Zevra Therapeutics, Inc.

Zevra Therapeutics, Inc. is a commercial-stage biopharmaceutical company dedicated to developing and commercializing therapies for rare diseases. The company's mission is to bring life-changing therapeutics to individuals living with rare conditions that have limited or no treatment options. Headquartered in Boston, MA, with a corporate office in Celebration, FL, Zevra Therapeutics was formerly known as KemPharm, Inc. and rebranded in February 2023 to reflect its intensified focus on the rare disease community.

Zevra Therapeutics' portfolio includes commercial products and a late-stage pipeline. Its approved therapies in the U.S. include MIPLYFFA® (arimoclomol) for Niemann-Pick disease type C (NPC) and OLPRUVA® (sodium phenylbutyrate) for certain urea cycle disorders (UCDs). The company is also advancing celiprolol, an investigational product candidate in Phase 3 clinical trials for vascular Ehlers-Danlos syndrome (vEDS).

Recent performance highlights Zevra Therapeutics' transition into a profitable commercial-stage entity, driven by the successful launch and uptake of MIPLYFFA, which received FDA approval in September 2024. The company reported significant revenue growth in 2025, largely attributed to MIPLYFFA sales, and is actively pursuing market expansion, including a regulatory application for MIPLYFFA in Europe. Neil F. McFarlane serves as the President, CEO & Director, and Justin Renz was appointed Chief Financial Officer in March 2026.