Elamipretide
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.
Mechanism of action
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.
Доказательная база
Что показывают исследования
Где исследования расходятся
Does elamipretide have an established clinical benefit, and how does the US approval reconcile with missed endpoints in several trials?
Фармакокинетика
Пути введения в литературе
Более безопасное применение и риски
Известные нежелательные явления из исследований
Взаимодействия и комбинации
Риски и гигиенические аспекты в литературе
Mixed evidence base despite accelerated approval in a niche indication
Правовой статус по странам
Калькулятор восстановления
Пептиды поставляются в виде сухого порошка. После растворения в жидкости (восстановление) этот калькулятор отвечает на единственный вопрос: сколько вещества содержится в одном миллилитре раствора после этого?
- 1Введите количество вещества во флаконе (указано на этикетке).
- 2Введите, сколько растворителя вы добавляете.
- 3Результат = концентрация в mg на mL.
Реестр исследований
Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial
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
Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 Trial
Источники и методика
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