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Ela3.0
Elamipretide Research
Too few graded studies
38 peer-reviewed studies
What the evidence says
Too few graded studies
Elamipretide doesn't have enough studies with structured effect-size data yet to pool a verdict. Browse the studies below for individual findings.
Most evidence is from high-quality meta-analyses and randomised trials published 2012–2026 with a typical study size of 27 participants.
Based on 38 studies · 1 meta-analysis · 17 RCTs · 46 total participants
Confidence
Moderate confidence
By outcome
Mitochondrial disease & myopathy
Mostly mechanism / observational22 studies
Cardiac & reperfusion
Too few graded studies10 studies
Energy & fatigueA mitochondria-targeted (cardiolipin-binding) peptide that improved subjective fatigue and showed an open-label signal in Barth syndrome; failed pivotal myopathy and infarct-size endpoints. · Not established
Mostly mechanism / observational6 studies
Vision & eye health
Mostly mechanism / observational3 studies
Safety profile
Too few graded studies3 studies
Active research area
29 studies in the last 5 years · Latest meta-analysis: 2022
201220192026
1Review2026
In plain English: By synthesizing established and next-generation therapeutic strategies, it highlights the potential of precision mitochondrial medicine to reshape the future management of cardiovascular disease.
Alia S, Pedriali G, Compagnucci P, Valeri Y, Membrino V, Di Crescenzo T, Tremoli E, Mazzanti L, Vignini A, Pinton P, Casella M. · Cells (2026)
These interventions aim to restore organelle structure, improve bioenergetics, and reestablish balanced quality control networks.
This review integrates recent mechanistic insights with emerging translational evidence, outlining how mitochondria function as bioenergetic and inflammatory hubs in CVD.
By synthesizing established and next-generation therapeutic strategies, it highlights the potential of precision mitochondrial medicine to reshape the future management of cardiovascular disease.
In plain English: Future success depends on multidisciplinary collaboration and the validation of mitochondrial biomarkers to advance precision medicine in CKM syndrome.
Kuo YJ, Chen LF, Chen Y, Chen PL, Lin HY. · International journal of molecular sciences (2026)
Finally, we review emerging mitochondrial-targeted strategies, such as mitoquinol, elamipretide and NAD+ boosters, which aim to restore the SIRT1-PGC-1 α signaling axis.
Mitochondria function as the central engines of the CKM axis.
A shift toward a mitocentric clinical model may enable earlier intervention and more precise targeting of the mechanisms driving organ crosstalk.
In plain English: Furthermore, we offer a comprehensive outlook on the expansive prospects of SS-31's future development and application.
Du X, Zeng Q, Luo Y, He L, Zhao Y, Li N, Han C, Zhang G, Liu W. · Mitochondrion (2024)
Its notable attributes encompass the mitigation of oxidative stress, the suppression of inflammatory processes, the maintenance of mitochondrial dynamics, and the prevention of cellular apoptosis.
As such, SS-31 may emerge as a viable choice for the treatment of mitochondrial dysfunction-related ailments in the foreseeable future.
Furthermore, we offer a comprehensive outlook on the expansive prospects of SS-31's future development and application.
In plain English: This article summarizes the milestones in the development of elamipretide leading to this first approval for Barth syndrome.
Shirley M. · Drugs (2026)
In September 2025, elamipretide was granted accelerated approval in the USA for use to improve muscle strength in adult and pediatric patients with Barth syndrome weighing ≥ 30 kg.
With this accelerated approval, elamipretide became the first disease-specific treatment approved for Barth syndrome, an ultra-rare X-linked recessive genetic disorder.
Elamipretide is also under phase III clinical development for use in the treatment of dry age-related macular degeneration and mitochondrial myopathies.