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SEGMENT INFORMATION (Tables)
9 Months Ended
Sep. 30, 2025
Segment Reporting [Abstract]  
Schedule of Reportable Segment Information
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.
Schedule of Disaggregation of Total Net Product Revenues by Product
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 
Schedule of Disaggregation of Total Net Product Revenues by Geographic Region 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 
Schedule of Total Net Product Revenue Concentrations Attributed to Largest Customers
The following table illustrates the percentage of the Company’s total Net Product Revenues attributed to the Company’s largest customers for the periods presented.
Three Months Ended
September 30,
Nine Months Ended
September 30,
2025202420252024
Customer A14 %11 %15 %13 %
Customer B12 10 11 10 
Customer C11 12 12 11 
Total37 %33 %38 %34 %