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FDA approved for specific usesModerate human evidence

Plain-English fact sheet

Elamipretide

Also known as Forzinity, SS-31, MTP-131, Bendavia

Mitochondrial healthMuscle and performanceRare and genetic conditions

Elamipretide is the active ingredient in Forzinity, which received accelerated FDA approval for improving muscle strength in certain people with Barth syndrome.

Quick answer

Elamipretide is FDA approved only as Forzinity to improve muscle strength in adults and children with Barth syndrome who weigh at least 30 kilograms. That approval does not establish a general mitochondrial, energy, anti-aging, or muscle-performance therapy.

By the PeptideFactSheets Editorial Team. Claims are source-checked under our editorial policy; clinician review is identified only when a named reviewer is shown.

What is Elamipretide?

Elamipretide is a small manufactured peptide that binds cardiolipin, a lipid in the inner mitochondrial membrane.

Forzinity received accelerated approval in 2025 based on improved knee-extensor muscle strength, an intermediate endpoint considered reasonably likely to predict clinical benefit.

Why are people interested in it?

It is the first FDA-approved treatment for Barth syndrome and one of the clearest examples of a mitochondria-targeting peptide reaching regulatory approval.

Broader mitochondrial-disease, heart, eye, fatigue, and healthy-aging claims remain separate research questions; a Phase 3 primary mitochondrial myopathy trial did not meet its primary endpoints.

Current regulatory status

FDA approved for specific uses

FDA granted accelerated approval to Forzinity on September 19, 2025, for improving muscle strength in adults and children with Barth syndrome weighing at least 30 kilograms. Continued approval may depend on a confirmatory trial.

What is it approved for?

  • Improving muscle strength in adults and children with Barth syndrome who weigh at least 30 kilograms under the Forzinity label

What is it being studied for?

Barth syndrome
Primary mitochondrial myopathy
Mitochondrial function and muscle energetics
Heart and eye disorders in clinical research

Investigational areas

  • Other mitochondrial diseases and genetically defined mitochondrial myopathies
  • Cardiac, ophthalmic, and muscle-function outcomes outside the approved indication

Evidence snapshot

Moderate human evidence

The approved claim rests on a very small rare-disease program and an intermediate endpoint. Evidence should not be generalized to other mitochondrial disorders or consumer performance goals, especially because MMPOWER-3 was negative on its primary endpoints.

Potential benefits being researched

  • The approval program observed improvement in knee-extensor muscle strength in a small group with Barth syndrome.
  • The larger MMPOWER-3 trial in primary mitochondrial myopathy did not show benefit over placebo for walking distance or fatigue at 24 weeks.

Potential does not mean proven. Study design, population, endpoint, and regulatory review matter.

Known or possible risks

  • The current label identifies injection-site reactions as the most common adverse reactions.
  • The formulation contains benzyl alcohol and carries a warning against use in neonates.
  • Evidence from a small rare-disease population cannot characterize every uncommon or long-term harm.

What we still do not know

  • Whether confirmatory research will verify a patient-centered clinical benefit in Barth syndrome
  • Which, if any, other genetically defined mitochondrial disorders benefit
  • Long-term safety across broader populations
  • Whether laboratory or muscle-energy effects translate into meaningful outcomes outside the approved use

Plain-English takeaway

Elamipretide is a landmark mitochondrial peptide, but its accelerated approval is narrow and based on an intermediate endpoint. It is not proof of a general mitochondrial boost or performance benefit.

Research and reference links

Use these primary and reputable sources to verify status and read beyond this summary. Trial registries may list studies without proving a benefit.

  1. 1
    FDA prescribing information: Forzinity

    Current approval scope, accelerated-approval basis, warnings, and adverse reactions.

  2. 2
    FDA: accelerated approval for Barth syndrome

    FDA announcement describing the September 2025 decision and required confirmatory research.

  3. 3
    MMPOWER-3 randomized clinical trial

    Phase 3 trial that did not meet its primary walking-distance or fatigue endpoints in primary mitochondrial myopathy.