Zevra Therapeutics Says FDA Accepts Resubmission Of Arimoclomol NDA In Niemann-Pick Disease Type C

(RTTNews) - Rare disease therapeutics company Zevra Therapeutics, Inc. (ZVRA) announced Monday that the U.S. Food and Drug Administration (FDA) has acknowledged receipt of the resubmission of the New Drug Application (NDA) for arimoclomol as an orally-delivered, first-in-class treatment for Niemann-Pick disease type C.

Under the Prescription Drug User Fee Act (PDUFA), 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 (CRL) issued by the FDA in response to the prior arimoclomol NDA filing.