SS-31 (Elamipretide)
Forzinity · MTP-131 · Bendavia
Mitochondria-targeting tetrapeptide; received FDA accelerated approval for Barth syndrome in 2025.
Overview
Elamipretide concentrates in the inner mitochondrial membrane, binding cardiolipin. After failing several primary endpoints in heart failure and mitochondrial myopathy, it received FDA accelerated approval for Barth syndrome based on muscle-strength data.
Mechanism of action
Binds cardiolipin, stabilizing cristae structure and electron-transport efficiency while reducing reactive oxygen species.
Benefits & evidence ratings
Dosing
Doses show what has been studied or discussed — not guidance. Always follow your prescriber.
| Source | Dose | Frequency | Duration | Route |
|---|---|---|---|---|
Forzinity label (Barth syndrome) A Established / approved | 40 mg (adults ≥30 kg) | Once daily | Chronic | Subcutaneous |
Clinical trials & studies
- Phase 22021–2024Elamipretide in Barth syndrome — TAZPOWER
Crossover trial and open-label extension.
- LabelCurrentForzinity prescribing information
Official label.
Safety
Side effects
- Injection-site reactions (very common)
- Headache, dizziness
Contraindications
- Hypersensitivity
Interactions
- None well characterized
Long-term safety
Open-label extension data in Barth syndrome; confirmatory trials required.
Regulatory status
FDA approved
FDA accelerated approval (Sept 2025) as Forzinity for Barth syndrome; other uses investigational.