Elamipretide
Elamipretide (SS-31, mitochondrial-targeted peptide)
Also known as: SS-31, MTP-131
An investigational mitochondria-targeting peptide studied for rare mitochondrial disease and age-related organ decline.
Overview
Elamipretide concentrates in the inner mitochondrial membrane by binding cardiolipin, aiming to restore efficient energy production. It has been studied in clinical trials for primary mitochondrial myopathy, Barth syndrome, and dry age-related macular degeneration, with mixed results. It is investigational and not approved. Update, September 2026: elamipretide received FDA accelerated approval on September 19, 2025 as Forzinity for Barth syndrome in patients weighing 30 kg or more, based on a muscle-strength endpoint with a confirmatory trial required. The approval does not cover longevity, fatigue or the research-grade 'SS-31' sold online; Phase 3 trials continue in dry macular degeneration and mitochondrial myopathy.
Mechanism of action
Binds cardiolipin in the inner mitochondrial membrane, stabilizing cristae structure and improving electron transport efficiency and ATP production.
Key studies & citations
- Review2025
Forzinity (elamipretide) injection, NDA 215244 accelerated approval letter
FDA accelerated approval of Forzinity (elamipretide) to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg, under section 506(c) with post-marketing confirmation required.
U.S. Food and Drug Administration - Human2023
Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial
Phase 3, 218 participants: 24 weeks of elamipretide 40 mg/day did not improve 6-minute walk distance or fatigue versus placebo (Class I evidence), though it was well tolerated.
Neurology - Human2024
Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Ten Barth syndrome patients in the open-label extension: 6-minute walk distance improved cumulatively by 96.1 m at week 168 with injection-site reactions the most common adverse event.
Genetics in Medicine - Animal2013
The mitochondrial-targeted compound SS-31 re-energizes ischemic mitochondria by interacting with cardiolipin
SS-31 binds cardiolipin on the inner mitochondrial membrane with high affinity, protecting cristae and accelerating ATP recovery after renal ischemia in rats.
Journal of the American Society of Nephrology
Frequently asked questions
Is elamipretide approved for any condition?
Yes, for one condition. The FDA granted accelerated approval to Forzinity (elamipretide) in September 2025 for Barth syndrome. Every other use, including mitochondrial myopathy, heart failure and aging, remains investigational.