This ultra-rare, progressive disease affects approximately 150 individuals in the United States, causing severe cardiac and skeletal muscle manifestations that significantly reduce life expectancy. The NDA has been granted priority review, with a Prescription Drug User Fee Act (PDUFA) action date of January 29, 2025.
"We are grateful to the patients, families, and physicians who participated in the studies that supported this new drug application, as well as to the patient and medical community members who requested a review of elamipretide as the first potential therapy for Barth syndrome," said Reenie McCarthy, Chief Executive Officer. "We look forward to meeting with the Cardiovascular and Renal Drugs Advisory Committee to discuss elamipretide's potential to improve the lives of individuals living with Barth syndrome, a devastating disease with a significant and urgent unmet medical need."
If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic. In addition to Barth syndrome, elamipretide is in Phase 3 trials for primary mitochondrial myopathy, with pivotal data expected by the end of 2024, and for dry age-related macular degeneration.
About Barth Syndrome:
Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with reduced life expectancy, with 85% of early deaths occurring by age 5. Barth syndrome occurs primarily in males and is estimated to affect one in 1,000,000 males worldwide or around 150 individuals in the United States. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome. Elamipretide has Orphan Drug, Fast Track and Rare Pediatric Designation from the FDA and Orphan Drug Designation from the EMA for the treatment of Barth syndrome.
About Stealth BioTherapeutics:
Our mission is to develop novel therapies to improve the lives of patients living with diseases of mitochondrial dysfunction. Our lead product candidate, elamipretide, is under review for Barth syndrome and in late-stage development for primary mitochondrial myopathy and dry age-related macular degeneration. We are also evaluating a topical ophthalmic formulation of our second-generation clinical-stage candidate, bevemipretide (SBT-272), for dry age-related macular degeneration, and have a deep pipeline of novel compounds under evaluation for rare neurological and cardiac disease indications.
