AdComm Votes for Arimoclomol for Niemann-Pick Disease Despite Some Data Doubts

Despite a mostly lukewarm reception to phase 2/3 trial findings, the FDA’s Genetic Metabolic Diseases Advisory Committee voted 11-5 in support of approving Zevra Therapeutics’ arimoclomol for type C Niemann-Pick disease in patients age two years and up.
Source: Drug Industry Daily