Rankings / Mitochondria & Cellular Energy
SS-31 (elamipretide)
Mitochondria & Cellular Energy · Mitochondrial peptide
Tier B+
Bottom line
Read Off Label grades SS-31 (elamipretide) as B+ (7.1/10) based on moderate evidence, moderate benefit magnitude, and a low-risk safety profile.
Specifically targets dysfunctional mitochondria (binds cardiolipin to stabilize cristae) — novel MOA.
Studied, labeled, or reported-use context: 40 mg SC daily in Phase 2/3 trials — Rx in US — FDA accelerated approval Sept 2025 as FORZINITY (elamipretide) for Barth syndrome (first approved mitochondria-targeted therapeutic); off-label/investigational for mitochondrial-energy and longevity uses; not individualized guidance.
Frontier potential — editorial judgment, not evidence
This is a separate axis from the grade above. It asks a different question: if this worked in humans, how much would it matter? It is our judgment, it is not part of the composite score, and it cannot raise a grade. A high number here is a statement about the idea, not about whether it works or whether anyone should take it.
What the ceiling rests on
A genuinely novel mechanism — it binds cardiolipin to stabilise mitochondrial cristae rather than acting as a general antioxidant — and it earned FDA accelerated approval in 2025 for Barth syndrome, making it the first approved mitochondria-targeted therapeutic. Proof that the target class is druggable in humans.
Why it may not get there
The approval is in an ultra-rare genetic disease with roughly 150 US patients, where mitochondrial dysfunction is the primary defect. Trials in primary mitochondrial myopathy and heart failure with preserved ejection fraction were mixed to negative, which is the more relevant test for age-related mitochondrial decline. Injectable, expensive, and unavailable for any healthy-ageing use.
What would change this A positive trial in an age-related rather than genetic mitochondrial indication.
Citation correction in progress
1 PubMed link was found to be unrelated to this entry and removed from public output. The remaining sources are shown below, but this row's claims and score still need a full source-by-source revalidation.
What the evidence says
Specifically targets dysfunctional mitochondria (binds cardiolipin to stabilize cristae) — novel MOA. FDA granted accelerated approval on Sept 19, 2025 (FORZINITY) for Barth syndrome in patients >=30 kg — the first approved mitochondria-targeted therapeutic, though an ultra-rare indication (~150 US patients). Earlier data in primary mitochondrial myopathy and heart failure with preserved EF were mixed; the mitochondrial-energy/longevity use readers care about remains off-label and unproven. Represents the emerging mitochondria-targeted therapeutic class. Fast-mover check Jul 27, 2026: Stealth BioTherapeutics began a Phase 3b/4 placebo-controlled trial of daily subcutaneous elamipretide in genetically confirmed Barth syndrome (NCT07531251, n=48, start Jul 2, 2026, primary completion Sep 2029) — a confirmatory commitment following the 2025 accelerated approval, still in the ultra-rare indication rather than the mitochondrial-energy or longevity uses.
Mechanism
Tetrapeptide that selectively targets cardiolipin in inner mitochondrial membrane; stabilizes cristae; reduces ROS; improves ETC efficiency
Studied, labeled, or reported dose & route
40 mg SC daily in Phase 2/3 trials
This records doses and routes described in studies, approved labeling, clinical practice, or documented use. It is not individualized guidance and does not establish safety; context changes with indication, formulation, health history, monitoring, and other medicines.
Citations
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Common questions
- What does the evidence show about SS-31 (elamipretide)'s effects?
- Read Off Label rates the evidence for SS-31 (elamipretide) as Moderate and the benefit magnitude as moderate, producing an overall grade of B+ (7.1/10). Specifically targets dysfunctional mitochondria (binds cardiolipin to stabilize cristae) — novel MOA.
- What safety findings are reported for SS-31 (elamipretide)?
- SS-31 (elamipretide) has a low risk profile in the database. Low (injection site reactions most common) Legal status: Rx in US — FDA accelerated approval Sept 2025 as FORZINITY (elamipretide) for Barth syndrome (first approved mitochondria-targeted therapeutic); off-label/investigational for mitochondrial-energy and longevity uses.
- What doses or routes are reported for SS-31 (elamipretide)?
- 40 mg SC daily in Phase 2/3 trials This is context from studies, approved labeling, clinical practice, or documented use; it is not individualized guidance and does not establish safety.
- How does SS-31 (elamipretide) work?
- Tetrapeptide that selectively targets cardiolipin in inner mitochondrial membrane; stabilizes cristae; reduces ROS; improves ETC efficiency
This is an independent synthesis of published research by a non-clinician. Scores are opinions supported by citations, not prescriptions. See the full disclaimer and methodology for how this score was produced and what it does and doesn't mean.