Mitochondria-targeted (cardiolipin-binding) tetrapeptide (elamipretide)
A peptide (also called elamipretide) that concentrates in mitochondria to protect and restore their energy-producing function, now an approved drug for a rare mitochondrial disease.
| Class / type | Mitochondria-targeted (cardiolipin-binding) tetrapeptide (elamipretide) |
|---|---|
| Category | Longevity & Cellular |
| Status (Jul 2026) | FDA-approved |
It selectively binds cardiolipin on the inner mitochondrial membrane, stabilizing membrane structure and the electron transport chain to improve ATP production and reduce reactive oxygen species.
FDA-approved (as FORZINITY) to improve muscle strength in Barth syndrome; in clinical trials for heart failure, primary mitochondrial myopathy, and dry age-related macular degeneration; also researched for aging.
First approved mitochondria-targeted therapeutic; clinical data support a muscle-strength benefit in Barth syndrome; broadly investigated for energy metabolism and mitochondrial dysfunction.
Several endpoints in prior trials (some heart-failure and myopathy studies) were mixed or negative; injection-site reactions reported; continued approval is contingent on a confirmatory trial; benefits outside Barth syndrome remain investigational.
Reported by patients and researchers as promising for mitochondrial energy support, with cautious optimism given ongoing trials.
FDA-approved: granted accelerated approval in September 2025 (marketed as FORZINITY) to improve muscle strength in Barth syndrome; all other uses remain investigational and continued approval depends on a confirmatory trial.