Posted by Michael Wonder on 19 Sep 2025
FDA grants accelerated approval to first treatment for Barth syndrome
19 September 2025 - New treatment for Barth syndrome showcases FDA’s commitment to bringing effective and safe medications to patients in need.
Today, the US FDA granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome, in patients weighing at least 30 kg.
