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Niemann-Pick Type C News Research News

FDA Advisory Committee Votes To Recommend Arimoclomol as Effective Treatment for NPC

PDUFA Action Date for the Arimoclomol NDA is September 21, 2024 CELEBRATION, Fla., Aug. 02, 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) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) …