Elamipretide
Elamipretide is designed to improve mitochondrial function by stabilising cardiolipin and the cristae architecture of the inner mitochondrial membrane. It has been evaluated across numerous conditions such as primary mitochondrial myopathy, Barth syndrome, Leber hereditary optic neuropathy, dry age-related macular degeneration and heart failure, with mixed results; its pivotal primary mitochondrial myopathy trial (MMPOWER-3) did not meet its primary endpoint. In September 2025 it received US FDA accelerated approval for Barth syndrome (as Forzinity) based on an improvement in knee-extensor muscle strength, with a confirmatory trial required; for all other indications it remains investigational.
Mechanism
In plain terms, elamipretide is a small peptide that concentrates inside mitochondria, the cell's energy-producing structures, and helps them make energy more efficiently while producing less oxidative stress. Technically, it is a cell-permeable, positively charged tetrapeptide (D-Arg-2',6'-dimethyltyrosine-Lys-Phe-NH2) that accumulates in the inner mitochondrial membrane and binds the phospholipid cardiolipin. By stabilising cardiolipin and cristae structure it is reported to improve electron-transport-chain efficiency and ATP production and to reduce reactive oxygen species, rather than acting on a conventional cell-surface receptor.
Regulatory Status by Region
- United States (FDA)Granted accelerated approval on September 19, 2025 as Forzinity (elamipretide HCl) to improve muscle strength in adult and paediatric patients with Barth syndrome who weigh at least 30 kg, based on an intermediate endpoint (knee-extensor muscle strength) with a confirmatory trial required. It is not approved for any other indication, and the pivotal primary mitochondrial myopathy trial (MMPOWER-3) did not meet its primary endpoint.
- Australia (TGA)Not on the ARTG; investigational only and not approved for supply.
- European Union (EMA)No EMA marketing authorisation; not authorised as a medicine in the EU.
- WADANot listed by name on the Prohibited List; athletes should verify current status with their anti-doping organisation.
Key Studies
- Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy (opens in a new tab) (Karaa A, Haas R, Goldstein A, Vockley J, Weaver WD, Cohen BH. Neurology. 2018;90(14):e1212-e1221. PMID 29500292)
- First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics (opens in a new tab) (Szeto HH. Br J Pharmacol. 2014;171(8):2029-2050. PMID 24117165)
Related Clinical Trials
- Clinical Trial in Patients With Barth Syndrome- 4TAZPowerPhase 4 · Recruiting
- Study of Healthy Aging and Physical Function With ElamipretidePhase 2 · Recruiting
- ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)Phase 3 · Active Not Recruiting
- Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)Phase 3 · Completed
- FRDA Investigator Initiated Study (IIS) With ElamipretidePhase 1/Phase 2 · Completed
- ReCLAIM-2 Study to Evaluate Safety,Efficacy & Pharmacokinetics of Elamipretide in Subjects With AMD With Non-central GAPhase 2 · Completed
- A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label ExtensionPhase 3 · Terminated
- A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth SyndromePhase 2/Phase 3 · Completed
Common Questions
- What is Elamipretide?
- Elamipretide (SS-31; formerly Bendavia) is a mitochondria-targeting tetrapeptide developed by Stealth BioTherapeutics that binds cardiolipin in the inner mitochondrial membrane to support energy production. In September 2025 the US FDA granted it accelerated approval, marketed as Forzinity (elamipretide HCl), to improve muscle strength in Barth syndrome patients who weigh at least 30 kg; it remains investigational for other conditions and is not approved by the EMA or Australia's TGA.
- Is Elamipretide approved for medical use?
- Elamipretide is approved for one or more medical uses in at least one major jurisdiction. United States (FDA): Granted accelerated approval on September 19, 2025 as Forzinity (elamipretide HCl) to improve muscle strength in adult and paediatric patients with Barth syndrome who weigh at least 30 kg, based on an intermediate endpoint (knee-extensor muscle strength) with a confirmatory trial required. It is not approved for any other indication, and the pivotal primary mitochondrial myopathy trial (MMPOWER-3) did not meet its primary endpoint. European Union (EMA): No EMA marketing authorisation; not authorised as a medicine in the EU. Australia (TGA): Not on the ARTG; investigational only and not approved for supply.
- How does Elamipretide work?
- In plain terms, elamipretide is a small peptide that concentrates inside mitochondria, the cell's energy-producing structures, and helps them make energy more efficiently while producing less oxidative stress. Technically, it is a cell-permeable, positively charged tetrapeptide (D-Arg-2',6'-dimethyltyrosine-Lys-Phe-NH2) that accumulates in the inner mitochondrial membrane and binds the phospholipid cardiolipin.
- Is Elamipretide legal in Australia?
- Elamipretide in Australia (TGA): Not on the ARTG; investigational only and not approved for supply.
- Is Elamipretide banned in sport?
- Elamipretide under the World Anti-Doping Agency (WADA) code: Not listed by name on the Prohibited List; athletes should verify current status with their anti-doping organisation.