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 (Forzinity) for Barth syndrome in patients weighing at least 30 kg — granted on a knee-extensor muscle-strength surrogate despite the failed blinded crossover primaries, with a confirmatory trial required as a condition.

Population

Regulatory commentary on the Barth syndrome approval

Intervention

n/a

Comparator

n/a

Limitations

Commentary, not primary data; the approval's knee-extensor endpoint measures muscle strength in a genetic myopathy and says nothing about cartilage or joints.