Ultragenyx Pharmaceutical Inc. (RARE) is a Healthcare stock trading at $26.30 (as of 2026-08-21), with a market capitalization of $2.59B. Figures are sourced from first-party U.S. SEC EDGAR filings and market data.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
Ultragenyx Pharmaceutical develops and commercializes therapies for rare and ultra-rare genetic diseases, such as Crysvita, and it makes money from the sales and royalties of its biologic medicines.
The companys profit model depends on how much revenue it generates from its commercial therapies and related royalties, while the latest quarter shows a deeply negative operating margin.
| Crysvita US/CA royalties | $0.1B |
Business quality (Mixed): Core strength is a portfolio of commercial rare-disease biologics, but the latest quarter indicates significant operating losses, so near-term performance is likely sensitive to product revenue levels.
| Price | $26.30 |
| Market Cap | $2.59B |
| Price / Sales | 3.62 |
| FCF Yield | -18.9% |
| Revenue (TTM) | $673.00M |
| Net Margin | -85.4% |
| Revenue Growth (YoY) | 28.5% |
| EPS Growth (YoY) | -23.1% |
| FCF Growth (YoY) | -11.5% |
| Altman Z-Score | -3.46 (distress) |
| Sector P/E (Healthcare) | 21.7 |
| Industry P/E (Pharmaceutical Products) | 17.2 |
| Top Institutional Holder | VANGUARD GROUP INC ($236.07M) |
| Institutional Holders Tracked | 10 |
| Congressional Trades (recent) | 4 |
| Sector | Healthcare |
| Industry | Pharmaceutical Products |
| CEO | Emil D. Kakkis |
| Employees | 1,294 |
| Country | US |
| IPO Date | 2014-01-31 |
Data as of 2026-08-21.
Figures sourced from U.S. Securities and Exchange Commission (SEC EDGAR) filings and market data. Not personalized investment advice.