– The FDA’s decision regarding elamipretide for Barth syndrome treatment is postponed –
– Labeling discussions are ongoing, but the FDA has not yet provided a revised action date –
NEEDHAM, Mass., April 29, 2025 — Stealth BioTherapeutics Inc. (“Stealth” or the “Company”), a biotechnology firm in the clinical stage focused on creating and marketing innovative therapies for illnesses linked to mitochondrial dysfunction, today reported that the U.S. Food and Drug Administration (FDA) has stated it will not meet today’s scheduled Prescription Drug User Fee Act (PDUFA) date for the New Drug Application (NDA) concerning elamipretide, its experimental drug for Barth syndrome. Barth syndrome is a very rare, life-threatening mitochondrial disease affecting children, known to affect fewer than 150 people in the United States and less than 300 worldwide.
Reenie McCarthy, Chief Executive Officer, stated, “We are grateful for the FDA’s dedicated work in reviewing our new drug application, including their confirmation of requirements for post-marketing and their recent commencement of discussions on labeling. We hope to receive further details on the updated action date soon, which will help us to continue offering expanded and emergency access to elamipretide as we work toward potential FDA approval.”
Stealth submitted its NDA to the FDA on January 29, 2024, and the FDA accepted it for standard review on March 29, 2024, setting a PDUFA date of January 29, 2025. Priority review was granted by the FDA on May 6, 2024. The Cardiovascular and Renal Drugs Advisory Committee (CRDAC) convened on October 10, 2024, and concluded that elamipretide is an effective treatment for Barth syndrome patients. On January 14, 2025, Stealth was informed by the FDA that new analyses requested in late November and early December 2024 would necessitate postponing the PDUFA date to April 29, 2025.
The FDA has granted Elamipretide Orphan Drug, Fast Track, Priority Review, and Rare Pediatric Disease Designations, as well as Orphan Drug Designation from the European Medicines Agency (EMA) for treating Barth syndrome. More than 35 patients globally have been given expanded or emergency access to elamipretide to treat Barth syndrome, including numerous critically ill infants and young children with the condition.
About Barth Syndrome
Barth syndrome is a very rare genetic mitochondrial condition causing exercise intolerance, muscle weakness, extreme fatigue, heart failure, recurring infections, and stunted growth. It reduces life expectancy, with 85% of deaths before age five. Predominantly affecting males, Barth syndrome affects about one in 1,000,000 males worldwide, or about 150 people in the United States. There are currently no approved treatments for Barth syndrome.
About Stealth BioTherapeutics
Our goal is to create innovative treatments that enhance the quality of life for individuals with mitochondrial dysfunction-related diseases. Elamipretide, our main product candidate, is being reviewed for Barth syndrome and is in advanced development for primary mitochondrial myopathy and dry age-related macular degeneration, where Phase 2 trials showed significant protection against photoreceptor loss. We are also assessing a topical ophthalmic version of bevemipretide (SBT-272), our second-generation clinical candidate, for dry age-related macular degeneration. We have an extensive pipeline of new compounds being studied for uncommon neurological, cardiac, and myopathic conditions.
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SOURCE Stealth BioTherapeutics Inc.