Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) is a biopharmaceutical company developing and commercializing treatments for serious rare and ultra-rare genetic diseases. Its approved therapies include Crysvita for X-linked hypophosphatemia and tumor-induced osteomalacia; Mepsevii for mucopolysaccharidosis VII; Dojolvi… From the company’s description.
Over the twelve months to Sep 30, 2026, Ultragenyx Pharmaceutical Inc. reported revenue of $730.28M, up 15.8% from a year earlier, and a net loss of $523.56M. Its operating margin was −64.0%, and operations used $436.83M in cash. In fiscal 2025, it reported revenue of $673.00M, up 20.1% from fiscal 2024, and a net loss of $575.00M.
Margins and return on equity change in ; everything else in percent. Fewer shares and less debt are shown plainly, not as bad news — it depends what you make of buybacks. Dotted labels explain each measure. is shown separately from the annual figures.
What Ultragenyx Pharmaceutical Inc. does
In the company’s own words
Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) is a biopharmaceutical company developing and commercializing treatments for serious rare and ultra-rare genetic diseases. Its approved therapies include Crysvita for X-linked hypophosphatemia and tumor-induced osteomalacia; Mepsevii for mucopolysaccharidosis VII; Dojolvi for long-chain fatty acid oxidation disorders; and Evkeeza for homozygous familial hypercholesterolemia. Crysvita is developed and commercialized with Kyowa Kirin, while Regeneron markets Evkeeza in the United States and Ultragenyx holds commercialization rights outside the country. The company also develops clinical-stage candidates using adeno-associated virus gene therapy, antisense oligonucleotides and antibody approaches, targeting conditions including Sanfilippo syndrome type A, glycogen storage disease type Ia, Angelman syndrome, ornithine transcarbamylase deficiency and Wilson disease. Ultragenyx was founded in 2010 and has built its portfolio through product development, licenses and collaborations, and the 2022 acquisition of GeneTx, which brought the GTX-102 Angelman syndrome candidate.
Company details
- Sector
- Health Care
- Industry (SIC)
- Pharmaceutical Preparations
- Headquarters
- NOVATO, CA
- Fiscal year end
- December
- CIK
- 1515673
- SIC
- 2834
Financial performance, year by year
Fiscal 2014–2025 · USDThe full income statement, balance sheet and cash flow for each of these years — every figure traceable to its filing — is on the Financials tab with a free account.
Who owns Ultragenyx Pharmaceutical Inc. — and what changed
Funds as of 2026Q2 · superinvestors as of 2026-06-30
Largest fund holders · Ultragenyx Pharmaceutical Inc.’s weight in each
Fund ownership is a floor, not a census — pensions, insurers and people holding shares directly are not visible here.
Fund ownership counts N-PORT filers only, so it is a floor rather than a census — pensions, insurers and direct holders are not visible here, and the remainder is not “held by individuals”.
Every fund and institutional holder, quarter by quarter, is on the Ownership tab with a free account.
Recent filings
All filings →Each filing opens into its own statements and disclosures here.
Put this business in context.
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