Zevra Therapeutics, Inc.

Zevra Therapeutics, Inc. is a commercial-stage company focused on developing and delivering therapies for people with rare diseases. The company's lead product, MIPLYFFA, is marketed in the United States for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disease. MIPLYFFA was approved by the U.S. Food and Drug Administration (FDA) on September 20, 2024, for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older, and became commercially available in the United States in November 2024. The company also has another product, OLPRUVA, which remains commercially available. Zevra Therapeutics is advancing the development of its clinical stage asset, celiprolol, and plans to expand its pipeline through inorganic growth. In May 2022, the company purchased all assets and operations of Orphazyme A/S related to arimoclomol. The name Zevra, derived from the Greek word for zebra, reflects the company's focus on rare diseases.