An injected peptide that gathers inside mitochondria to help failing cell power plants work better. It is the first mitochondria-directed medicine approved, but only for one ultra-rare inherited disorder. The strongest human results sit outside longevity, three larger controlled trials missed their goals, and the one older-adult study's energy gain vanished within a week. Harms are mostly local injection-site reactions. (Full Review)
| Marker | Target | Why |
|---|---|---|
| Estimated glomerular filtration rate (eGFR) | ≥ 90 mL/min/1.73 m² | Sole elimination route; the one value that changes the dose |
| Absolute eosinophil count | < 0.35 × 10³/µL at baseline | A predictable drug-induced rise peaks near day 90 and then resolves |
| High-sensitivity C-reactive protein (hs-CRP) | < 0.5 mg/L | Tracks the body-wide inflammation that mitochondrial dysfunction sustains |
| Peak oxygen uptake (VO₂peak) | ≥ 37.3 mL/kg/min (men), ≥ 25.7 (women) | Identifies the low-mitochondrial-capacity phenotype in which every human signal appeared |
| Six-minute walk distance | No established target; change from the individual's own baseline, roughly 30 m is the usual meaningful shift | The primary endpoint of every registrational elamipretide trial |
| Knee extensor strength (hand-held dynamometry) | No established target; change from the individual's own baseline, Barth trial baseline median 124 newtons | The measure the accelerated approval itself rests on |
| Resting fasting lactate | < 1.5 mmol/L | Crude readout of how far oxidative metabolism is falling short |
Cadence: Kidney filtration, full blood count with eosinophil differential and an objective functional baseline before starting; blood work at 4 and 12 weeks; functional retesting at 12 and 24 weeks; then kidney function and functional measures every 6 months.