Zevra Therapeutics Provides FDA Update on the PDUFA Action Date for Arimoclomol as a Treatment for Niemann-Pick Disease Type C

The new Prescription Drug User Fee Act (PDUFA) action date set by the FDA is September 21, 2024

CELEBRATION, Fla., March 04, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for arimoclomol, an investigational orally delivered, first-in-class treatment for Niemann-Pick disease type C (NPC). In addition, the FDA has set a new Prescription Drug User Fee Act (“PDUFA”) 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.

“While the PDUFA action date extension represents a delay, we remain confident in the potential for arimoclomol to help people living with NPC, and we will continue to work closely with the FDA as they complete their review,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra. “We applaud the NPC Patient Advocacy Community who recently submitted an informal petition to the FDA in support of arimoclomol and reinforcing the urgent need for an FDA-approved treatment to improve the course of this relentless and fatal disease. As the review continues, Zevra will maintain the early access program for arimoclomol and work tirelessly to bring this potential therapy to patients as soon as possible.”