ZVRA

Zevra Therapeutics, Inc. is a commercial-stage rare disease company with a late-stage pipeline committed to bringing therapeutics to people living with rare diseases. Its commercial products and active development assets are MIPLYFFA, Arimoclomol, OLPRUVA, and Celiprolol. Its lead product, Niemann-Pick disease type C (NPC), is a rare, progressive neurodegenerative disorder. NPC is an ultra-rare and progressive neurodegenerative disease characterized by an inability of the body to transport cholesterol and lipids inside of cells. OLPRUVA (sodium phenylbutyrate) for oral suspension is approved in the United States as adjunctive therapy to standard of care, which includes dietary management, for the chronic management of UCDs involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinic acid synthetase. Celiprolol is a selective adrenoceptor modulator (SAM). Celiprolol is approved in certain EU states for the treatment of hypertension and angina.