Is Elamipretide legal in the US?
Yes, FDA approved for muscle strength in Barth syndrome, on a conditional basis.
Approved under the accelerated pathway. On September 19, 2025 the FDA approved elamipretide as Forzinity (Stealth BioTherapeutics) to improve muscle strength in adults and children weighing at least 30 kg who have Barth syndrome, a rare mitochondrial disease. The approval rests on a surrogate measure, knee extensor strength, and a confirmatory trial is required. It is not approved for any other condition.
Yes, for one use. Forzinity received accelerated approval in September 2025. It is approved to improve muscle strength in people with Barth syndrome who weigh at least 30 kg. The approval depends on a confirmatory trial. 2
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Citations
- Thompson 2024, Genetics in Medicine: 168-week open-label extension of TAZPOWER, elamipretide in Barth syndrome doi.org
- Forzinity (elamipretide) prescribing information drugs.com
- 2025, Orphanet J Rare Dis: psychometric evaluation of the Barth Syndrome Symptom Assessment in the TAZPOWER trial pubmed.ncbi.nlm.nih.gov
- Karaa 2023, Neurology: efficacy and safety of elamipretide in primary mitochondrial myopathy (MMPOWER-3) pubmed.ncbi.nlm.nih.gov
Reference information only, not medical advice. Drawn from the Elamipretide article, fact-checked against the cited sources. No dosing anywhere on this site.