Elamipretide for Barth Syndrome receives priority review designation.-Stealth BioTherapeutics.
Stealth BioTherapeutics, announced that the 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. There are no other therapies approved or known to be in clinical development for this disease.
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.
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, and dry age-related macular degeneration, with pivotal Phase III clinical trials initiating this quarter..
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