FDA Advisory Committee Votes Favorably that the Data Support Arimoclomol as Effective Treatment for Patients with Niemann-Pick Disease Type C

FDA's GeMDAC voted favorably (11 yes, 5 no) that arimoclomol is effective for Niemann-Pick disease type C (NPC). Zevra Therapeutics remains confident in arimoclomol's clinical benefit and its path to approval, with a PDUFA action date of September 21, 2024.