Stealth BioTherapeutics Concludes Mid-Cycle Review on Elamipretide for Barth Syndrome

Stealth BioTherapeutics Inc., a biotechnology firm concentrating on innovative treatments for conditions linked to mitochondrial dysfunction, has announced the successful completion of the mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) for elamipretide, aimed at treating Barth syndrome. Barth syndrome is a severely debilitating, ultra-rare genetic disorder characterized by progressive cardiac and skeletal muscle weakness, affecting about 150 individuals in the United States. If elamipretide receives FDA approval, it would be the first therapeutic option available for this life-shortening disease.

The FDA has expressed its intention to hold an advisory committee meeting this fall to discuss the NDA for elamipretide, which has been granted priority review. The Prescription Drug User Fee Act (PDUFA) action date for the application is January 29, 2025. Reenie McCarthy, Chief Executive Officer of Stealth BioTherapeutics, emphasized the importance of this development, expressing gratitude to the FDA's Center for Drug Evaluation and Research and the Office of Cardiology, Hematology, Endocrinology, and Nephrology for their guidance in addressing the needs of those affected by Barth syndrome. McCarthy highlighted the critical necessity of a treatment option for this community and expressed confidence in elamipretide's potential to fulfill this unmet need.

Elamipretide, a first-in-class mitochondrial targeted therapeutic, represents a significant breakthrough if approved, as it would be the first marketed therapy for Barth syndrome. Beyond Barth syndrome, elamipretide is also undergoing Phase 3 trials for primary mitochondrial myopathy, with crucial data expected by the end of 2024. Additionally, the drug is being studied for dry age-related macular degeneration.

Barth syndrome is a genetic disorder marked by cardiac dysfunction that leads to severe symptoms such as exercise intolerance, muscle weakness, extreme fatigue, heart failure, recurrent infections, and delayed growth. The condition has a high mortality rate, with 85% of affected individuals dying by the age of five. It predominantly affects males, with an incidence rate of approximately one in 1,000,000 males worldwide. As of now, there are no approved therapies by the FDA or the European Medicines Agency (EMA) for Barth syndrome. Elamipretide has received several designations from the FDA, including Orphan Drug, Fast Track, and Rare Pediatric Designation, and has Orphan Drug Designation from the EMA for treating Barth syndrome.

Stealth BioTherapeutics is committed to developing groundbreaking therapies to enhance the quality of life for patients suffering from diseases related to mitochondrial dysfunction. The company's leading product candidate, elamipretide, is under review for Barth syndrome and is in advanced stages of development for other conditions such as primary mitochondrial myopathy and dry age-related macular degeneration. Stealth BioTherapeutics is also testing a topical ophthalmic version of their second-generation clinical-stage candidate, bevemipretide (SBT-272), for dry age-related macular degeneration. The company maintains a robust pipeline of new compounds aimed at treating rare neurological and cardiac diseases.

Stealth BioTherapeutics continues to drive forward with its mission, focusing on the discovery, development, and commercialization of novel therapies to address the needs of patients with mitochondrial dysfunction diseases. The progress with elamipretide for Barth syndrome is a significant milestone in the company's journey toward offering new hope and improved outcomes for patients grappling with these challenging conditions.