Zevra Therapeutics announces FDA GeMDAC meeting to review arimoclomol

Zevra Therapeutics announced that the U.S. Food and Drug Administration has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee on August 2, 2024, to review the New Drug Application for arimoclomol as an orally delivered, first-in-class treatment for Niemann-Pick disease type C. 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 action date of September 21, 2024.