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 serious genetic disorder that weakens the heart and skeletal muscles and can be life-threatening. Forzinity (elamipretide) is a small protein designed to stabilize the inner membrane of cellular power plants called mitochondria. By supporting mitochondrial health in muscle cells, this drug may improve muscle strength for those affected by the condition.
