FDA to Review Elamipretide for Barth Syndrome on October 10, 2024

Stealth BioTherapeutics Inc. announced that the FDA will hold an advisory committee meeting on October 10, 2024, to review the New Drug Application for elamipretide, a potential treatment for Barth syndrome. This condition is an ultra-rare genetic disorder affecting around 150 individuals in the U.S., characterized by severe cardiac and skeletal muscle issues that lead to significantly reduced life expectancy. The company expressed gratitude to all who contributed to the studies supporting this application, highlighting the urgent need for effective therapies for this devastating disease. Elamipretide has been granted priority review status, with a Prescription Drug User Fee Act action date set for January 29, 2025. If approved, it would be the first marketing authorization for this first-in-class mitochondria-targeted therapeutic. The company is also advancing elamipretide in Phase 3 trials for primary mitochondrial myopathy and dry age-related macular degeneration, with pivotal data expected by the end of 2024. Barth syndrome primarily affects males and is associated with a range of serious health issues, including exercise intolerance, muscle weakness, and heart failure. The disease's severity often leads to early mortality, with a significant percentage of deaths occurring by age five. Currently, there are no approved therapies for this condition, underscoring the critical need for new treatment options. The upcoming advisory committee meeting represents a significant step in the regulatory process for elamipretide, as stakeholders hope to discuss its potential benefits for patients suffering from Barth syndrome. The outcome of this meeting could pave the way for a much-needed therapeutic intervention for this vulnerable patient population.