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Zevra Therapeutics Resubmits Arimoclomol NDA To FDA
(RTTNews) - Zevra Therapeutics, Inc. (ZVRA) announced Wednesday it resubmitted its New Drug Application (NDA) for arimoclomol to the U.S. Food and Drug Administration (FDA) on December 22, 2023. Arimoclomol is an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC).
The new data included in the resubmission comes from multiple non-clinical studies, natural history comparisons, real-world data generated from the ongoing early access programs in the U.S. and the European Union. It also included data from the four-year open-label extension of the Phase 2/3 clinical trial (NCT02612129). Results from this open-label trial suggest that arimoclomol reduces the long-term progression of NPC.
Arimoclomol has been evaluated in a total of 21 studies across a range of Phase 1, 2 or 3 clinical trials evaluating its safety and efficacy across more than 600 subjects in NPC, other disease or healthy subjects.
The primary efficacy trial evaluating arimoclomol for the treatment of NPC was a Phase 2/3 double-blind, placebo-controlled trial (CT-ORZY-NPC-002) of arimoclomol in 50 patients with NPC.
Based on standard NDA resubmission review timelines, an acknowledgment letter from the FDA that the resubmission is complete and setting the PDUFA date is expected within 30 days. Zevra expects the NDA to be classified as a Class II submission which would be subject to a review period by the FDA within six months from the date of submission.
Zevra Therapeutics Says FDA Extends NDA Review Period For Arimoclomol
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