Zevra Therapeutics, Inc. discovers, develops, and commercializes small-molecule drugs and prodrugs for rare and ultra-rare diseases. The company's commercial portfolio is anchored by MIPLYFFA (arimoclomol), an FDA-approved treatment for Niemann-Pick disease type C, and OLPRUVA (sodium phenylbutyrate), which is indicated for certain urea cycle disorders. Additionally, Zevra earns royalty revenue from AZSTARYS, a partnered ADHD medication utilizing its proprietary Ligand Activated Therapy platform. The company's clinical pipeline features KP1077, currently in development for idiopathic hypersomnia and narcolepsy, alongside celiprolol for vascular Ehlers-Danlos syndrome. Headquartered in Celebration, Florida, Zevra is actively expanding its geographic footprint by pursuing marketing authorizations in the European Union. Formerly known as KemPharm, the company rebranded in 2023 to reflect its strategic pivot toward commercial-stage rare disease therapeutics.
