Stealth BioTherapeutics Announces Positive Vote from FDA Advisory Committee Meeting Supporting Potential Approval of Elamipretide for the Treatment of Barth Syndrome

Advisory Committee Voted 10 to 6 in Favor, Concluding that Elamipretide is Effective for the Treatment of Barth Syndrome

Elamipretide NDA Received Priority Review with a PDUFA Action Date of January 29, 2025

If Approved, Elamipretide Would Be the First Therapy for Barth Syndrome, a Life-Threatening Ultra-Rare Mitochondrial Disease

NEEDHAM, Mass., Oct. 11, 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 that the U.S. Food and Drug Administration ("FDA") Cardiovascular and Renal Drugs Advisory Committee ("CRDAC") voted 10 to 6 in favor of Stealth's New Drug Application ("NDA"), concluding that elamipretide is effective for the treatment of patients with Barth syndrome.

The CRDAC discussed the benefits and risks of elamipretide for the treatment of Barth syndrome, including results from the TAZPOWER Part 2 baseline-controlled extension study, the SPIBA-001 Phase 3 natural history control study, and additional supportive biomarker and preclinical data. The CRDAC's vote, while not binding, will be considered by the FDA when making its decision regarding the potential approval of elamipretide for Barth syndrome. If approved, elamipretide would be the first therapy for this ultra-rare, progressive, life-shortening, cardioskeletal disease that affects approximately 150 individuals in the United States. 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 are pleased that the FDA advisory committee has thoughtfully considered the elamipretide data package – including the positive results from the rigorously constructed natural history control trial, the durable open-label extension data, and expanded access cases – and recognized that these data support the potential of elamipretide to improve the lives of patients with this devastating disease," said Reenie McCarthy, Chief Executive Officer. "We are grateful to the many patients, caregivers, advocates, and healthcare providers who shared their perspectives and experiences with the advisory committee. We look forward to collaborative conversations with the FDA as the agency completes its review of elamipretide, the first potential therapy for Barth syndrome."

If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic. In addition to Barth syndrome, elamipretide is in Phase 3 trials for primary mitochondrial myopathy, with pivotal data expected by the end of 2024, and for 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 5. 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 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.

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SOURCE Stealth BioTherapeutics Inc.