What's Happening?
Stealth BioTherapeutics has received FDA approval for Forzinity, a treatment for Barth syndrome, under the accelerated approval pathway. Forzinity is the first FDA-authorized mitochondria-targeted treatment for this ultra-rare pediatric mitochondrial disease, affecting approximately 150 individuals in the U.S. The approval is based on the Phase II TAZPOWER trial, which demonstrated improvements in knee extensor muscle strength. However, the approval is limited to patients weighing at least 66 pounds, excluding younger children who are often more severely affected.
Why It's Important?
The approval of Forzinity represents a significant advancement for the Barth syndrome community, offering hope for expedited regulatory attention to other ultra-rare diseases. This development underscores the challenges and opportunities in drug development for rare conditions, where traditional clinical trial designs may not be feasible. The approval may encourage further research and investment in treatments for ultra-rare diseases, potentially improving outcomes for affected patients.
What's Next?
Stealth plans to continue providing expanded access to Forzinity for children under 66 pounds while working with the FDA to expand the indication. The company will focus on generating additional data to support broader approval. The Barth syndrome community and Stealth will monitor the impact of Forzinity on patient outcomes and advocate for further regulatory support for ultra-rare disease treatments.
