FDA Accepts Elamipretide NDA for Barth Syndrome Treatment
Key Insights
The FDA has accepted Stealth BioTherapeutics (search)' NDA for elamipretide in treating Barth syndrome (search), a rare genetic condition with no approved therapies.
The application is supported by Phase 3 data and additional efficacy/safety data, with the FDA planning an advisory committee meeting for review.
Elamipretide, a mitochondrial (search)-targeted therapeutic, has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations for Barth syndrome (search).
Stealth BioTherapeutics (search) has announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for elamipretide for the treatment of Barth syndrome (search). This decision marks a significant step forward for a condition with no currently approved therapies.
The NDA is supported by positive data from the SPIBA-001 Phase 3 Natural History Control Study, along with additional efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide has previously been granted Fast Track Designation in 2017, Orphan Drug Designation in 2018, and Rare Pediatric Disease Designation in 2020.
Advisory Committee Meeting Planned
The FDA has indicated plans to hold an advisory committee meeting to discuss the application, a decision welcomed by Stealth BioTherapeutics (search). Reenie McCarthy, Chief Executive Officer, stated the company looks forward to the committee's input on the seriousness of Barth syndrome (search) and the urgency of the unmet need.
Elamipretide: A Potential First-in-Class Therapy
Elamipretide is a first-in-class mitochondrial (search)-targeted therapeutic. If approved, it would be the first marketing authorization for this drug. It is also in development for primary mitochondrial myopathy (search), with pivotal data from the fully-enrolled Phase 3 NuPOWER trial expected in late 2024.
About Barth Syndrome
Barth syndrome (search) is an ultra-rare genetic condition primarily affecting males, estimated to impact one in 1,000,000 males worldwide, or about 150 individuals in the United States. It is characterized by cardiac abnormalities, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with reduced life expectancy, with 85% of early deaths occurring by age 5. Currently, there are no FDA- or EMA-approved therapies for Barth syndrome.