Zevra edges closer to FDA review of resubmitted rare disease drug

July 10, 2024
The U.S. FDA will convene an AdComm meeting to discuss Zevra Therapeutics' orally delivered, potential first-in-class treatment for the ultra-rare neurodegenerative lysosomal storage disorder, Niemann-Pick disease type C (NDC), the company announced.
The recently formed Genetic Metabolic Diseases Advisory Committee will meet on August 2, 2024, to review the resubmitted NDA for arimoclomol, a heat shock protein amplifier. The application has a September 21 PDUFA date, which was extended from June 21.

This will be arimoclomol's second go-around with the FDA. Back in 2021, the agency rejected the drug — which at the time was owned by Denmark-based Orphazyme — requesting additional qualitative and quantitative efficacy evidence. Zevra (known as KemPharm at the time) picked up the drug in May 2022 with plans to refile the NDA for arimoclomol in NPC.
According to Zevra, the company addressed the earlier FDA concerns by providing additional evidence to support the use of the Niemann-Pick type C Clinical Severity Scale and, conducting additional studies used to support the potential mechanism of action. Additionally, the company included new trial data in the resubmission.