SS-31 (Elamipretide)
Stealth BioTherapeutics' mitochondria-targeted tetrapeptide. Binds cardiolipin in the inner mitochondrial membrane and improves electron transport chain efficiency. Multiple Phase 3 trials in genetic mitochondrial diseases (Barth syndrome, primary mitochondrial myopathy) with mixed regulatory outcomes.
Educational only — not medical advice. SmartPeptide does not prescribe, diagnose, or treat. Always consult a licensed healthcare provider before using any peptide, supplement, medication, or protocol.
What the research shows
Phase 3 trials in Barth syndrome and primary mitochondrial myopathy show meaningful improvements in muscle function in specific subgroups. Heart failure trials had mixed results. FDA approval status varies by indication and is evolving.
What's still experimental
Use outside FDA-recognized genetic mitochondrial diseases is experimental. Anti-aging / longevity applications are extrapolated from mechanism + animal data, not from RCTs in healthy adults.
Anecdotal / community reports
Longevity-focused communities discuss SS-31 for general mitochondrial support / age-related muscle decline. These uses are not supported by published RCTs in healthy adults.
Anecdotal reports are NOT scientific evidence. They reflect personal experience and may not generalize.
FDA approval status
Source: openFDA + DailyMed (NIH/NLM)Doses studied in research
No established dose rangeWhat published trials tested or FDA-approved labels specify. Reporting research facts — not a SmartPeptide recommendation.
This is the honest answer for peptides like BPC-157, IGF-1 LR3, MOTS-c, Epitalon and similar research compounds — robust human dose- ranging studies have not been published. Always work with a licensed clinician familiar with experimental peptides if you are considering any use.
FDA enforcement & recalls
Live · openFDA Drug Enforcement APIMechanism & targets
ChEMBL · UniProt · Open TargetsMolecule (ChEMBL)
View on ChEMBLLive research
PubMed · ClinicalTrials.gov · Europe PMC · OpenAlexClinical trials (ClinicalTrials.gov)
- Effect of Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Reduced Ejection FractionCOMPLETEDNCT02788747 · PHASE2 · n=71 · 2016-06
- A Study to Evaluate the Effects of 4 Weeks Treatment With Subcutaneous Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Preserved Ejection FractionCOMPLETEDNCT02814097 · PHASE2 · n=46 · 2016-09-02
- An Intermediate Size Expanded Access Protocol of ElamipretideAVAILABLENCT04689360 · N/A
- FRDA Investigator Initiated Study (IIS) With ElamipretideCOMPLETEDNCT05168774 · PHASE1, PHASE2 · n=20 · 2022-03-03
- Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)TERMINATEDNCT02976038 · PHASE2 · n=28 · 2016-12
Europe PMC — 355,994 additional records
Includes EU/UK studies and PubMed Central full-text articles. Often surfaces research weeks before PubMed indexes it.
- Severe anaemia and invasive bacterial infections in Kenyan children: a 26-year hospital surveillance observational studyAbuga KM, Muriuki JM, Mutua AM, et al. · 2027
- Identity without milestones: a narrative inquiry into the rites of passage shaping professional identity formation in health professions educatorsEnnab F, Kovacevic M, Guraya SS., et al. · 2026
- Pre-qualifications, learning strategies, and study satisfaction among medical students: insights from a multicenter German studySchröpel C, Herrmann-Werner A, Festl-Wietek T, et al. · 2026
- Gender equity and social inclusion in mass drug administration for neglected tropical diseases in post-conflict South SudanRenfroe SJ, Peter A, Pensotti C, et al. · 2026Open access
Research volume (OpenAlex topic graph)
Human clinical evidence
Semantic Scholar · AI TLDRs · influence-rankedMost influential human studies
- Elamipretide: A Review of Its Structure, Mechanism of Action, and Therapeutic PotentialReviewOpen access3 influential / 87 citedCheryl Tung, Fahimeh Varzideh, Emanuele Farroni · International Journal of Molecular Sciences · 2025
TLDR Current knowledge of the structure, mechanisms of action, and the promising therapeutic role of Elamipretide in stabilizing mitochondrial fitness, improving mitochondrial bioenergetics, and minimizing oxidative stress is synthesized.
