Peptide library / Heart & blood / Elamipretide
Elamipretide
A four amino acid peptide that targets mitochondria, approved in 2025 for Barth syndrome.
Effects studied or claimed
What it is used or promoted for, and where each effect has actually been seen
- Barth syndromeFDA approved
- Primary mitochondrial diseaseSmall human studies
- Heart failureSmall human studies
How it is thought to work
Elamipretide binds cardiolipin in the inner mitochondrial membrane and stabilizes it, which may improve energy production.
What the evidence shows
Trials in Barth syndrome were small. Larger trials in heart failure and mitochondrial myopathy had mixed results.
Safety and unknowns
Injection site reactions are common.
Regulatory status
Received FDA accelerated approval for Barth syndrome in 2025 on the basis of small studies.
Regulatory status changes quickly. This entry was last reviewed on 27 October 2026. Confirm current status with the FDA before relying on it.
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General information, not medical advice. Do not buy or inject unregulated peptides.