Stealth BioTherapeutics has announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for elamipretide for the treatment of Barth syndrome. This marks a significant step toward potentially providing the first approved therapy for this rare and life-threatening condition.
The NDA is supported by positive data from the SPIBA-001 Phase 3 Natural History Control Study and additional efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide has previously been granted Fast Track Designation in 2017, Orphan Drug Designation in 2018, and Rare Pediatric Disease Designation in 2020, highlighting the unmet need and potential benefit of this therapy.
Clinical Significance and Unmet Need
Barth syndrome is an ultra-rare genetic disorder primarily affecting males, with an estimated prevalence of one in 1,000,000 males worldwide, or approximately 150 individuals in the United States. The condition is characterized by cardiac abnormalities, 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. Currently, there are no FDA- or EMA-approved therapies specifically for Barth syndrome.
Kate McCurdy, Chair of the Barth Syndrome Foundation, emphasized the critical need for new treatments, stating, "This devastating disease has claimed the lives of too many of our children, loved ones, and friends... Our community desperately needs a treatment to enable us to live healthy and thriving lives."
FDA Review Process
The FDA has indicated plans to hold an advisory committee meeting to discuss the elamipretide application. While the application received a standard review designation, Stealth BioTherapeutics has requested the FDA reconsider this designation, advocating for a priority review given the severity of the disease and lack of available treatments.
Reenie McCarthy, Chief Executive Officer of Stealth BioTherapeutics, expressed optimism about the FDA's decision to convene an advisory committee, stating, "We welcome the input of committee experts to advise FDA on the seriousness of this devastating disease and the urgency of the unmet need."
Elamipretide: A Potential First-in-Class Therapy
Elamipretide is a first-in-class mitochondrial-targeted therapeutic. It is also in development for primary mitochondrial myopathy, with pivotal data from the fully-enrolled Phase 3 NuPOWER trial expected in late 2024. If approved, elamipretide would represent a significant advancement in the treatment of Barth syndrome, addressing a critical unmet need for patients and their families.
