Overview
- Eligibility covers patients with Barth syndrome who weigh at least 30 kilograms, according to the FDA authorization.
- Forzinity is a once‑daily subcutaneous injection that binds to the inner part of mitochondria to improve their structure and function.
- The surrogate endpoint reflects improved strength in the muscle that straightens the knee, which the FDA says may translate into standing more easily or walking farther.
- Stealth Biotherapeutics must run a post‑market confirmatory trial, and the FDA can seek withdrawal if effectiveness is not verified.
- Barth syndrome is a rare mitochondrial disorder that primarily affects males, often causing severe heart failure in infancy, with an estimated 230 to 250 identified cases worldwide as of 2020.