FDA Approves First-Ever Treatment for Rare Barth Syndrome

The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Forzinity (elamipretide) injection, marking a significant milestone as the first treatment available for Barth syndrome in patients weighing at least 30 kilograms. This rare and potentially fatal genetic disorder affects the mitochondria, the energy-producing powerhouses within cells, and primarily impacts males.

Barth syndrome is characterized by severe heart failure in infancy, often leading to premature death. Survivors may experience debilitating fatigue, reduced stamina, and exercise intolerance throughout their lives, significantly impacting their quality of life and daily activities. Forzinity functions by targeting the inner mitochondrial membrane, aiming to enhance both the structure and function of these vital cellular components. The FDA’s decision for accelerated approval was based on observed improvements in the strength of the quadriceps muscle, which is responsible for straightening the leg at the knee. This improvement is considered a reasonable indicator of potential patient benefit, such as enhanced mobility and the ability to walk longer distances.

As a condition of this accelerated approval, the manufacturer of Forzinity is mandated to conduct a subsequent randomized, double-blind, placebo-controlled trial. The purpose of this post-approval study is to definitively confirm whether the observed gains in knee muscle strength translate into tangible benefits for patients. Forzinity is administered once daily via subcutaneous injection, meaning it is injected under the skin. Clinical trials identified injection site reactions, ranging from mild to moderate, as the most common side effects. However, the trials also reported instances of serious reactions associated with the drug.

The development and review of this medication were expedited through the FDA’s priority review pathway. Furthermore, Forzinity received a rare pediatric disease designation, recognizing its potential to address a critical unmet need in children. Stealth Biotherapeutics Inc. received the accelerated approval for Forzinity, along with a rare pediatric disease priority review voucher. This voucher can be utilized by the company to expedite the review of a future drug application. Patients and healthcare providers seeking more information can contact the FDA’s consumer inquiries at 888-INFO-FDA.

Article by Mel Anara, based upon information from the U.S. Food and Drug Administration.

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