Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) is a biopharmaceutical company that develops and commercializes therapies for rare and ultra-rare genetic diseases. Revenue comes from product sales of approved therapies, with four marketed products: Crysvita (burosumab) for X-Linked Hypophosphatemia and Tumor-Induced Osteomalacia, Dojolvi (triheptanoin) for Long-Chain Fatty Acid Oxidation Disorders, Evkeeza (evinacumab) for Homozygous Familial Hypercholesterolemia, and Mepsevii for MPS VII. Product candidates span biologics, small molecules, AAV gene therapy, and nucleic acid modalities, with assets including DTX401 for GSDIa, DTX301 for OTC deficiency, and GTX-102 for Angelman syndrome. Ultragenyx in-licenses product candidates primarily from academic institutions and pharmaceutical partners, retaining global commercialization rights. The company operates its own commercial organization across North America, the EU, the UK, Latin America, Türkiye, and Asia, supplemented by third-party distributors in smaller markets. It was founded and is headquartered in the United States, trading on NASDAQ under the ticker RARE.

Revenue model

Product sales from four approved rare disease therapies sold through a direct commercial organization in major developed markets and third-party distributors in smaller markets. Assets are primarily in-licensed from academic institutions and pharmaceutical partners, with Ultragenyx retaining global commercialization rights.

Products and services

Approved products: Crysvita (burosumab) for XLH and TIO; Dojolvi (triheptanoin) for LC-FAOD; Evkeeza (evinacumab) for HoFH; Mepsevii for MPS VII. Clinical pipeline: DTX401 (pariglasgene brecaparvovec, AAV8 gene therapy) for GSDIa; DTX301 (avalotcagene ontaparvovec, AAV8 gene therapy) for OTC deficiency; GTX-102 (antisense oligonucleotide) for Angelman syndrome; UX111 (BLA under FDA review) with an Incomplete Response Letter received February 2026.

Customers and end markets

Patients with rare and ultra-rare genetic diseases in developed markets. Addressable patient populations disclosed in the FY2025 10-K include approximately 48,000 XLH patients in the developed world, 8,000 to 14,000 LC-FAOD patients, and 6,000 GSDIa patients in the developed world, and approximately 10,000 OTC deficiency patients in the developed world.

Value-chain role

Drug developer and commercial-stage biopharmaceutical company. In-licenses clinical-stage assets from academic institutions and pharmaceutical partners, conducts clinical development, seeks regulatory approval, and commercializes approved therapies through its own sales organization and third-party distributors.

Geographic exposure

North America, European Union, United Kingdom, Latin America, Türkiye, Asia, and select international markets via owned commercial operations. Smaller markets served through third-party distributor networks.

Competitors

Oral phosphate replacement and vitamin D therapy (standard of care competing with Crysvita for XLH)

Source: SEC 10-K, filed 2026-02-18

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