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 genetic disorder that disrupts energy production in cells, leading to muscle weakness and potentially life-threatening complications. Forzinity (elamipretide) is a peptide that stabilizes the inner membrane of mitochondria, the cell's energy factories, by binding to a key lipid called cardiolipin. By supporting mitochondrial function, Forzinity may improve muscle strength and help manage the symptoms of this complex condition.
