Elamipretide Reference

Reference summary

Human clinical trial data exist across several mitochondrial-disease programs, and the Barth-syndrome program supported the FORZINITY accelerated approval. The community longevity / mitochondrial / anti-aging research use that pepSmart describes falls outside that narrow approved indication; pepSmart displays elamipretide as trial-context and off-label educational content and does not imply approved use for those purposes.

Regulatory and posture

Categories

Longevity, Metabolic

Aliases

SS-31, MTP-131, Bendavia, D-Arg-Dmt-Lys-Phe-NH2

Evidence posture

human - FDA-approved only for Barth syndrome muscle weakness (FORZINITY, accelerated approval, September 2025). The broad primary-mitochondrial-myopathy program received a Complete Response Letter, and the longevity / research use described here is off-label and unvalidated.

Regulatory status

FDA-approved for a narrow rare-disease indication. Elamipretide (SS-31 / MTP-131), a cardiolipin-binding mitochondrial peptide developed by Stealth BioTherapeutics, received FDA accelerated approval as FORZINITY in September 2025 to improve muscle strength in adult and pediatric patients weighing at least 30 kg with Barth syndrome, the first approved therapy for that ultra-rare mitochondrial disease. Continued approval may be contingent on confirmatory-trial verification. The separate primary-mitochondrial-myopathy program received an FDA Complete Response Letter and is not approved.

Content review status

label verified

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