Ultragenyx Pharmaceutical Inc

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases. The company markets four approved products: Crysvita (burosumab) for X-linked hypophosphatemia, Dojolvi (triheptanoin) for long-chain fatty acid oxidation disorders (LC-FAOD), Evkeeza (evinacumab) for homozygous familial hypercholesterolemia, and Mepsevii (vestronidase alfa) for mucopolysaccharidosis VII. Ultragenyx generates revenue from direct product sales in markets including Latin America, Türkiye, and Japan, as well as royalty revenue from its collaboration with Kyowa Kirin for Crysvita in the U.S., Canada, and Europe. The company maintains a deep pipeline of gene therapy and other novel modalities, including programs in glycogen storage disease type Ia (DTX401), Sanfilippo syndrome type A (UX111), Angelman syndrome (GTX-102), Wilson disease (UX701), OTC deficiency (DTX301), and GNE myopathy (UX016). The company's strategy emphasizes time- and cost-efficient drug development targeting diseases with high unmet medical need, clear biology, and typically no existing approved therapies addressing the underlying disease. Updated Aug 4, 2026