- Cadmium-cardiolipin disruption of respirasome assembly and redox balance through mitochondrial membrane rigidificationJournal articleOpen accessN. Romanova, K. Sule, T. Issler · Journal of Lipid Research · 2025
TLDR A novel mechanism for Cd toxicity is reported in which Cd-CL interactions cause IMM rigidification, thereby disrupting correct SC assembly and increasing ROS.
- Restoring mitochondrial superoxide levels with elamipretide (MTP-131) protects db/db mice against progression of diabetic kidney diseaseJournal articleOpen accessS. Miyamoto, Guanshi Zhang, D. Hall · Journal of Biological Chemistry · 2020
TLDR It is found that 18-week-old db/db mice have reduced renal and cardiac superoxide levels, as measured by dihydroethidium oxidation, and increased levels of albuminuria, mesangial matrix accumulation, and urinary H2O2, and that MTP-131 protects against DKD and preserves physiologicalsuperoxide levels.
Research funding & verification
NIH RePORTER · CrossRef DOI registryPublication landscape
CrossRef · DOI registry- National Natural Science Foundation of China41,093 works
- National Institutes of Health7,683 works
- National Science Foundation6,426 works
- National Key Research and Development Program of China4,677 works
- Fundamental Research Funds for the Central Universities4,219 works
- National Research Foundation of Korea3,942 works
Funder diversity is a credibility signal. Research concentrated in a single drug company's funding warrants more scrutiny than research funded across NIH, charities, and academic grants.
Preprints — cutting edge
bioRxiv · medRxiv · via Europe PMCPreprints have NOT been peer-reviewed. They are early research shared by authors before formal validation. Treat findings as preliminary.
- PreprintMitochondrial-targeted therapy with elamipretide preserves cardiac function and prevents late mortality in murine polymicrobial sepsisVu J, Wagg CS, Holody C, et al. · 2026-07-09
- PreprintMaternal and perinatal outcomes among pregnant women with sickle cell disease in Uganda: a prospective cohort studyAkello J, Kiguli S, Ndeezi G, et al. · 2026-07-04
- PreprintSpatial Clusters and Predictors of Substance-Related Mortality in Iran: A Geographically Weighted Regression AnalysisBaberi F, Kavousi A, Kalan E, et al. · 2025-12-09
- PreprintPatterns of Dysglycemia Identified by Continuous Glucose Monitoring among Critically Ill Children in Malawi and BangladeshHarawa PP, Bourdon C, Khoshnevisan F, et al. · 2026-02-01
- PreprintPlasma proteome signatures in sickle cell anaemia and effect of hydroxyurea treatmentKumari N, Paliwal S, Umesh A, et al. · 2026-02-10
- PreprintUptake of early infant diagnosis and factors associated with its timely completion among HIV exposed infants at Lira Regional Referral Hospital: a retrospective cohort studyAwili R, Kalyango J, Puleh SS, et al. · 2026-03-02
Known risks
Injection-site reactions (notable in trials). Headache, GI symptoms. Long-term safety being characterized. FDA has issued complete-response letters on prior submissions — efficacy outside specific genetic indications remains debated.
Reported side effects
Injection-site erythema (common), headache, nausea, dizziness, fatigue.
FDA adverse event reports (FAERS)
Updated quarterly by FDAWhat requires medical supervision
Restricted to clinical trials or specific approved indications. Off-label use should only be under a clinician familiar with mitochondrial medicine.
Questions for your clinician
- Do I have any indication where SS-31 has demonstrated efficacy?
- What's the evidence vs. mechanism-based extrapolation distinction for my use case?
- Are there active trials I could enroll in?