Positive FDA AdCom vote for Zevra arimoclomol

US rare disease drug focussed company Zevra Therapeutics (NasdaqGS: ZVRA) saw its shares rise nearly 43% to $9.00 in after-hours trading on Friday as it announced a positive development for its arimoclomol.

Zevra noted that the US Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC).

This was welcome news for Zevra, given that in 2021 the FDA rejected approval of arimoclomol, issuing a complete response letter(CRL) to the then owner of the drug Orphazyme. At the time, the agency called for additional evidence around a scale used to measure progression in disease severity in a pivotal study, as well as more efficacy data beyond that generated in the single Phase II/III trial.