What's Happening?
Stealth BioTherapeutics has resubmitted its New Drug Application (NDA) for elamipretide to the FDA for the treatment of Barth syndrome. The resubmission follows FDA's complete response in May 2025, addressing manufacturing deficiencies and including post-marketing commitments. Elamipretide, a mitochondrial targeted therapeutic, showed significant improvement in knee extensor muscle strength in clinical trials, correlating with gains in the six-minute walk test.
Why It's Important?
Barth syndrome is a rare genetic condition with no current FDA-approved therapies, affecting life expectancy and quality of life. The resubmission of elamipretide's NDA represents a potential breakthrough in treatment options for this debilitating disease. Approval would mark the first marketing authorization for elamipretide, offering hope to patients and families affected by Barth syndrome.
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What's Next?
Stealth BioTherapeutics is urging the FDA to expedite the review process, aiming for a Class 1 review to avoid further delays. If approved, elamipretide could pave the way for additional applications in treating other mitochondrial dysfunction-related diseases. The company continues to develop its pipeline, focusing on rare and age-related diseases.
