Stealth BioTherapeutics Completes Mid-Cycle Review Meeting on Elamipretide for the Treatment of Barth Syndrome

Company is preparing for Advisory Committee Meeting with the Cardiovascular and Renal Drugs Advisory Committee

NEEDHAM, Mass., July 29, 2024 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced completion of the mid-cycle review meeting with the U.S. Food and Drug Administration ("FDA") regarding its New Drug Application ("NDA") for elamipretide for the treatment of Barth syndrome. If approved, elamipretide would be the first therapy for Barth syndrome, an ultra-rare, progressive, life-shortening, cardioskeletal disease that affects approximately 150 individuals in the United States. The FDA has confirmed its intent to hold an advisory committee meeting, which the Company expects will occur this fall. The NDA for elamipretide was granted priority review and has been assigned a Prescription Drug User Fee Act ("PDUFA") action date of January 29, 2025. 

"We look forward to sharing the evidence supporting elamipretide's potential to improve the lives of patients living with Barth syndrome with the advisory committee," said Reenie McCarthy, Chief Executive Officer. "We thank the Center for Drug Evaluation and Research and the Office of Cardiology, Hematology, Endocrinology and Nephrology for their leadership in charting pathways for devastating ultrarare diseases like Barth syndrome. This community is in dire need of a treatment option, and we believe the evidence supports elamipretide as the first therapy to address this unmet need." 

If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondrial targeted therapeutic. Elamipretide is also in Phase 3 development for primary mitochondrial myopathy, with pivotal data expected in late 2024, and 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 five. Barth syndrome occurs almost exclusively 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.

Media Contact 
Anna Stallmann Communications 
Anna Stallmann 
[email protected] 

Investor Contact 
Austin Murtagh 
Stern Investor Relations 
[email protected] 

SOURCE Stealth BioTherapeutics Inc.