The U.S. Food and Drug Administration (FDA) announced the accelerated approval of the first treatment for Barth syndrome in a press release.
The therapy, Forzinity (elamipretide), is given as a daily injection to patients who weigh a minimum of 30 kg. The medication sticks to the inside of the mitochondria, “improving mitochondrial structure and function.” The FDA observed the medicine’s ability to strengthen muscle in the leg and believes patients who take it will have an easier time standing and walking. Forzinity’s manufacturer must carry out a “post-approval randomized, double-blind, placebo-controlled trial” to validate this.
In addition to accelerated approval, Stealth Biotherapeutics Inc. also received rare pediatric disease designation from the FDA for Forzinity.
