FDA accelerated approval for first treatment for Barth syndrome

The US Food and Drug Administration (FDA) late Friday granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome, in patients weighing at least 30 kg. Barth syndrome is a rare, serious and life-threatening disease of the mitochondria (the energy-producing parts of cells).

This application was granted priority review, and Forzinity was granted a rare pediatric disease designation. The FDA granted the accelerated approval of Forzinity, as well as a rare pediatric disease priority review voucher, to Stealth Biotherapeutics (OTCQB: MITO).

The decision comes after an earlier setback, when the agency in April this year pushed back, despite a positive advisory committee vote followed in October last year, the outcome was not unanimous, with 6 voting against and 10 in favor.