FDA accelerated approval — Barth syndrome (as Forzinity, 2025)

After an initial 2021 filing the FDA declined to accept and a 2025 complete response letter on a resubmission, elamipretide received FDA accelerated approval in September 2025 for improving muscle strength in Barth syndrome, under the brand name Forzinity — a narrow, rare-disease indication, not a general longevity approval.

TAZPOWER / open-label extension data

The approval leaned on results from the Phase 2/3 TAZPOWER study; the randomized crossover phase missed its primary endpoint, and the case for approval ultimately rested on functional improvements in a small (n=10, 8 completers), open-label 168-week extension — data an independent expert cautioned cannot be compared to a placebo-controlled trial.

Primary mitochondrial myopathy — pivotal trial missed endpoints

A separate pivotal Phase 3 trial in primary mitochondrial myopathy did not meet its primary endpoints (6-minute walk distance and symptom score), illustrating how difficult mitochondrial-disease outcomes are to move and measure even for a molecule with a strong preclinical rationale.

Broader trial program (dry AMD, heart failure, ischemia-reperfusion)

Elamipretide has also been studied in dry age-related macular degeneration and cardiac contexts, with a mixed record across that broader program that defines both the promise and the real limits of mitochondria-targeted peptide therapy.