WASHINGTON (WDNews) — The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Forzinity (elamipretide) injection, marking the first approved treatment for Barth syndrome, a rare and life-threatening mitochondrial disease that primarily affects males.
Barth syndrome often begins with severe heart failure in infancy and can cause premature death. Survivors into adolescence and adulthood typically face fatigue, poor stamina, and exercise intolerance, affecting daily life and overall health.
“Forzinity works by binding to the inner part of the mitochondria, improving mitochondrial structure and function,” FDA officials said. The drug is given as a once-daily injection under the skin.
The approval was based on trial data showing improved strength in the muscle used to straighten the knee, which FDA considers reasonably likely to predict patient benefits such as standing or walking farther. As part of its accelerated approval, the manufacturer must complete a post-approval, placebo-controlled trial to confirm patient benefit.
“The FDA remains committed to facilitating the development of effective and safe therapies for rare diseases and will continue to work diligently to help ensure patients with rare diseases have access to innovative treatments,” said Dr. George Tidmarsh, Director of FDA’s Center for Drug Evaluation and Research.
Forzinity’s most common side effects are mild-to-moderate injection site reactions, though some serious reactions have also been reported. The drug received priority review and rare pediatric disease designation.
