FDA grants accelerated approval to elamipretide (Forzinity) for Barth syndrome

The FDA granted accelerated approval to Stealth BioTherapeutics' elamipretide (Forzinity) for Barth syndrome, an ultra-rare inherited mitochondrial disease, in patients weighing at least 30 kg. It is the first treatment for the disease and the first FDA-approved therapy aimed at mitochondria, where the peptide binds cardiolipin in the inner membrane. Approval was based on improved knee extensor muscle strength in the TAZPOWER trial, and continued approval depends on confirmatory evidence of clinical benefit.