Elamipretide
Also known as: Bendavia, D-Arg-2',6'-Dmt-Lys-Phe-NH2, Elamipretide, MTP-131, SS-31, SS‑31
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Summary
Elamipretide (also known as SS‑31, MTP‑131, Bendavia) is an investigational synthetic tetrapeptide that targets mitochondria. It is being studied for conditions linked to mitochondrial dysfunction such as Barth syndrome, heart failure, neurodegeneration, metabolic disorders, and retinal diseases like age‑related macular degeneration. The compound is administered by injection (intravenous or subcutaneous) and remains under clinical investigation, with no regulatory approval for any indication.
Mechanism of Action
Elamipretide is a mitochondria‑targeting tetrapeptide that selectively binds cardiolipin, a phospholipid unique to the inner mitochondrial membrane. By anchoring to cardiolipin, it stabilises cristae architecture, reduces lipid peroxidation, lowers reactive oxygen species production, and improves the efficiency of oxidative phosphorylation, leading to increased ATP generation. These actions collectively enhance mitochondrial bioenergetics and protect cells from stress‑induced injury.
What the Research Shows
Preclinical work across heart‑failure, neurodegeneration, ischemia‑reperfusion, metabolic syndrome, and muscle‑wasting models shows that elamipretide can preserve mitochondrial structure, lower oxidative stress and improve functional outcomes. Early‑phase clinical trials such as PROGRESS‑HF, MMPOWER‑3 and ReCLAIM have explored its potential, but the most detailed human data come from the TAZPOWER programme in Barth syndrome. In a 168‑week open‑label extension, daily subcutaneous 40 mg dosing was well tolerated and yielded significant gains in six‑minute walk distance, reduced fatigue, and modest improvements in left‑ventricular volumes, alongside favorable shifts in cardiolipin biomarkers. Review articles also list elamipretide among emerging therapies for retinal disease and age‑related macular degeneration, although efficacy in these ophthalmic settings remains untested in humans.
Reported Benefits
Evidence from the Barth‑syndrome extension suggests elamipretide can improve exercise capacity, lessen fatigue, and modestly enhance cardiac chamber dynamics after prolonged treatment. Preclinical studies indicate broad cytoprotective effects against mitochondrial injury, supporting its theoretical benefit in a range of mitochondrial‑related disorders. The peptide’s ability to restore cardiolipin balance may also address oxidative damage implicated in age‑related macular degeneration and muscle wasting.
Limitations of the Evidence
Human data are limited to a small cohort of Barth‑syndrome patients and early‑phase trials; efficacy in heart failure, neurodegeneration, or ocular diseases has not been demonstrated in large, controlled studies. The compound remains investigational with no approved indication, and long‑term safety beyond the reported open‑label period is unknown. Many of the cited benefits derive from animal models, which may not translate to clinical outcomes.
Safety Considerations
In the 168‑week open‑label study, elamipretide was generally well tolerated, with injection‑site reactions reported as the most frequent adverse event. No serious systemic toxicities were noted, but the limited sample size precludes a comprehensive safety profile. Review of peptide use in sports medicine highlights a paucity of rigorous human safety data for elamipretide, underscoring the need for further monitoring in larger trials.
How It Is Administered
Elamipretide is administered by injection, either intravenously or subcutaneously. Clinical investigations have used a daily subcutaneous dose of 40 mg, while formulation strategies for ocular delivery (e.g., sustained‑release implants) are being explored in review literature. No oral or topical formulations have been reported in the cited sources.
Routes of Administration
Goals & Uses
- Heart failureCardiovascularModerate
- Age‑related macular degenerationOphthalmologyLow
- Ischemia‑reperfusion injuryTissue ProtectionModerate
- Reduction of oxidative stressAntioxidant / CytoprotectionModerate
- mitochondrial diseaseGeneticModerate
- Mitochondrial dysfunction improvementBioenergeticsModerate
- Barth syndrome treatmentRare Genetic DiseaseModerate
- Heart failure with preserved ejection fraction (HFpEF)CardiovascularModerate
- Renal ischemia-reperfusion protectionNephroprotectionModerate
- Primary mitochondrial myopathyNeuromuscular DiseaseModerate
Contraindications
- Severe hepatic impairmentOrganLowLiver function concerns
- PregnancyPopulationModeratePotential fetal risk or insufficient safety data
- Known hypersensitivity to elamipretide or excipientsAllergyHigh
Adverse Effects
- Injection site reactionsLocalCommon
- Injection site painLocalCommonPain at the injection site
- HeadacheNeurologicUncommonPain in the head or upper neck
- Hypersensitivity reactionImmunologicRare
- NauseaGastrointestinalUncommonFeeling of sickness or urge to vomit
- FatigueGeneralUncommonLow energy or tiredness
Drug Interactions
- CyclosporineModerate
- ImmunosuppressantsLowPotential interaction with immune pathways or infection risk
- Anticoagulants (e.g., warfarin, heparin)Low
Population Constraints
- Severe renal impairmentOrgan ImpairmentRelative
- Pediatric patientsAgeRelative
- Pediatric (<12 y)AgeRelative
- Pregnant or breastfeeding womenReproductiveRelative
Regulatory Status
- European UnionInvestigationalUnder clinical evaluation; not yet authorized.
- United StatesInvestigationalPhase II/III trials ongoing for mitochondrial disease and heart failure.
- United KingdomInvestigationalNo approval; investigational status following EMA framework pre-Brexit alignment.
Not approved in any major jurisdiction; currently in Phase II/III clinical trials for several indications.
Evidence & Sources
- Journal ArticleModerateRowe LW, et al.2025-01-01T00:00:00.000000Z
- Journal ArticleModerateTung C, et al.2025-01-01T00:00:00.000000Z
- Journal ArticleModerateThompson WR, et al.2024-01-01T00:00:00.000000Z
- Journal ArticleModerateMendias CL, Awan TM2026-01-01T00:00:00.000000Z
- Journal ArticleModerateNashine S2021-01-01T00:00:00.000000Z
Frequently Asked Questions
What condition has the strongest clinical evidence for elamipretide?
The longest‑term human data come from the TAZPOWER open‑label extension in Barth syndrome, where daily subcutaneous dosing improved walking distance, fatigue scores, and cardiac volumes over 168 weeks.
How does elamipretide reach the mitochondria?
The tetrapeptide contains positively charged residues that drive its electrophoretic uptake into cells and selective binding to cardiolipin in the inner mitochondrial membrane, anchoring the drug where it can stabilise cristae.
Is elamipretide approved for any medical use?
No. Elamipretide remains an investigational peptide with no regulatory approval for any indication as of the latest literature.
What are the common side effects reported so far?
Injection‑site reactions such as redness, pain or swelling have been the most frequently reported adverse events; serious systemic side effects have not been observed in the limited studies.
Can elamipretide be used for eye diseases like AMD?
Elamipretide is listed as a potential therapeutic candidate for age‑related macular degeneration based on its mitochondrial protective properties, but no clinical trials in humans have yet demonstrated efficacy for ocular conditions.
What is Elamipretide?
Elamipretide (also known as SS‑31, MTP‑131, Bendavia) is an investigational synthetic tetrapeptide that targets mitochondria. It is being studied for conditions linked to mitochondrial dysfunction such as Barth syndrome, heart failure, neurodegeneration, metabolic disorders, and retinal diseases like age‑related macular degeneration. The compound is administered by injection (intravenous or subcutaneous) and remains under clinical investigation, with no regulatory approval for any indication.
What is Elamipretide used for?
Elamipretide is educationally associated with: Heart failure, Age‑related macular degeneration, Ischemia‑reperfusion injury, Reduction of oxidative stress, mitochondrial disease, Mitochondrial dysfunction improvement, Barth syndrome treatment, Heart failure with preserved ejection fraction (HFpEF), Renal ischemia-reperfusion protection, Primary mitochondrial myopathy. Educational only — not medical advice.
How is Elamipretide administered?
Recorded routes of administration: Intravenous, Subcutaneous.
What are the potential side effects of Elamipretide?
Reported adverse effects include: Injection site reactions, Injection site pain, Headache, Hypersensitivity reaction, Nausea, Fatigue. This list is not exhaustive — consult a qualified clinician.
Who should avoid Elamipretide?
Recorded contraindications: Severe hepatic impairment, Pregnancy, Known hypersensitivity to elamipretide or excipients. Consult a qualified clinician before use.