STEALTH BIOTHERAPEUTICS RESUBMITS NEW DRUG APPLICATION FOR ELAMIPRETIDE FOR THE TREATMENT OF BARTH SYNDROME

Core Insights - Stealth BioTherapeutics has resubmitted a New Drug Application (NDA) to the FDA for elamipretide to treat Barth syndrome, addressing previous FDA concerns regarding manufacturing and safety [1][2][3] - The FDA's May 2025 complete response indicated the need for resubmission without new clinical efficacy data, but with a minor safety update and confirmation of resolved manufacturing deficiencies [1][4] - Elamipretide has shown significant improvement in knee extensor muscle strength (>45%) in the TAZPOWER Phase 2 trial, correlating with clinical benefits measured by the six-minute walk test [2][4] Company Overview - Stealth BioTherapeutics is a clinical-stage biotechnology company focused on developing therapies for diseases related to mitochondrial dysfunction, with elamipretide as its lead candidate [7] - The company is also developing elamipretide for other conditions such as primary mitochondrial myopathy and dry age-related macular degeneration [5][7] - The company has a pipeline of novel compounds targeting rare neurological and cardiac diseases, following promising preclinical results [7] Barth Syndrome Insights - Barth syndrome is an ultra-rare genetic condition affecting approximately one in 1,000,000 males, characterized by severe symptoms including muscle weakness and heart failure [6] - There are currently no FDA- or EMA-approved therapies for Barth syndrome, highlighting the unmet medical need [6] - Elamipretide has received Orphan Drug, Fast Track, and Rare Pediatric Designation from the FDA, indicating its potential significance in treating this condition [6]