Here are the 10-K annual reports from Ultragenyx Pharmaceutical Inc. (RARE) in our archive since June 2025, newest first, and each one links through to the document itself with the summary and the scores our analysis gives it.
A 10-K covers the audited annual report, with the full financial statements, so if you follow RARE and want that one kind of document rather than the whole filing history, this is the page to keep. The company's other filings, of every form, are on the full RARE filings page.
Ultragenyx Pharmaceutical focuses on therapies for serious rare and ultra-rare genetic diseases and reports a broad mix of approved drugs and late-stage programs. Approved products include Crysvita for X-linked hypophosphatemia and tumor-induced osteomalacia, Mepsevii for MPS VII, Dojolvi for long-chain fatty acid oxidation disorders, and Evkeeza for homozygous familial hypercholesterolemia in partnered territories.
The company highlights an advanced pipeline across four modalities: biologics, small molecules, AAV gene therapy, and nucleic acids. Key programs include UX111 for Sanfilippo syndrome type A, where a resubmitted BLA received an Incomplete Response Letter focused on CMC documentation, and DTX401 for glycogen storage disease type Ia, supported by 96-week Phase 3 data and a completed rolling BLA with an expected PDUFA action date in the third quarter of 2026.
Ultragenyx is also running pivotal studies for Angelman syndrome (GTX-102), OTC deficiency (DTX301), Wilson disease (UX701), and osteogenesis imperfecta (UX143). The company notes that Phase 3 Orbit and Cosmic studies for UX143 did not meet their primary fracture endpoints, and additional analyses are underway to assess a potential path forward. As of June 30, 2025, non-affiliate equity market value was approximately $3.4 billion, and 96,629,788 shares of common stock were outstanding as of February 13, 2026.