Scientific context only. Not medical advice, not a recommendation to use.
At a glance
Elamipretide (SS-31, MTP-131, formerly Bendavia) is a synthetic, mitochondria-targeting tetrapeptide (sequence D-Arg-Dmt-Lys-Phe-NH2) that binds cardiolipin on the inner mitochondrial membrane and is proposed to stabilise cristae structure and support mitochondrial bioenergetics. It was investigated clinically by Stealth BioTherapeutics across several indications, including primary mitochondrial myopathy, Barth syndrome, heart failure, and dry age-related macular degeneration (geographic atrophy). The trial record is mixed, with several pivotal studies missing their primary endpoints. In September 2025 elamipretide (brand name Forzinity) received accelerated FDA approval in the United States solely for the ultra-rare Barth syndrome; for all other investigated indications it remains investigational and it is not approved as a medicine outside the United States.
On 19 September 2025 the FDA granted accelerated approval to elamipretide hydrochloride (brand name Forzinity, Stealth BioTherapeutics) solely for the treatment of Barth syndrome in patients weighing at least 30 kg. For this single ultra-rare indication it is therefore an approved prescription medicine; continued approval is contingent on verification of clinical benefit in confirmatory trials. For all other investigated indications (e.g. mitochondrial myopathy, heart failure, dry AMD) elamipretide remains investigational and unapproved.
Elamipretide is a cell-permeable tetrapeptide with alternating aromatic and basic residues that selectively concentrates on cardiolipin — a phospholipid found almost exclusively in the inner mitochondrial membrane that is important for cristae curvature and the organisation of the respiratory-chain complexes. By binding cardiolipin, elamipretide is proposed to stabilise cristae architecture, support electron transport and ATP production, and reduce the formation of reactive oxygen species. These mechanistic models derive largely from cell and animal models and biophysical work; the extent to which they explain clinical efficacy in humans is, given the mixed trial results, a matter of ongoing research.
02
Evidence at a glance
Reading note. The distribution shows on which evidence tier each observation sits. Strong colours mark stronger evidence — weaker tiers are deliberately visible, not hidden.
4 observations · 3 tiers
Human RCT
2
Human trial
1
In vitro
1
03
What the studies show
In vitro
Isolierte Mitochondrien / Zellkultur
Karaa A, Bertini E, Carelli V, et al. 2023
Binding to cardiolipin on the inner mitochondrial membrane with stabilisation of cristae structure and supported bioenergetics
What does NOT follow: This is a molecular and cell-biology finding. Stabilisation of the mitochondrial membrane in a model does not establish clinical benefit in humans; several human trials missed their primary endpoints.
Human RCT
Mensch (randomisierte kontrollierte Studie)
Karaa A, Bertini E, Carelli V, et al. 2023
No statistically significant benefit over placebo on the primary endpoints (6-minute walk test, fatigue) in the phase 3 primary mitochondrial myopathy trial
What does NOT follow: MMPOWER-3 missed both primary endpoints in the overall population; a reported benefit in a subgroup (nDNA variants) comes from a post hoc analysis and is hypothesis-generating, not confirmatory.
Human trial
Mensch (Phase-2/3-Crossover, sehr kleine Fallzahl)
Reid Thompson W, Hornby B, Manuel R, et al. 2021
Signals of improved functional measures and cardiac function in Barth syndrome in a small controlled trial with subsequent open-label long-term extension
What does NOT follow: The randomised phase of TAZPOWER missed its primary endpoints; positive signals derive mainly from the uncontrolled open-label extension with a very small sample. The subsequent US approval was accelerated for an ultra-rare disease and is contingent on confirmatory studies.
No improvement in left ventricular end-systolic volume versus placebo in heart failure with reduced ejection fraction
What does NOT follow: The PROGRESS-HF trial missed its primary imaging endpoint; while the peptide was tolerated, a clinical benefit in heart failure was not demonstrated.
04
Where studies disagree
Open question
Does elamipretide have an established clinical benefit, and how does the US approval reconcile with missed endpoints in several trials?
POSITION A
The cardiolipin-binding rationale is mechanistically plausible, and in ultra-rare Barth syndrome signals from a small trial plus an open-label long-term extension led to a 2025 accelerated US approval (Forzinity) as the first therapy for this disease.
POSITION B
Several larger controlled trials (MMPOWER-3 in mitochondrial myopathy, PROGRESS-HF in heart failure) missed their primary endpoints; the Barth data come from a very small population and largely from uncontrolled phases. A broad, robustly established clinical benefit is not in evidence.
CURRENT STATE · As of 2026: elamipretide is approved in the United States on an accelerated basis solely for Barth syndrome, contingent on confirmatory studies; in all other indications and outside the United States it remains investigational. The overall evidence is mixed and insufficient for a broad assessment of benefit.
