FDA Advisory Committee Votes Favourably that the Data Support Arimoclomol as Effective Treatment for Patients with Niemann-Pick Disease Type C
Read the letter from Zevra Therapeutics here:
Here is a summary of the article:
FDA Advisory Committee Vote: The U.S. FDA Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favourably (11 yes, 5 no) that Arimoclomol is effective in treating patients with NPC.
Clinical Data Support: The committee considered clinical data from pivotal trials, long-term open-label extension studies, and expanded access programs.
About NPC: NPC is an ultra-rare, progressive, neurodegenerative lysosomal storage disorder caused by mutations in NPC1 or NPC2 genes.
Arimoclomol: Zevra’s investigational drug candidate for NPC has received Orphan Drug designation and other regulatory designations (2). The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024 (3,4).
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