,269 
BRINEURA47,104 40,362 
ALDURAZYME36,742 48,977 
KUVAN23,880 25,136 
ROCTAVIAN2,636 10,510 
Total net product revenues760,078 734,644 
Royalty and other revenues6,130 10,501 
Total revenues$766,208 $745,145 
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
March 31,
20262025
United States$242,944 $241,694 
Europe218,836 229,578 
Latin America108,164 88,282 
Rest of world153,392 126,113 
Total net product revenues marketed by the Company723,336 685,667 
ALDURAZYME net product revenues marketed by Sanofi36,742 48,977 
Total net product revenues$760,078 $734,644 
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
March 31,
20262025
Customer A15 %14 %
Customer B11 12 
Customer C10 11 
Total36 %37 %
Concentration Information
On a consolidated basis, one customer accounted for 20% of the Company’s March 31, 2026 accounts receivable balance compared to December 31, 2025, when two customers accounted for 20% and 10% of the accounts receivable balance, respectively. As of March 31, 2026, and December 31, 2025, the accounts receivable balance for Sanofi included $152.6 million and $148.0 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, wars and military conflicts, 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 March 31, 2026 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.