STEALTH BIOTHERAPEUTICS' BARTH SYNDROME NDA RECEIVES PRIORITY REVIEW DESIGNATION
NEEDHAM, Mass., May 7, 2024 /PRNewswire/ -- Stealth BioTherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration ("FDA") has now determined that its New Drug Application ("NDA") for elamipretide for the treatment of Barth syndrome is entitled to a Priority Review designation. Per FDA policy, the user fee goal date of January 29, 2025 is unchanged.
- The FDA grants Priority Review to applications for therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
- Barth syndrome is an ultra-rare cardioskeletal disease which results in frequent pediatric deaths and imposes a significant disease burden on all affected individuals.
- The FDA's decision to grant Priority Review designation follows its March 29, 2024 filing decision in which the FDA assigned a standard review designation.
- Elamipretide has previously received Orphan Drug, Fast Track and Rare Pediatric Designations for elamipretide for the treatment of Barth syndrome.