Zevra Therapeutics, Inc. is a commercial-stage rare disease company that combines science, data and patient needs to create therapies for diseases with limited or no treatment options. The company is headquartered in Celebration, Florida and currently employs 61 full-time employees. The company went IPO on 2015-04-16. 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.
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