FDA

FDA OKs first treatment for Barth syndrome

September 25, 2025

Brand name: Forzinity

Generic name: elamipretide

Manufacturer: Stealth BioTherapeutics

Approval date: September 19, 2025

FDA has granted accelerated approval to Forzinity (elamipretide HCl) to improve muscle strength in adult and pediatric patients with Barth syndrome weighing ≥30 kg (approx. 66 pounds).

Barth syndrome is a life-limiting pediatric mitochondrial cardioskeletal disease that affects approximately 150 individuals in the U.S.

Efficacy

Forzinity’s accelerated approval was based on data from a randomized, double-blind, placebo-controlled trial (TAZPOWER; NCT03098797) which showed improved knee extensor muscle strength. FDA considers this improvement reasonably likely to predict patient benefit, such as an ability to stand more easily or walk farther. As a condition of accelerated approval, FDA is requiring the manufacturer to conduct a post-approval randomized, double-blind, placebo-controlled trial to confirm that the changes seen on knee muscle strength translate into patient benefit.

Safety

The most common adverse reactions in the trial were mild-to-moderate injection site reactions.

Recommended dose

For patients weighing ≥30 kg, the recommended dosage of Forzinity is 40 mg SC once daily.

Sources:

FDA Grants Accelerated Approval to First Treatment for Barth Syndrome [News release]. 2025. https://www.fda.gov/news-events/press-announcements/fda-grants-accelerated-approval-first-treatment-barth-syndrome

Forzinity (elamipretide) [package insert]. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/215244s000lbl.pdf Revised September 2025. Accessed September 24, 2025.