FDA Advisory Committee positive forarimoclomol as effective in the treatment of patients with Niemann-Pick disease type C (NPC)
Zevra Therapeutics, Inc. announced that the FDA Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC)
The GeMDAC, which consists of experts in the fields of medical genetics, inborn errors of metabolism, epidemiology and other related specialties, discussed the benefits and risks of arimoclomol, including the data recently presented at the 45th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), and reviewed comments received from independent experts, NPC patients, and patient advocacy group representatives. The committee’s recommendation will be considered by the FDA as it completes its independent review of the arimoclomol NDA; however, the feedback from the GeMDAC is not binding upon the Agency.
The arimoclomol NDA has been assigned a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024.
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