FDA advisory committee supports Stealth's drug for Barth syndrome

A significant advancement for the biotechnology company Stealth Biotherapeutics: An advisory committee of the U.S. Food and Drug Administration (FDA) has voted in favor of using the drug Elamipretide for the treatment of Barth syndrome, despite mixed study results. Barth syndrome is an extremely rare genetic disorder that primarily affects males and is caused by mutations in the TAZ gene. The panel, consisting of ten experts from the Cardiovascular and Renal Drugs Advisory Committee, voted 10 to 6 in support of Elamipretide. Although the previous clinical trial, SPIBA-201, did not meet its primary endpoint of improving walking distance, patients and their families advocated for the medication's use due to its positive effects, such as weight gain and increased endurance. Despite the FDA report raising doubts about the statistical significance of the study results and the drug having been previously rejected by the FDA, Stealth aims to obtain approval by January 2025. The discussion surrounding the medication has highlighted not only the potential to improve the lives of Barth patients but also could be groundbreaking for similar rare diseases. Whether the FDA will follow the recommendations of the advisory committee remains open until the final decision.