Progressive(US:PGR) Prnewswire·2025-09-20 00:07Core Insights - Stealth BioTherapeutics Inc. has received accelerated approval from the FDA for FORZINITY (elamipretide HCl) to improve muscle strength in patients with Barth syndrome weighing at least 30 kg, marking it as the first treatment option for this ultra-rare disease [1][2][10] Group 1: Approval Details - FORZINITY is indicated for improving muscle strength in adult and pediatric patients with Barth syndrome who weigh at least 30 kg, based on data from the TAZPOWER clinical trial [7][10] - The approval is contingent upon verification of clinical benefit in a confirmatory trial, and the most common adverse reactions reported were injection site reactions [2][7] Group 2: Patient Access and Future Plans - The company plans to provide expanded access to children weighing less than 30 kg who are currently receiving treatment or require emergency access while working with the FDA to gather additional data for potential label expansion [2][5] - Stealth is committed to ensuring uninterrupted access for all current patients and plans to work with payers and providers to facilitate timely access to FORZINITY, which is expected to be available for prescriptions in the U.S. by year-end [6][10] Group 3: Community Impact - The approval is seen as a significant victory for the Barth syndrome community, providing hope for expedited regulatory attention to other ultra-rare diseases [2][3] - The Barth Syndrome Foundation has expressed gratitude for the approval, emphasizing the need for broader access to the treatment for younger patients who are currently ineligible due to weight restrictions [4][10] Group 4: Company Background - Stealth BioTherapeutics focuses on developing therapies for diseases related to mitochondrial dysfunction and is also studying elamipretide for other indications, including dry age-related macular degeneration and primary mitochondrial myopathy [10]