Rankings / Mitochondria & Cellular Energy

SS-31 (elamipretide)

Mitochondria & Cellular Energy · Mitochondrial peptide

Tier B+

peptidemitochondrialinvestigational
7.1 / 10
Tier B+
Ev 6 Bn 5 Sf 9

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.

Typical use: 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.

What this is

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.

Mechanism

Tetrapeptide that selectively targets cardiolipin in inner mitochondrial membrane; stabilizes cristae; reduces ROS; improves ETC efficiency

Dose & route

40 mg SC daily in Phase 2/3 trials

Common questions

Does SS-31 (elamipretide) work?
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.
Is SS-31 (elamipretide) safe?
SS-31 (elamipretide) has a low risk profile in published human data. 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. This is not medical advice — see the disclaimer.
What is the typical dose for SS-31 (elamipretide)?
40 mg SC daily in Phase 2/3 trials
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.