Buddy Carter (R-GA) applauded action on Sept. 19 by the Food and Drug Administration (FDA) to grant an accelerated approval for Stealth Biotherapeutics’ Barth syndrome treatment, Forzinity. Barth syndrome primarily affects males, typically starts with severe heart failure in infancy, and causes premature death. The quality of life and daily functioning of patients with Barth syndrome are significantly affected throughout their lives, the FDA said. “FDA considers this improvement reasonably likely to predict patient benefit, such as an ability to stand more easily or walk farther,” the administration added. “As a condition of accelerated approval, FDA is requiring the manufacturer of Forzinity to conduct a post-approval randomized, double-blind, placebo-controlled trial to confirm that the changes seen on knee muscle strength translate into patient benefit.”