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Peptides for mitochondrial disease and dysfunction: what the evidence shows

By the PeptideAgent Editorial Team. Draft, pending editorial review.  Last verified

At a glance

Elamipretide (SS-31) received FDA accelerated approval in September 2025 for Barth syndrome, a rare inherited mitochondrial disease, but it missed its primary endpoints in the larger MMPOWER-3 trial in primary mitochondrial myopathy. MOTS-c and humanin are mitochondrial derived peptides with animal data only, and NAD+ precursors raise NAD+ levels in people without proven clinical benefit.

Evidence: Strongest: Human RCT evidence4 peptides gradedVerified: Verified Sep 27, 2026

Do peptides help with mitochondrial disease and dysfunction?

4 peptides are graded for mitochondrial disease and dysfunction on this page. The strongest grade is human RCT evidence, for SS-31 (elamipretide) and NAD+. Each one is weighed against the standard of care below, and the evidence table gives the grade and a note for every peptide.

What is the standard of care for mitochondrial disease and dysfunction?

Genetic diagnosis at a specialist center; supportive care for affected organs (heart, muscle, eyes, hearing, endocrine); exercise as tolerated; avoid mitochondrial toxic drugs, such as valproate in POLG disease; supplements such as coenzyme Q10 are commonly used despite limited evidence.

Any peptide below should be judged against this baseline, not against doing nothing. Talk to a licensed provider before replacing or adding to an established treatment.

Which peptides have been studied for mitochondrial disease and dysfunction?

Ordered by strength of evidence for this use. Grades follow the methodology; each peptide page lists the individual studies.

Peptides studied for Mitochondrial disease and dysfunction with evidence grades
PeptideEvidence for this useNote
SS-31 (elamipretide)Evidence: Human RCT evidenceBarth syndrome crossover missed its primary endpoints, with gains in walking distance and strength in the open label extension; MMPOWER-3 (n = 218) was negative; FDA approved as Forzinity for Barth syndrome. [1] [2] [3]
NAD+Evidence: Human RCT evidenceOral NR 1,000 mg daily raised blood NAD+ by about 60% in older adults; no mitochondrial disease outcome trial. [6]
MOTS-cEvidence: Animal-only evidenceImproved metabolism and physical capacity in mice; human data measure natural levels after exercise only. [4]
HumaninEvidence: Animal-only evidenceExtended lifespan in worms and improved metabolic healthspan in mice; human data are associations with age only. [5]

Head-to-head comparisons

Cost and access

Price ranges, lawful channels, and insurance notes for peptides graded here. State rules are in the legal guides by state.

Frequently asked questions

Is any peptide FDA approved for mitochondrial disease and dysfunction?

SS-31 (elamipretide) is an FDA approved drug, but an approval covers only the indications on that label, so check the label before assuming it applies to mitochondrial disease and dysfunction. The other peptides graded here are not FDA approved. [1] [2] [3] [7]

Has any peptide been tested in a randomized trial for mitochondrial disease and dysfunction?

Yes, for SS-31 (elamipretide) and NAD+. Check each note for the population and endpoints: a randomized trial in a related group or on a surrogate marker is weaker than one on the outcome you care about. [1] [2] [3] [6]

Do any peptides boost energy?

No peptide has been shown to improve energy or fatigue in a randomized trial. In MMPOWER-3 (n = 218), elamipretide (SS-31) did not improve fatigue scores or walking distance versus placebo over 24 weeks in primary mitochondrial myopathy. MOTS-c and humanin, often marketed for energy, have animal data only, and NAD+ precursors raise blood NAD+ levels without a trial showing more energy. [2] [4] [5] [6]

Sources

  1. [1]A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome (TAZPOWER)PubMed 33077895, 2021
  2. [2]Efficacy and safety of elamipretide in individuals with primary mitochondrial myopathy: the MMPOWER-3 randomized clinical trialPubMed 37268435, 2023
  3. [3]FDA prescribing information for Forzinity (elamipretide) injection, via DailyMedFDA, 2025
  4. [4]The mitochondrial-derived peptide MOTS-c promotes metabolic homeostasis and reduces obesity and insulin resistance (Cell Metab 2015)PubMed 25738459, 2015
  5. [5]The mitochondrial derived peptide humanin is a regulator of lifespan and healthspanPubMed 32575074, 2020
  6. [6]Chronic nicotinamide riboside supplementation is well-tolerated and elevates NAD+ in healthy middle-aged and older adultsPubMed 29599478, 2018
  7. [7]Drugs@FDA: FDA-approved drugs databaseFDA