Elamipretide: The first cardiolipin-directed mitochondrial therapeutic for Barth syndrome approved under accelerated approval

Zhao C, Zhuang X, Gao J · Drug Discoveries & Therapeutics

Records the 19 September 2025 FDA accelerated approval of elamipretide for Barth syndrome: the 12-patient blinded crossover showed no significant improvement on either primary endpoint (six-minute walk and fatigue score), the 168-week open-label extension showed sustained improvement from its own baseline, and knee-extensor muscle strength, a secondary endpoint not detected in the blinded phase, emerged during the extension; a confirmatory randomised trial is a condition of the approval.

Population

Regulatory commentary on the Barth syndrome approval

Intervention

n/a

Comparator

n/a

Limitations

A two-page commentary, not primary data; the knee-extensor endpoint measures muscle strength in a genetic myopathy and says nothing about cartilage or joints. No funding, no conflicts declared.