What's Happening?
Stealth BioTherapeutics has resubmitted its application for the drug elamipretide, aimed at treating Barth syndrome, to the FDA under an accelerated review pathway. Barth syndrome is a rare genetic disorder causing muscle weakness, fatigue, and heart failure, with a high mortality rate by age five. The initial application was rejected due to manufacturing issues and concerns about clinical efficacy. The resubmission focuses on improvements in knee extensor muscle strength, a key endpoint in the Phase II TAZPOWER trial, which showed significant improvement. The FDA has agreed to review the application by September 26, 2025, instead of the standard February 2026 timeline.
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Why It's Important?
The accelerated review of elamipretide could provide a much-needed treatment option for Barth syndrome, a condition with limited therapeutic alternatives. Approval would represent a breakthrough for patients and families affected by this debilitating disorder. The resubmission highlights the importance of addressing FDA concerns and refining clinical endpoints to meet regulatory standards. Success in this review could also bolster Stealth BioTherapeutics' position in developing treatments for other mitochondrial disorders, potentially leading to advancements in rare disease therapeutics.
What's Next?
The FDA's decision on elamipretide is expected by September 26, 2025. If approved, Stealth BioTherapeutics plans to conduct a post-marketing trial to confirm clinical benefits. The company may also explore further applications of elamipretide for other conditions, such as primary mitochondrial myopathy and dry age-related macular degeneration, expanding its therapeutic portfolio.
