BRIEF—Zevra’s arimoclomol to face FDA AdCom

Florida, USA-based drugmaker Zevra Therapeutics revealed that the US Food and Drug Administration (FDA) has indicated it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2, 2024, to review the New Drug Application (NDA) for arimoclomol as an orally delivered, first-in-class treatment for Niemann-Pick disease type C (NPC).

According to the notice, the FDA intends to make the background materials available to the public no later than two business days before the meeting.

The GeMDAC consists of experts in the fields of medical genetics, inborn errors of metabolism, epidemiology, and other related specialties.