Evidence Grade A — Regulatory approved. 187 published studies. 21 registered clinical trials.
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Elamipretide (sold as Forzinity) is the first treatment specifically approved for Barth syndrome, an ultra-rare genetic disorder that affects the energy-producing structures (mitochondria) in cells, causing muscle weakness, heart problems, and fatigue. Given as a daily injection, it is also the first mitochondria-targeted therapy to gain regulatory approval. Barth syndrome affects approximately 150 people in the US.
Elamipretide is also known by these brand and alternate names:
187 published studies: 84 human, 48 animal, 15 in-vitro, 50 reviews
Elamipretide (Forzinity) was approved by the FDA for Barth syndrome based on the TAZPOWER trial. The randomised crossover phase (12 patients) did not meet its primary endpoints, but the open-label extension (168 weeks) demonstrated durable improvements in walking distance and muscle strength that formed the basis for approval.
Barth syndrome affects approximately 1 in 300,000–400,000 births. A larger Phase III trial in primary mitochondrial myopathy (218 patients, MMPOWER-3) did not meet its primary endpoint, and the drug was not approved for that broader indication. Elamipretide remains approved exclusively for Barth syndrome. See also SS-31 (#158) for the research compound context.
Elamipretide selectively binds cardiolipin in the inner mitochondrial membrane, stabilising the membrane structures (cristae) that are essential for energy production. In Barth syndrome, a genetic defect causes cardiolipin abnormalities that disrupt mitochondrial energy production. Elamipretide directly addresses this structural defect, restoring mitochondrial function at its physical foundation.
Forzinity received FDA accelerated approval in September 2025 based on data from the TAZPOWER trial. The evidence is limited by the ultra-rare disease context — only 12 patients were enrolled in the randomised trial, which did not meet its primary endpoints for walking distance or fatigue scores. However, during the open-label extension period (where patients knew they were receiving the drug), improvements in knee extensor muscle strength were observed over 168 weeks. More than half of the original trial patients continue on treatment after more than eight years. The approval pathway was notable for its challenges: the initial application was refused, a second submission received a complete response rejection in May 2025, and a third submission targeting accelerated approval based on muscle strength was finally accepted in September 2025. A larger trial in primary mitochondrial myopathy (a more common condition) failed to meet its primary endpoint, and the drug was not approved for that broader use. Continued approval may depend on a confirmatory trial. Injection site reactions occur in virtually all patients.
PeptideTrace tracks 21 registered clinical trials for Elamipretide sourced from ClinicalTrials.gov.
An Intermediate Size Expanded Access Protocol of Elamipretide
Clinical Trial in Patients With Barth Syndrome- 4TAZPower
Study of Healthy Aging and Physical Function With Elamipretide
ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
FDA ORIG 1
Elamipretide (brand name FORZINITY) is a cell-permeable mitochondria-targeted tetrapeptide with the sequence D-Arg-Dmt-Lys-Phe-NH2 (4 amino acids), developed by Stealth BioTherapeutics. Molecular weight 639.8 Da (free base); 749.2 Da as HCl salt. CAS number: 736992-21-5. This entry covers the FDA-approved commercial formulation. Elamipretide received FDA accelerated approval on September 19, 2025 for improving muscle strength in adults and pediatric patients with Barth syndrome weighing at least 30 kg, making it the first FDA-approved mitochondria-targeted therapeutic. Dosing: 40 mg subcutaneous injection daily.
Elamipretide selectively and reversibly binds cardiolipin on the mitochondrial inner membrane, stabilizing cristae structure and respiratory chain supercomplexes. This improves electron transport chain efficiency, enhances oxidative phosphorylation coupling, increases ATP production, and reduces pathological reactive oxygen species formation. In Barth syndrome, caused by TAFAZZIN gene mutations leading to defective cardiolipin remodeling, elamipretide directly ameliorates the fundamental bioenergetic defect.
The TAZPOWER pivotal trial (NCT03098797; N=12; randomized crossover plus 168-week open-label extension) formed the basis for FDA approval. The randomized 12-week crossover phase did not meet its primary endpoints for 6-minute walk test or fatigue score. However, the open-label extension demonstrated durable improvements: knee extensor strength increased by more than 45% (median +63 newtons at week 168), 6-minute walk test improved by +96.1 meters representing approximately 60% improvement (P=0.003), and left ventricular volumes improved by more than 40% indicating reverse cardiac remodeling. Natural history comparison study SPIBA-001 (N=8 versus 19 controls) showed 6MWT difference of 79.7 meters at week 64 (P=0.0004).
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