Stealth BioTherapeutics Inc, a biotech company specializing in treatments for mitochondrial disorders, has received fast track approval from the FDA for FORZINITY. This medication is designed to improve muscle strength in both adults and children with Barth syndrome who weigh at least 30 kilograms. Barth syndrome is a rare genetic condition in the U.S. that causes serious health problems including muscle weakness and heart issues.
Health Technology Insights: Availity Names Bala Sekaran Chief Customer Officer
Reenie McCarthy, CEO of Stealth, described the FDA approval as a significant milestone for the Barth syndrome community and a hopeful sign for future therapies targeting rare diseases. She thanked everyone involved over the last decade, including patients, caregivers, advocates, researchers, and healthcare providers. Stealth plans to seek FDA approval for treating children under 30 kilograms and will gather additional data to support this effort while continuing to provide access for those in urgent need.
The FDA’s decision was based on results from the TAZPOWER clinical trial, where patients showed improved knee muscle strength during the open-label phase. The most common side effects were injection site reactions, which can be managed with oral antihistamines or topical corticosteroids. Continued approval depends on further evidence of clinical benefits from a follow-up study.
Health Technology Insights: Tandem Study: Type 2 Diabetes Patients Benefit from AID
Kate McCurdy, Chair of the Barth Syndrome Foundation, emphasized the profound impact of the disease on patients and their families. She welcomed the availability of FORZINITY but highlighted the challenge that many patients do not yet qualify due to weight restrictions. Hilary Vernon, M.D., Ph.D., a professor at Johns Hopkins University and director of a Barth syndrome clinic, expressed optimism about having a treatment option for patients facing severe muscle weakness and other serious symptoms.
FORZINITY has received several FDA designations, including Orphan Drug, Fast Track, Priority Review, and Rare Pediatric. It also earned a Rare Pediatric Disease Priority Review Voucher. Currently, the treatment is approved only for patients weighing 30 kilograms or more. Stealth is working with the FDA to broaden this approval and plans to continue compassionate use access for younger or urgently needing patients. The drug is expected to be available through specialty pharmacies across the U.S. by year-end.
Health Technology Insights: Hercules Pharmaceuticals Appoints Elie Bahou as Executive Vice President
To participate in our interviews, please write to our HealthTech Media Room at sudipto@intentamplify.com
