FDA delays PDUFA action date for elamipretide

US clinical-stage drug developer Stealth BioTherapeutics announced that the US Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) action date for the New Drug Application (NDA) for elamipretide, a first-in-class mitochondria-targeted therapeutic in development for individuals with Barth syndrome. The new PDUFA target action date is April 29, 2025.

Stealth was notified last week that the agency requires additional time to conduct a full review of supplemental information provided in response to recent FDA requests that were received following the positive Cardiovascular and Renal Drugs Advisory Committee (CRDAC) meeting on October 10, 2024.

The FDA advised that these submissions constitute Major Amendments to the NDA, resulting in a standard three-month extension from the original action date of January 29, 2025. Importantly, the FDA has not identified any safety issues and has not requested any new pre-marketing studies. In addition, the FDA has reconfirmed previously communicated post-marketing commitments and Stealth has addressed all information requests from the Agency to date.