05
Pharmacokinetics
No robust pharmacokinetic human data available. A model curve is not invented.
06
Routes of administration in the literature
Which routes of administration the available studies describe — neutral reporting, not a usage guide.
Subcutaneous
In the pivotal clinical trials (MMPOWER-3, TAZPOWER) elamipretide was investigated as a once-daily subcutaneous administration. This describes study practice and is not a usage instruction.
Intravenous
In earlier cardiology studies (under the name Bendavia) elamipretide was also investigated as an intravenous infusion. This describes historical study practice and is not a usage instruction.
06d
Safer use & risks
Risk notes for harm reduction — descriptive, not a usage or dosing guide.
⚠ Important — please read
This platform does NOT provide usage or dosing instructions. The points below describe risks and are meant to help avoid harm — they do not replace medical advice. Anyone who uses a substance should discuss it with a doctor.
This substance is approved (in at least one country) — use belongs in medical hands, within the approved indication and a physician-set dose.
Online numbers are not a benchmark
Amounts from TikTok, YouTube and forums are mostly imitation rather than data — and are often wrongly derived from animal studies (µg/kg). Not a reliable benchmark for humans.
Sterility & infection risk
Injection solutions prepared or stored non-sterile carry an infection and abscess risk. Contamination is common with grey-market product.
Unknown product quality
Research-/grey-market product is not quality-tested: identity, purity and actual content are often unknown, and counterfeits occur.
Mind interactions
Combinations with medications or pre-existing conditions can carry risks (see the Interactions section). Clarify with a doctor beforehand.
Warning signs — seek medical help
With persistent pain, redness/swelling at the injection site, fever, shortness of breath, racing heart, chest pain or allergic reactions, seek medical help immediately.
A doctor, not a forum
Concrete questions about use and amount belong in a conversation with a doctor — not in a comment thread.
07
Known adverse events from studies
Factual reporting of what studies observed. Not a safety statement for individual use.
Human trial
Mild-to-moderate subcutaneous injection-site reactions as the most commonly reported adverse event in clinical trials
This description reflects the safety profile reported in the studies and is not a complete listing of possible risks. It is not a recommendation for use.
häufig in Studien berichtet
07b
Interactions & combinations
Documented interactions and contraindications from studies, prescribing information and guidelines. Where no data exists, this is stated.
Reporting of risks, NOT a combination guide. The absence of an entry does not mean „safe to combine“ but „not sufficiently studied“.
No documented interactions recorded
We have not yet found robustly documented interactions for this peptide. This does NOT mean none exist — the data is limited.
08
Risks & hygiene aspects in the literature
What regulatory and scientific literature reports on risks, sterility and identity in non-pharmaceutical sources — descriptive, not a hygiene guide.
Mixed evidence base despite accelerated approval in a niche indication
The accelerated US approval for Barth syndrome rests on data from a very small population, including substantial contributions from uncontrolled extension phases, and is contingent on confirmatory studies. Larger controlled trials in other indications (mitochondrial myopathy, heart failure) missed their primary endpoints. The narrow approval does not imply a broadly established benefit.
11
Legal status by country
Country
Status
Note
Checked
United States
Prescription
On 19 September 2025 the FDA granted accelerated approval to elamipretide hydrochloride (brand name Forzinity, Stealth BioTherapeutics) solely for the treatment of Barth syndrome in patients weighing at least 30 kg. For this single ultra-rare indication it is therefore an approved prescription medicine; continued approval is contingent on verification of clinical benefit in confirmatory trials. For all other investigated indications (e.g. mitochondrial myopathy, heart failure, dry AMD) elamipretide remains investigational and unapproved.
2026-06-07
Germany
Unapproved
There is no central EMA approval and no national German approval for elamipretide; the substance is not marketable as a medicine in Germany and is considered investigational. Use outside clinical trials is not covered by medicines law. Material offered as a 'research chemical' does not circumvent medicines-law requirements.
2026-06-07
12
Reconstitution calculator
Pure mg/mL maths — works like a calculator. Not a usage recommendation.
Peptides ship as a dry powder. Once dissolved in a liquid (reconstitution), this calculator answers a single question: how much substance is in one millilitre of solution afterwards?
1Enter the vial's substance amount (printed on the label).
2Enter how much solvent you add.
3Result = concentration in mg per mL.
Printed on the label
/
Liquid you add
=
2.50
mg / mL
5 mg in 2 mL gives 2.50 mg/mL — each millilitre contains 2.50 mg of substance.
Reid Thompson W, Hornby B, Manuel R, et al. · 2021
A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism