Elamipretide hydrochloride is an investigational mitochondria-targeted peptide. Here's how it works, what conditions it targets, its FDA status, and cost.
Elamipretide hydrochloride is a synthetic, mitochondria-targeted tetrapeptide being studied for rare mitochondrial diseases, heart failure, and certain eye conditions. It is not approved by the FDA for any human use in the United States, so it cannot be prescribed outside of a clinical trial. Nearly all human data on the peptide come from studies that used daily subcutaneous injections.
Elamipretide Hydrochloride: The Basics
People searching what is elamipretide usually want to know whether it is a medicine, a dietary supplement, or a laboratory chemical. The accurate answer is that it is an investigational drug: a four-amino-acid peptide developed by Stealth BioTherapeutics and also known by the research codes SS-31 and MTP-131.
The word "hydrochloride" in the name refers only to the salt form, which manufacturers use to improve stability and handling. The active peptide is small for a biologic, with a molecular weight of roughly 640 g/mol and the formula C32H49N9O5.
Its amino acid sequence is D-arginine, 2',6'-dimethyltyrosine, lysine, and phenylalanine, capped with an amide group. Those unusual D-arginine and dimethyltyrosine residues help the peptide cross cell membranes and concentrate inside mitochondria.
Elamipretide Mechanism of Action
The elamipretide mechanism of action does not involve a receptor or an enzyme. The peptide instead binds cardiolipin, a phospholipid found almost exclusively on the inner mitochondrial membrane.
Cardiolipin helps organize the membrane's folds and supports the protein complexes of the electron transport chain. When cardiolipin is damaged by oxidative stress, energy production falls and mitochondria release more reactive oxygen species.
Elamipretide appears to stabilize cardiolipin, which may preserve electron transport efficiency and limit oxidative damage in cell and animal models. That mechanism is well documented in the laboratory, but it has not produced consistent clinical benefit in people.
Conditions Studied in Clinical Trials
Elamipretide uses in human research have centered on conditions in which mitochondria cannot meet the body's energy demands. The table below summarizes the main programs and their publicly reported status.
| Condition | How It Was Given | Reported Status |
|---|---|---|
| Barth syndrome | Subcutaneous injection, 40 mg daily | Application received a Complete Response Letter; not approved |
| Primary mitochondrial myopathy | Subcutaneous injection, 40 mg daily | Phase 3 trial missed its primary endpoint |
| Leber's hereditary optic neuropathy | Subcutaneous injection | Early trials reported mixed visual outcomes |
| Dry age-related macular degeneration | Subcutaneous injection | Phase 2; results considered exploratory |
| Heart failure with preserved ejection fraction | Subcutaneous injection | Phase 2; no approval pathway announced |
Barth syndrome remains the most closely watched program because it is rare, serious, and has no approved therapy. Families in expanded-access programs have reported less fatigue and better muscle strength, but open-label reports are not the same as controlled evidence.
Results in primary mitochondrial myopathy were less encouraging. The Phase 3 MMPOWER-3 trial did not meet its primary endpoint, and later analyses have not changed that conclusion.
Dosage and Administration
In clinical trials, elamipretide was given as a subcutaneous injection once daily, most often at 40 mg. The peptide has a short half-life in blood, which is why researchers chose daily dosing instead of a longer interval.
Oral dosing has not been practical. Digestive enzymes break peptides apart and absorption through the gut is poor, so no pill or capsule version has reached human testing.
Injecting an unapproved peptide at home carries real risks, including infection, dosing errors, and reactions to impurities in the product. Anyone considering that route should talk with a licensed healthcare professional first.
Elamipretide FDA Approval Status
Elamipretide FDA approval has not been granted for any indication. The company's application for Barth syndrome received a Complete Response Letter, and an FDA advisory committee voted against approval after reviewing the data.
The peptide holds orphan drug and rare pediatric disease designations, which create financial incentives for development but do not make a product legal to sell. Additional controlled trials would be needed to address the agency's questions about durability and clinical meaningfulness.
Availability, Cost, and Safety
Because no approved product exists, there is no official elamipretide cost. Trial participants receive the drug at no charge, and health insurers do not cover an unapproved peptide.
Online vendors list elamipretide as a "research chemical" with prices that commonly range from about $50 to $200 per vial. These products are labeled not for human consumption, and their purity, sterility, and actual content are rarely verified by an independent laboratory.
| Factor | Trial Supply | Online Research Chemical |
|---|---|---|
| Legal status | Investigational; dispensed under a study protocol | Not approved for human use |
| Quality testing | Made under GMP with a certificate of analysis | Vendor claims; seldom independently verified |
| Dosing guidance | Physician-supervised, protocol-defined | None; the buyer assumes all risk |
| Typical user cost | Free to enrolled participants | Roughly $50 to $200 per vial |
Reported side effects in trials were mostly mild and included injection-site reactions, headache, nausea, fatigue, and dizziness. Long-term safety data remain limited because most studies lasted weeks to months rather than years.
No dietary supplement version of elamipretide exists, and no regulator has reviewed it for safety or effectiveness in humans.
Frequently Asked Questions
Is elamipretide FDA-approved?
No. Elamipretide is not FDA-approved for any human indication in the United States. The manufacturer's application for Barth syndrome received a Complete Response Letter, and an FDA advisory committee voted against approval after reviewing the data, so the peptide remains investigational.
What is elamipretide used for?
Elamipretide has been studied for Barth syndrome, primary mitochondrial myopathy, Leber's hereditary optic neuropathy, dry age-related macular degeneration, and heart failure with preserved ejection fraction. None of these are approved uses, and several late-stage trials missed their primary endpoints. All human use so far has occurred inside clinical trials or expanded-access programs.
Can you buy elamipretide as a pill or supplement?
No. Elamipretide is a peptide drug candidate, not a dietary supplement, and no oral version has reached human testing because peptides are poorly absorbed and quickly broken down in the digestive tract. Products sold online as "elamipretide" are research chemicals labeled not for human consumption, with no guarantee of purity, sterility, or actual content.
This page provides educational research information and does not replace medical advice, diagnosis, or treatment.