Ultragenyx Pharmaceutical, Inc. (RARE)

Ultragenyx's stock price has declined, with a 31.34% decrease from November 20, 2024, to November 19, 2025. Key events include a wider-than-expected loss in the Q3 2025 earnings report, reaffirmation of 2025 revenue guidance, advancements in the rare disease pipeline, analyst rating changes with lowered price targets, and insider selling exceeding $11 million.

Ultragenyx anticipates some quarter-to-quarter revenue variability but remains confident in the growing underlying demand for its products globally. Demand for treatments is likely driven by patient diagnosis and ongoing medical need rather than typical seasonal consumer trends. The company's financial reporting acknowledges and plans for some quarterly revenue variability.

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing treatments for serious rare and ultra-rare genetic diseases, utilizing various drug modalities. Its approved products include Crysvita for XLH and TIO, Mepsevii for MPS VII, Dojolvi for LC-FAOD, and Evkeeza for HoFH (non-U.S. rights). The company has a clinical-stage pipeline with candidates for GSDIa, ornithine transcarbamylase deficiency, Wilson disease, osteogenesis imperfecta, Angelman syndrome, Sanfilippo syndrome type A, and glycogen storage disease type III.

Ultragenyx Pharmaceutical Inc. has a broad international market presence, including operations in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific region. The company is headquartered in Novato, California, with a presence in the Boston area and a Latin American headquarters in Miami. Its stock is traded on NASDAQ and various European, Mexican, and Brazilian exchanges.

In 2024, Ultragenyx received positive recognition for its corporate responsibility and as an employer. The company was named one of the 100 Most Sustainable Companies by Barron's and recognized as one of the Top Places to Work in the USA for the second consecutive year, receiving six Cultural Excellence Awards from Top Workplaces. It also received accolades from The Boston Globe and the San Francisco Chronicle and was honored with the MassEcon Gold Award for Economic Impact in the Northeast region.

Ultragenyx Pharmaceutical Inc. is primarily owned by institutional shareholders, holding approximately 94.45% of the company's shares. Insiders own about 16.60%. Major institutional owners include Vanguard Group Inc., BlackRock Inc., Fmr LLC, State Street Corporation, Sands Capital Management LLC, and JPMorgan Chase & Co. The largest individual shareholder is Emil D. Kakkis.