SS-31 (Elamipretide)

Brand names: Forzinity® (approved, Barth syndrome), Bendavia (investigational), MTP-131

Mechanism of action: Targets cardiolipin in mitochondria; Improves electron transport chain efficiency; Supports ATP production; Reduces oxidative stress; Stabilizes mitochondrial membranes; Improves cellular energy production

Approved indications: Improving muscle strength in adult and pediatric patients with Barth syndrome weighing ≥30 kg (Forzinity, FDA accelerated approval, September 2025)

Proposed indications: Primary mitochondrial myopathy; Exercise tolerance in mitochondrial disorders; Dry age-related macular degeneration (dry AMD); Muscle recovery; Reduced fatigue; Cardiovascular support; Kidney protection; Neuroprotection; Healthy aging

Reported dosing range*: 40 mg once daily, subcutaneous (investigational, non-Barth-syndrome use)

For investigational uses outside the approved Barth syndrome indication, the MMPOWER-2 trial in primary mitochondrial myopathy evaluated 40 mg once daily by subcutaneous injection (Karaa A et al. (2020). A Randomized Crossover Trial of Elamipretide in Adults With Primary Mitochondrial Myopathy. Journal of Cachexia, Sarcopenia and Muscle). Note the later Phase 3 MMPOWER-3 trial did not meet its primary endpoints -- this dose was studied, not proven effective for that use. Does not apply to the approved Barth syndrome indication, which remains prescriber-determined.

*General range reported in available research or other sources -- not an individualized recommendation. Actual use should be supervised by a qualified provider.

Typical clinical dosing schedule: Forzinity has a defined dosing regimen per its FDA-approved prescribing information for the Barth syndrome indication, determined by your prescriber. No standardized dosing exists outside that indication; clinical trials have used varying regimens depending on the condition being studied.

Administration: Subcutaneous injection or intravenous infusion (research/clinical trial settings) — No standardized regimen — varies by clinical trial protocol

Compare with: MOTS-cTesamorelinAOD-9604GHK-Cu (Copper Tripeptide-1)

Evidence rating: ⭐⭐⭐⭐ (Approved for Barth syndrome ≥30 kg; investigational for other indications) — FDA accelerated approval (Forzinity) for improving muscle strength in Barth syndrome. Evidence for other indications is more limited — the Phase 3 MMPOWER-3 trial in primary mitochondrial myopathy was negative and failed both primary endpoints; dry AMD remains investigational (Phase 3).

Common side effects: Injection-site reactions, Headache, Nausea, Fatigue

Serious risks: No major systemic safety concerns have been identified in clinical trials to date, but long-term safety data remain limited given its investigational status.

Legal status: FDA-approved as Forzinity specifically for improving muscle strength in adult and pediatric patients with Barth syndrome weighing ≥30 kg (accelerated approval, September 2025) — available by prescription for that indication only. All other uses remain investigational and are not available by prescription; accessible only through clinical trial enrollment or compounded/research settings outside the approved indication.

Regulatory status: FDA Approved (Forzinity) — Barth syndrome ≥30 kg only • Investigational for all other indications

Contraindications: No established contraindications beyond standard hypersensitivity precautions. Outside the approved Barth syndrome indication, use is investigational only.