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FDA Approves FORZINITY™ for Barth Syndrome Treatment

On September 19, 2025, Stealth BioTherapeutics Inc., a biotechnology company dedicated to improving lives affected by mitochondrial dysfunction, announced a significant milestone: the U.S. Food and Drug Administration (FDA) has granted accelerated approval for FORZINITY™ (elamipretide HCl). This groundbreaking treatment is the first therapeutic option for Barth syndrome, a rare genetic disease that predominantly affects young males.

Overview of Barth Syndrome

Barth syndrome is characterized by mitochondrial abnormalities that lead to exercise intolerance, debilitating muscle weakness, heart problems, and increased risk of early death. It is estimated to affect approximately 150 individuals in the United States, making it an ultra-rare condition. Tragically, many young boys with Barth syndrome do not survive long enough to meet the criteria for existing treatments.

Significance of FORZINITY™ Approval

The approval of FORZINITY marks a pivotal moment for the Barth syndrome community. As Reenie McCarthy, Stealth’s CEO, stated, this represents a victory not only for patients but also highlights the potential for expedited regulatory pathways for other ultra-rare diseases. The FDA’s support emphasizes the shared commitment towards securing timely treatment access for patients with severe health conditions.

FORZINITY is designed to enhance muscle strength in both adult and pediatric patients weighing at least 30 kilograms (about 66 pounds). The FDA’s decision was based on compelling efficacy and safety data derived from the TAZPOWER clinical trial, where participants exhibited improved knee extensor muscle strength.

Ongoing Developments and Future Plans

In response to the approval, Stealth has committed to continuing access for patients weighing less than 30 kilograms, which represents a vulnerable population severely impacted by Barth syndrome. They will be working closely with the FDA to provide additional data required for potential expansion of treatment eligibility. Members of the Barth syndrome community, such as Kate McCurdy, Board Chair of the Barth Syndrome Foundation, have expressed deep gratitude for this decision while also advocating for rapid inclusion of the youngest patients who face daunting challenges due to the disease.

Healthcare professionals, including Dr. Hilary Vernon from Johns Hopkins University, have stressed the urgency for effective treatment options for Barth syndrome patients who suffer from severe manifestations including crippling muscle weakness. The availability of FORZINITY not only offers hope but also strengthens the community's resolve in pushing for further advancements in treatment access.

Path Forward for Stealth BioTherapeutics

Stealth BioTherapeutics aims to ensure that FORZINITY is readily available by the end of the year, facilitated through specialty pharmacies. They are set to introduce initiatives to support patients, ensuring equitable access while working through reimbursement challenges with healthcare providers.

Furthermore, FORZINITY has received multiple designations from the FDA, including Orphan Drug and Fast Track, highlighting its importance and potential in treating ulcers rare diseases like Barth syndrome. Moving forward, Stealth’s focus will remain on developing additional therapies that address mitochondrial dysfunction, with studies underway for other conditions.

The approval of FORZINITY™ not only illuminates the path for Barth syndrome patients but also ignites hope for families affected by other severe and rare diseases. As the landscape of treatment evolves, the commitment to improving patient lives remains at the forefront of Stealth BioTherapeutics’ mission.