- FDA decision on elamipretide for the treatment of Barth syndrome delayed -
- Labelling discussions underway, but no revised action date communicated by FDA -
NEEDHAM, Mass., April 29, 2025 /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 that the U.S. Food and Drug Administration (FDA) communicated that it would not meet today's prescription drug user fee action (PDUFA) date for its new drug application (NDA) for its investigational medicine, elamipretide, for the treatment of Barth syndrome. Barth syndrome is an ultra-rare, life threatening pediatric mitochondrial disease which is known to affect less than 150 individuals in the United States and less than 300 individuals worldwide.
"We appreciate the diligent efforts of the FDA to progress its review of our new drug application, including its confirmation of post-marketing requirements and its recent initiation of labeling discussions," said Reenie McCarthy, Chief Executive Officer. "We hope to gain more information on the revised action date in the coming days to inform our ability to continue supporting expanded and emergency access to elamipretide while advancing towards a potential FDA approval."
Stealth's NDA was submitted to the FDA on January 29, 2024 and filed by the FDA for standard review on March 29, 2024 with a PDUFA date of January 29, 2025. On May 6, 2024, FDA granted priority review. On October 10, 2024, the Cardiovascular and Renal Drugs Advisory Committee (CRDAC) met and concluded that elamipretide is effective for the treatment of patients with Barth syndrome. On January 14, 2025, the FDA notified Stealth that new analyses requested by FDA in late November and early December 2024 required a delay of the PDUFA date to April 29, 2025.
Elamipretide has received Orphan Drug, Fast Track, Priority Review, and Rare Pediatric Designations from the FDA and Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of Barth syndrome. More than 35 patients worldwide have received expanded or emergency access to elamipretide for the treatment of Barth syndrome, including many critically ill infants and toddlers affected by the disease.
About Barth Syndrome
Barth syndrome is an ultra-rare genetic mitochondrial disease 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 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 no approved therapies 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, where we observed a highly protective effect on photoreceptor loss in Phase 2. We are evaluating a topical ophthalmic formulation of our second-generation clinical-stage candidate, bevemipretide (SBT-272), for dry age-related macular degeneration. We have a deep pipeline of novel compounds under evaluation for rare neurological, cardiac and myopathic diseases.
Media Contact
Anna Stallmann Communications
Anna Stallmann
anna@annacomms.com
Investor Contact
Precision AQ
Austin Murtagh
Austin.Murtagh@precisionaq.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/stealth-biotherapeutics-announces-delay-in-fda-action-date-for--barth-syndrome-application-302440425.html
SOURCE Stealth BioTherapeutics Inc.
