Zevra Therapeutics says FDA extends review period for arimoclomol NDA

Zevra Therapeutics announced the U.S. Food and Drug Administration has extended the review period for the New Drug Application for arimoclomol, an investigational orally delivered, first-in-class treatment for Niemann-Pick disease type C. In addition, the FDA has set a new Prescription Drug User Fee Act action date of September 21, 2024, and has re-affirmed its intent to present the resubmission for discussion at an advisory committee meeting to be scheduled. As previously reported, Zevra received acceptance of the NDA resubmission for arimoclomol in early January 2024, with an original PDUFA action date of June 21, 2024. The arimoclomol NDA resubmission included evidence supporting trial metrics, FDA-preferred analyses, and data from a multitude of additional studies that provide supportive evidence of arimoclomol’s impact in clinical and nonclinical settings. As part of the ongoing review, Zevra received notification from the FDA that it required more time to review the additional analyses provided by the Company in responses to recent information requests generated from the FDA’s review. The FDA has determined that the additional information constitutes a Major Amendment to the NDA, thereby resulting in an extension of the PDUFA action date.