FDA accepts filing NDA for elamipretide for the treatment of Barth Syndrome
Stealth BioTherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, announced that the FDA has accepted for filing its New Drug Application for elamipretide for the treatment of Barth syndrome
The NDA is supported by the positive data from the SPIBA-001 Phase III Natural History Control Study and additional supporting efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020.
The FDA indicated that it is currently planning to hold an advisory committee meeting to discuss the application. The application was assigned a standard review designation, which the Company has asked FDA to reconsider.
If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondrial targeted therapeutic. Elamipretide is also in development for primary mitochondrial myopathy, with pivotal data from the fully-enrolled Phase III NuPOWER trial expected in late 2024.
About Barth Syndrome: Barth syndrome is an ultra-rare genetic condition characterized by muscle weakness, cardiac abnormalities often leading to heart failure, recurrent infections, delayed growth, and reduced life expectancy. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 300,000 to 400,000 individuals worldwide at birth.
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