FDA Advisory Committee Supports Approval of Elamipretide for Barth Syndrome

In an important development for the treatment of Barth syndrome, the U.S. Food and Drug Administration (FDA) Cardiovascular and Renal Drugs Advisory Committee (CRDAC) has voted 10 to 6 in favor of Stealth BioTherapeutics Inc.'s New Drug Application (NDA) for elamipretide. This decision recognizes the drug's effectiveness for managing Barth syndrome, a severe and ultra-rare mitochondrial condition affecting a small population in the United States.

Barth syndrome is a genetic disorder marked by serious cardiac issues, including exercise intolerance, muscle weakness, extreme fatigue, heart failure, frequent infections, and delayed growth in affected individuals. This condition significantly reduces life expectancy, with a majority of early deaths occurring by the age of five. The syndrome primarily affects males and is extremely rare, with an estimated 150 individuals affected in the United States and a global incidence rate of about one in a million.

The CRDAC's recommendation is based on the evaluation of multiple studies including the TAZPOWER Part 2 baseline-controlled extension study, the SPIBA-001 Phase 3 natural history control study, and additional biomarker and preclinical data. These investigations provided substantial evidence supporting the potential benefits of elamipretide for patients with Barth syndrome. Although the advisory committee's vote is not binding, it will be a vital consideration for the FDA as it decides on the approval of elamipretide.

If the FDA grants approval, elamipretide would become the first therapy available for Barth syndrome, offering a significant breakthrough for patients and their families. The NDA for elamipretide has received priority review status, and the FDA has set a Prescription Drug User Fee Act (PDUFA) action date of January 29, 2025, for its final decision.

Reenie McCarthy, Chief Executive Officer of Stealth BioTherapeutics, expressed satisfaction with the advisory committee's recognition of elamipretide's potential. McCarthy highlighted the comprehensive nature of the data considered, which included positive results from the rigorous natural history control trial, durable open-label extension data, and expanded access cases. She also acknowledged the contributions of patients, caregivers, advocates, and healthcare providers who shared their experiences during the review process.

Elamipretide, if approved, would not only mark the first therapy specifically for Barth syndrome but also represent the first marketing authorization for this first-in-class mitochondria-targeted therapeutic. Beyond Barth syndrome, elamipretide is undergoing Phase 3 trials for primary mitochondrial myopathy, with pivotal data anticipated by the end of 2024. Additionally, trials are being conducted for its potential use in treating dry age-related macular degeneration.

Stealth BioTherapeutics is dedicated to developing innovative treatments for diseases caused by mitochondrial dysfunction. Their lead product, elamipretide, is in the spotlight for its potential to significantly impact the lives of Barth syndrome patients. The company is also working on other therapeutic candidates, including bevemipretide (SBT-272) for dry age-related macular degeneration, and has a broad pipeline of novel compounds targeting rare neurological and cardiac diseases.

Barth syndrome remains without any FDA- or EMA-approved therapies, making the potential approval of elamipretide a groundbreaking development in the field of mitochondrial medicine. This drug has already received Orphan Drug, Fast Track, and Rare Pediatric Designations from the FDA, as well as Orphan Drug Designation from the EMA for the treatment of Barth syndrome.