Zevra Therapeutics Receives FDA Acceptance of Resubmission of NDA for Arimoclomol as a Treatment for Niemann-Pick Disease Type C

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[{"type":"text","content":"Arimoclomol NDA has been assigned a PDUFA action date of June 21, 2024 CELEBRATION, Fla., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced 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. “We are very pleased that the FDA has accepted the resubmission of the arimoclomol NDA following multiple collaborative and constructive meetings,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra. “This significant milestone brings us one step closer to the potential approval of arimoclomol for a community of patients with debilitating unmet medical needs. We would like to take the opportunity to acknowledge the NPC community for their continued support throughout the development of arimoclomol.” 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. 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. About Niemann-Pick Disease Type C (NPC): Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissue areas, including brain tissue. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those...

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