August 2, 2024 Meeting of the Genetic Metabolic Diseases Advisory Committee (GeMDAC)

The Committee will discuss new drug application (NDA) 214927, for arimoclomol, submitted by Zevra Denmark A/S, for the proposed indication of treatment of adults and pediatric patients 2 years of age and older with Niemann-Pick disease type C (NPC).
Meeting Information and Materials: https://www.fda.gov/advisory-committees/advisory-committee-calendar/updated-public-participation-information-august-2-2024-meeting-genetic-metabolic-diseases-advisory