FDA grants accelerated approval to Forzinity (elamipretide/SS-31), the first treatment for Barth syndrome
The FDA approved Forzinity (elamipretide HCl) to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg — the first approved therapy for this ultra-rare mitochondrial disease and the first FDA-approved mitochondria-targeted therapeutic of any kind. Approval was based on improvement in knee extensor muscle strength as an intermediate clinical endpoint under the accelerated approval pathway.
This is a summary. The reporting is U.S. Food & Drug Administration's, and the link below goes to the original article.