On February 12, 2026 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter and year ended December 31, 2025.
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"The year ahead marks an important turning point for the company, as we approach two potential product launches and a pivotal data readout that, together, could significantly accelerate our commercial revenue trajectory," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We are implementing a strategic restructuring plan to reduce our operating expenses and ensure our resources are squarely aligned with our highest-impact opportunities, while leading the future of rare disease with multiple first ever treatments."
Fourth Quarter 2025 Selected Financial Data Tables and Financial Results
Revenues (dollars in millions), (unaudited)
Three Months Ended December 31, Year Ended December 31,
2025 2024 2025 2024
Crysvita
Product sales – Latin America and Türkiye $ 40 $ 22 $ 177 $ 135
Royalty revenue – U.S. and Canada 97 87 275 249
Royalty revenue – Europe 8 7 29 26
Total Crysvita Revenue 145 116 481 410
Dojolvi 32 31 96 88
Evkeeza 17 10 59 32
Mepsevii 13 8 37 30
Total revenues $ 207 $ 165 $ 673 $ 560
Revenues
Ultragenyx reported $207 million in total revenue for the fourth quarter 2025, which represents 25% growth compared to the same period in 2024. Crysvita revenue in the fourth quarter 2025 was $145 million, which includes product sales of $40 million from Latin America and Türkiye. Dojolvi revenue in the fourth quarter 2025 was $32 million. Evkeeza revenue in the fourth quarter 2025 was $17 million.
Total revenue for the year ended December 31, 2025 was $673 million, which represents 20% growth compared to the prior year. Full year 2025 Crysvita revenue was $481 million, which represents 17% growth compared to the prior year. This includes product sales of $177 million from Latin America and Türkiye, which represents 31% growth compared to the prior year. Dojolvi revenue in 2025 was $96 million, which represents 9% growth compared to the prior year. Evkeeza revenue in 2025 was $59 million, which represents 84% growth compared to the prior year, as demand continues to build following launches in the company’s territories outside of the United States.
Selected Financial Data (dollars in millions, except per share amounts), (unaudited)
Three Months Ended December 31, Year Ended December 31,
2025 2024 2025 2024
Total revenues $ 207 $ 165 $ 673 $ 560
Operating expenses:
Cost of sales 29 17 109 77
Research and development 203 188 750 698
Selling, general and administrative 89 82 349 321
Total operating expenses 321 287 1,208 1,096
Net loss $ (129 ) $ (133 ) $ (575 ) $ (569 )
Net loss per share, basic and diluted $ (1.29 ) $ (1.39 ) $ (5.83 ) $ (6.29 )
Operating Expenses
Total operating expenses for the fourth quarter 2025 were $321 million. Total operating expenses for the year ended December 31, 2025 were $1.2 billion, including $153 million of non-cash stock-based compensation.
Net Loss
Net loss for the fourth quarter 2025 was $129 million, or $1.29 per share basic and diluted, compared with a net loss for the fourth quarter 2024 of $133 million, or $1.39 per share basic and diluted. Net loss for the year ended December 31, 2025 was $575 million, or $5.83 per share basic and diluted, compared with a net loss the prior year of $569 million, or $6.29 per share, basic and diluted.
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable securities were $737 million as of December 31, 2025. For the three months ended December 31, 2025, net cash used in operations was $100 million and for the year ended December 31, 2025 was $466 million.
Financial Guidance and Strategic Restructuring Plan
2026 revenue guidance
Total revenues, excluding potential revenue from new product launches, in the range of $730 million to $760 million, an increase of 8% to 13% compared to 2025
Crysvita revenue in the range of $500 million to $520 million, reflecting growing underlying global demand partially offset by expected timing of ordering patterns in Brazil
Dojolvi revenue in the range of $100 million to $110 million
Strategic restructuring plan and path to profitability in 2027
Ultragenyx has initiated a strategic restructuring plan designed to reduce its headcount and expenses and focus resources on its largest value drivers. The significant reduction and partial reinvestment of expenses, and the planned growth in revenue from current and new product launches, are designed to keep the company on its path to profitability in 2027.
Today, in connection with the restructuring plan, the company announced a 10% workforce reduction, impacting approximately 130 employees.
Based on the progression of the business and the reductions from the restructuring plan:
In 2026, combined R&D and SG&A expenses are expected to be flat to down low-single digits versus 2025. This includes the impact of spend reductions and approximately $50 million for severance, manufacturing, and other non-recurring restructuring charges.
In 2027, R&D expenses are expected to decrease from 2025 levels by 38%, or approximately $280 million, driven by the completion of multiple phase 3 studies and reduction of early-stage research efforts. SG&A expenses are expected to increase in support of new product launches and existing approved products. On a combined basis, 2027 R&D and SG&A expenses are expected to decrease at least 15% versus 2025.
2026 Clinical and Regulatory Catalysts
DTX401 (pariglasgene brecaparvovec) AAV8 gene therapy for glycogen storage disease type Ia (GSDIa): Biologics License Application (BLA) rolling submission completed in December 2025, with an anticipated Prescription Drug User Fee Act (PDUFA) action date in the third quarter of 2026.
UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome type A (MPS IIIA): The BLA was resubmitted in January 2026 and included substantial longer-term data, that were recently presented at the 2026 WORLDSymposium, on multiple measures of neurologic benefit to support an intermediate clinical endpoint for accelerated approval supported further by CSF heparan sulfate and other biomarker data, as agreed with the FDA during the last clinical review.
Earlier today the company received an Incomplete Response Letter (IRL) regarding its resubmitted BLA. The IRL requests additional supportive documentation related to its CRL CMC responses, which the company will provide in a resubmission.
GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): Data from the fully enrolled, pivotal, Phase 3 Aspire study in patients with a genetically confirmed diagnosis of UBE3A deletion is expected in the second half of 2026. Enrollment in the Phase 2/3 Aurora study is also underway in other genotypes and ages, with the first patient dosed in October 2025.
UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for Wilson disease: Enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data from this stage are expected in 2026.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Thursday, February 12, 2026, at 2 p.m. PT/5 p.m. ET to discuss the fourth quarter and full year 2025 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source The replay of the call will be available for three months.
(Press release, Ultragenyx Pharmaceutical, FEB 12, 2026, View Source [SID1234662646])