What it is
Elamipretide is a synthetic aromatic-cationic tetrapeptide that concentrates in the inner mitochondrial membrane through interaction with cardiolipin.
Evidence snapshot
Category: Mitochondrial. Status: Mixed. See the references section below for the underlying primary sources.
Mechanism
Binds cardiolipin on the inner mitochondrial membrane, stabilising its structure and improving electron transport chain efficiency. Reduces reactive oxygen species production.
Key research findings
Studied across mitochondrial myopathies, heart failure, age-related mitochondrial dysfunction, and several rare diseases.
Limits of the evidence
Clinical efficacy in larger Phase 3 trials has not consistently demonstrated primary endpoint benefits. FDA approval status pending for several indications.