Ultragenyx Pharmaceutical, Inc. (RARE)

In August 2025, Ultragenyx reported a 13% revenue growth and announced FDA Breakthrough Therapy Designation for GTX-102. In November 2025, the Q3 earnings report showed a wider loss per share, but revenues increased by 15%. In December 2025, Phase 3 studies for setrusumab failed, leading to a significant stock price drop. Later in December 2025, Ultragenyx completed the BLA submission for DTX401. In January 2026, an investigation was announced regarding potential securities fraud, and Ultragenyx reported preliminary 2025 revenue exceeding guidance with approximately 20% growth.

There is no specific information indicating demand seasonality for Ultragenyx Pharmaceutical Inc.'s products and services. Demand is generally driven by medical need rather than seasonal fluctuations.

Ultragenyx Pharmaceutical Inc. is focused on developing treatments for serious rare and ultra-rare genetic diseases, employing various drug modalities across multiple disease categories. Its major approved products include Crysvita for X-linked hypophosphatemia and tumor-induced osteomalacia, Dojolvi for long-chain fatty acid oxidation disorders, Mepsevii for mucopolysaccharidosis type VII, and Evkeeza (non-U.S. rights) for homozygous familial hypercholesterolemia. The company also has a pipeline including gene therapy programs and GTX-102 for Angelman syndrome.

Ultragenyx Pharmaceutical Inc. is headquartered in Novato, California, and has a presence in the Boston area and Miami. Its therapies are approved in Canada, Latin America, Europe, and Japan. The company's stock is traded on Nasdaq and exchanges in Stuttgart, Frankfurt, Dusseldorf, Hamburg, London, Berne, Mexico, and Sao Paulo.

In May 2025, Ultragenyx released its 2024 Impact Report, highlighting its commitment to corporate responsibility. However, the company's reputation faced a setback in December 2025 when its Phase 3 studies for setrusumab failed, leading to a stock price decline and an investigation by a law firm in January 2026.

Ultragenyx Pharmaceutical Inc. is predominantly owned by institutional investors, holding approximately 98.96% of the shares. Major institutional owners include Vanguard Group Inc., Fmr Llc, and BlackRock, Inc. The largest individual shareholder is CEO Emil D. Kakkis, owning about 7.55% of the company. Insiders collectively own 16.60% of the company, while retail investors hold 0.00%.