Zevra Therapeutics announces FDA acceptance of resubmission of arimoclomol NDA

Zevra Therapeutics announced that the U.S. Food and Drug Administration has acknowledged receipt of the resubmission of the New Drug Application for arimoclomol as an orally-delivered, first-in-class treatment for Niemann-Pick disease type C. Under the Prescription Drug User Fee Act, the FDA has deemed the arimoclomol NDA resubmission to be a Class II complete response which has a six-month review period from the date of resubmission. As a result, the FDA has assigned a PDUFA action date of June 21, 2024, and currently intends to present the resubmission for discussion in an advisory committee. Zevra believes that its resubmission of the arimoclomol NDA addresses the concerns previously raised in the June 2021 complete response letter issued by the FDA in response to the prior arimoclomol NDA filing. The resubmission includes additional evidence supporting trial metrics, FDA-preferred analyses, and data from multiple additional studies that provide supporting evidence of arimoclomol’s efficacy in clinical and non-clinical settings.