About Elamipretide

Mechanism of Action: A mitochondria-targeted tetrapeptide that binds cardiolipin in the inner mitochondrial membrane, stabilizing mitochondrial structure, improving electron transport chain efficiency, and reducing oxidative stress.

Indications & Off-Label Uses: Investigated for primary mitochondrial myopathies, Barth syndrome, and heart failure. Off-label/experimental interest includes improving muscle endurance, energy, and longevity though human data are limited.

FDA Status: Not FDA-approved for any indication at this time; clinical trials are ongoing.

Clinical Evidence: Phase II/III trials report improvements in exercise tolerance, cardiac and renal function in mitochondrial disorders and heart failure. Data supporting anti-aging or performance enhancement are preliminary.

Safety & Monitoring: Generally well tolerated; mild adverse effects include headache, dizziness, and injection-site reactions. Long-term safety in healthy adults is unknown.