WASHINGTON, D.C. — The U.S. Food and Drug Administration has granted accelerated approval to Forzinity (elamipretide) injection, the first treatment for Barth syndrome, a rare and life-threatening mitochondrial disease that primarily affects males.
The approval, announced this week, applies to patients weighing at least 30 kilograms and represents a milestone for a condition that has long lacked any approved therapies. Barth syndrome, which often presents as severe heart failure in infancy, causes premature death in many patients. Those who survive into adolescence and adulthood frequently suffer from fatigue, poor stamina, and exercise intolerance, with symptoms significantly impairing quality of life.
“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 George Tidmarsh, M.D., Ph.D., director of the FDA’s Center for Drug Evaluation and Research.
Forzinity works by binding to the inner mitochondrial membrane, where it improves mitochondrial structure and function. The FDA based its accelerated approval on trial data showing improved strength in the quadriceps muscle, which the agency said is reasonably likely to predict broader patient benefits such as improved mobility.
As a condition of approval, Stealth Biotherapeutics Inc., the drug’s manufacturer, will be required to conduct a post-approval, randomized, placebo-controlled trial to confirm that the observed muscle strength improvements translate into real-world functional gains for patients.
Forzinity is administered once daily by subcutaneous injection. The most common side effects observed in clinical trials were mild to moderate injection site reactions, though more serious adverse events have also been reported.
The FDA granted the drug priority review and rare pediatric disease designation. In addition to the approval, Stealth Biotherapeutics received a rare pediatric disease priority review voucher, which can be applied to speed the review of another therapy in the future.
The decision provides a long-awaited option for patients and families confronting the devastating effects of Barth syndrome and underscores the FDA’s broader push to advance treatments for rare diseases.
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