FDA Advisory Committee to Review Elamipretide for Barth Syndrome
Key Insights
The FDA's Cardiovascular and Renal Drugs Advisory Committee will review elamipretide for Barth syndrome (search) on October 10, 2024.
Elamipretide, a first-in-class mitochondria-targeted therapeutic, is under review as a potential treatment for this ultra-rare genetic condition.
The FDA has granted priority review to the NDA for elamipretide, with a PDUFA action date set for January 29, 2025.
Stealth BioTherapeutics (search) Inc. announced that the U.S. Food and Drug Administration (FDA) will convene its Cardiovascular and Renal Drugs Advisory Committee (CRDAC) on October 10, 2024, to evaluate the New Drug Application (NDA) for elamipretide. Elamipretide is a first-in-class, mitochondria-targeted therapeutic being considered for the treatment of Barth syndrome (search), an ultra-rare and progressive genetic condition. The NDA has been granted priority review, with a Prescription Drug User Fee Act (PDUFA) action date of January 29, 2025.
Elamipretide's Potential Impact on Barth Syndrome
Barth syndrome (search) is characterized by cardiac abnormalities, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The condition primarily affects males and is estimated to impact 1 in 1,000,000 males worldwide, with approximately 150 individuals affected in the United States. A significant concern is the reduced life expectancy associated with Barth syndrome; approximately 85% of early deaths occur by age 5. Currently, there are no FDA- or EMA-approved therapies available for patients with Barth syndrome.
Addressing Unmet Needs
"We are grateful to the patients, families, and physicians who participated in the studies that supported this new drug application, as well as to the patient and medical community members who requested a review of elamipretide as the first potential therapy for Barth syndrome (search)," said Reenie McCarthy, Chief Executive Officer of Stealth BioTherapeutics (search). "We look forward to meeting with the Cardiovascular and Renal Drugs Advisory Committee to discuss elamipretide's potential to improve the lives of individuals living with Barth syndrome, a devastating disease with a significant and urgent unmet medical need."
Elamipretide's Development Pipeline
Elamipretide has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug Designation from the EMA for the treatment of Barth syndrome (search). Beyond Barth syndrome, elamipretide is also in Phase 3 trials for primary mitochondrial myopathy (search), with pivotal data expected by the end of 2024, and for dry age-related macular degeneration (search). Stealth BioTherapeutics (search) is also evaluating bevemipretide (SBT-272), a second-generation clinical-stage candidate, in a topical ophthalmic formulation for dry age-related macular degeneration.