FDA Accelerates Rare Disease Drug Approval for Barth Syndrome

Stealth BioTherapeutics secured accelerated FDA approval for elamipretide (Forzinity), a treatment for Barth syndrome, an ultra-rare pediatric mitochondrial cardioskeletal disorder. After an earlier complete response letter and substantial development delays, the approval underscores progress in therapeutic options for rare diseases with limited treatments. This marks the first FDA-approved therapy for Barth syndrome, offering muscle strength improvements to affected patients and reflecting a growing regulatory emphasis on rare disease drug development.