FDA granted accelerated approval to Forzinity (elamipretide) on September 19, 2025 — just ~1 month after Class 2 resubmission acceptance, well ahead of the projected Feb 15 2026 PDUFA. First-ever approved therapy for Barth syndrome and the first FDA-approved therapy for any mitochondrial disease. Approval based on improved knee extensor muscle strength (intermediate clinical endpoint) from TAZPOWER trial.
About
Barth syndrome is a rare, serious genetic disorder that affects how the body handles fats, can harm multiple organ systems, and is sometimes fatal. Forzinity is a small protein-like drug that binds to cardiolipin, a fat in the inner mitochondrial membrane, helping steady the energy-producing machinery inside cells. Because Barth syndrome weakens muscles, stabilizing mitochondria may help preserve muscle strength in adults and children who weigh at least 30 kilograms.
