Ultragenyx has several approved treatments, yet it still burns cash because finding rare-disease patients is costly and some of its biggest drug sales don’t fully flow back to the company. With expensive gene-therapy launches ahead, the path to a true turnaround may be far less comfortable than it looks.Read more
Ultragenyx is betting that new gene therapies and faster global rollouts can help more people with rare genetic diseases and push growth beyond what many expect. But its future also hinges on a small set of programs, with pricing pressure, tougher regulators, and rising competition that could slow progress.Read more
Ultragenyx has several rare-disease treatments already on the market, but its next chapter depends on a handful of high-stakes trial results and regulatory decisions. If those go well, new launches could reignite growth—if not, rising costs, payer pushback, and setbacks could keep pressure on the business.Read more
Ultragenyx builds medicines for rare genetic diseases, and wider genetic testing means more patients can now be found and treated. New late-stage programs and gene-therapy work could broaden its lineup, but the company still burns cash and faces approval delays, pricing pushback, and tough competition.Read more
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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, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. 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 the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company’s products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; 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; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701, an adeno-associated AAV9 gene therapy which is in Phase 2 clinical trial for the treatment of Wilson liver disease. The company has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Regeneron; and Abeona. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
