Stealth's Patience Rewarded as FDA Approves Barth Drug

Stealth's patience rewarded as FDA approves Barth drug

After a long haul, Stealth Bio has finally claimed FDA approval for Forzinity, the first treatment for ultra-rare disease Barth syndrome.

U.S. FDA approves first treatment for rare, life-threatening disease in males

FDA approves Stealth Biotherapeutics' Forzinity injection for rare genetic disease Barth syndrome, impacting males, with accelerated approval pathway.

Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY™ (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome

NEEDHAM, Mass., Sept. 19, 2025 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a commercial-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and…

FDA clears first treatment for rare male genetic disorder, Barth syndrome

The FDA approved Stealth Biotherapeutics’ Forzinity, the first treatment for Barth syndrome, a rare genetic disease affecting males, under its accelerated pathway.

FDA Approves First Drug for Rare, Fatal Barth Syndrome

WASHINGTON, D.C. — The U.S. Food and Drug Administration has granted accelerated approval to Forzinity (elamipretide) injection, the first treatment for Barth syndrome, a rare and life-threatening mitochondrial disease that primarily affects males. The approval, announced this week, applies to patients weighing at least 30 kilograms and represents a milestone for a condition that has long lacked any approved therapies. Barth syndrome, which ofte…

FDA Grants Accelerated Approval to FORZINITY for Treatment of Barth Syndrome

Stealth BioTherapeutics Inc. announced that the U.S. Fo […]