Stealth BioTherapeutics: FDA Advisory Panel to Review Elamipretide for Barth Syndrome

Stealth BioTherapeutics Inc., a biotechnology firm specializing in therapies for mitochondrial dysfunction, has announced an upcoming advisory committee meeting with the U.S. Food and Drug Administration (FDA). This meeting, scheduled with the Cardiovascular and Renal Drugs Advisory Committee (CRDAC) on October 10, 2024, will focus on evaluating the New Drug Application (NDA) for elamipretide. Elamipretide is poised to become the first treatment option for Barth syndrome, a rare and progressive disease impacting around 150 individuals in the U.S. who suffer from severe cardiac and skeletal muscle issues that drastically reduce life expectancy. The NDA has been fast-tracked with a Prescription Drug User Fee Act (PDUFA) action date set for January 29, 2025.

Reenie McCarthy, CEO of Stealth BioTherapeutics, expressed gratitude to all who participated in the studies backing the NDA, as well as the broader patient and medical community advocating for a review of elamipretide. McCarthy emphasized the significance of this meeting, noting its potential to improve life quality for those afflicted by Barth syndrome, a condition with an urgent need for effective medical solutions.

The approval of elamipretide would mark its first marketing authorization, positioning it as a pioneering mitochondria-targeted therapeutic. Besides Barth syndrome, elamipretide is also undergoing Phase 3 trials for primary mitochondrial myopathy, with crucial data anticipated by the end of 2024, and for dry age-related macular degeneration.

Barth syndrome is a genetic disorder primarily affecting males, characterized by cardiac irregularities that lead to exercise intolerance, muscle weakness, severe fatigue, heart failure, frequent infections, and stunted growth. The disease significantly reduces life expectancy, with 85% of early deaths occurring before the age of five. It affects an estimated 1 in 1,000,000 males globally, translating to about 150 individuals in the United States. Currently, there are no approved therapies by the FDA or the EMA for Barth syndrome. Elamipretide has received multiple designations from the FDA, including Orphan Drug, Fast Track, and Rare Pediatric Designations, as well as Orphan Drug Designation from the EMA.

Stealth BioTherapeutics is dedicated to creating innovative therapies aimed at enhancing the lives of patients with mitochondrial dysfunction-related diseases. Their leading product candidate, elamipretide, is under review for Barth syndrome and is in advanced development stages for primary mitochondrial myopathy and dry age-related macular degeneration. The company is also exploring a topical ophthalmic formulation of bevemipretide (SBT-272), another clinical-stage candidate, for dry age-related macular degeneration. Additionally, Stealth BioTherapeutics has a robust pipeline of new compounds in development for rare neurological and cardiac diseases.

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