Zevra Announces FDA Advisory Committee Meeting to Review Arimoclomol for the Treatment of Niemann-Pick Disease Type C

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[{"type":"text","content":"Meeting with the recently formed GeMDAC set for August 2, 2024\nCELEBRATION, Fla., July 09, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2, 2024, to review the New Drug Application (NDA) for arimoclomol as an orally delivered, first-in-class treatment for Niemann-Pick disease type C (NPC). According to the notice provided, the FDA intends to make the background materials available to the public no later than two business days before the meeting. The GeMDAC consists of experts in the fields of medical genetics, inborn errors of metabolism, epidemiology, and other related specialties. The NDA for arimoclomol has been assigned a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024. “We look forward to presenting data from the arimoclomol NDA to the recently formed GeMDAC, which will also provide the opportunity for patients and other members of the NPC community to share their treatment journeys and highlight the desperate need for an approved therapy,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra. About Niemann-Pick Disease Type C (NPC) Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissues and organs, including the brain. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease progression is irreversible and can be fatal within months or take years to be diagnosed and advance in severity. About Arimoclomol Arimoclomol, Zevra’s orally delivered, first-in-class investigational drug product candidate for the treatment of NPC, has been granted Or...

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