Ultragenyx Pharmaceutical Inc. reported a total revenue of $673 million for the fiscal year ending December 31, 2025, marking a 20% increase from $560 million in 2024. This growth was primarily driven by increased demand for its approved products, particularly Crysvita, which saw sales rise by 31% to $177 million, and Evkeeza, which experienced an 84% increase in revenue to $59 million. The company also noted a rise in royalty revenue from Crysvita, which increased by 11% to $304 million. Despite this revenue growth, Ultragenyx continued to operate at a loss, reporting a net loss of $575 million for 2025, slightly higher than the $569 million loss in the previous year.
In terms of operational changes, Ultragenyx initiated a strategic restructuring plan in February 2026, which included a 10% reduction in workforce, amounting to approximately 130 employees. This decision was made to align the company’s resources with its current pipeline requirements and to manage costs more effectively. The company also reported a total employee count of 1,371 as of December 31, 2025, with a significant portion dedicated to research and development.
Ultragenyx's product pipeline includes several gene therapy candidates, with UX111 (rebisufligene etisparvovec) for Sanfilippo syndrome type A being a focal point. The company resubmitted its Biologics License Application (BLA) for UX111 in January 2026, following a Complete Response Letter from the FDA in February 2026, which requested additional documentation. The company is also advancing other gene therapy candidates, including DTX401 for Glycogen Storage Disease Type Ia and GTX-102 for Angelman Syndrome, with ongoing clinical trials expected to yield data in the coming years.
Looking ahead, Ultragenyx anticipates continued operating losses in the near term but expects to achieve profitability by 2027, contingent on the successful commercialization of its products and product candidates. The company has $737 million in cash, cash equivalents, and marketable securities as of year-end 2025, which it believes will be sufficient to fund its operations for at least the next 12 months. However, it acknowledges the need for additional capital to support ongoing clinical studies and commercialization efforts, which may require future fundraising activities.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing therapies for rare and ultrarare genetic diseases. Its portfolio includes approved biologics and gene therapies targeting conditions like XLH, MPS VII, and HoFH. The company emphasizes efficient drug development, strategic licensing, and global commercialization to address high unmet medical needs in niche markets.
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