Elamipretide received accelerated FDA approval in September 2025 as the first treatment specifically approved for Barth syndrome, a rare genetic disease that damages mitochondria and causes heart and muscle weakness. It works by binding to cardiolipin on the inner mitochondrial membrane, stabilizing the machinery that produces cellular energy. While a pivotal trial showed no significant improvement on a standard walk test at the primary endpoint, benefits emerged over a 168-week open-label follow-up period, which supported the accelerated approval pathway.
Shirley, Matt