FDA Grants Accelerated Approval to Forzinity®, First Treatment for Rare Barth Syndrome

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Forzinity (elamipretide) injection, marking it as the first-ever treatment for Barth syndrome. This groundbreaking approval is for patients weighing at least 30 kg and represents a significant step forward for individuals living with this rare, serious, and life-threatening mitochondrial disease.

Understanding Barth Syndrome and Forzinity’s Impact

Barth Syndrome: Primarily affecting males, Barth syndrome typically manifests with severe heart failure in infancy and can lead to premature death. Survivors often experience chronic fatigue, poor stamina, and severe exercise intolerance, significantly impacting their quality of life.
Mechanism of Action: Forzinity works by directly targeting the inner part of the mitochondria, the cell’s energy-producing components. By binding to this area, the drug is designed to improve mitochondrial structure and function, thereby addressing the root cause of the disease.
Accelerated Approval Basis: The accelerated approval was based on evidence of improved strength in the quadriceps muscle, which is used to straighten the leg at the knee. The FDA considers this a measure reasonably likely to predict clinical benefit, such as improved ability to stand or walk. As a condition of this approval, Stealth Biotherapeutics Inc. is required to conduct a post-approval randomized, double-blind, placebo-controlled trial to confirm these patient benefits.

Administration and Side Effects

Forzinity is administered once daily via subcutaneous (under the skin) injection. Clinical trials identified the most common side effects as mild-to-moderate injection site reactions. However, serious reactions have also been reported.

Regulatory Designations

The application for Forzinity received priority review, and the drug was granted both a rare pediatric disease designation and a rare pediatric disease priority review voucher by the FDA to Stealth Biotherapeutics Inc., highlighting the urgent unmet medical need addressed by this therapy.