PTC Therapeutics announced on December 19, 2024, the submission of its New Drug Application (NDA) for vatiquinone to the U.S. Food and Drug Administration (FDA). The application seeks approval for the treatment of children and adults living with Friedreich ataxia (FA).
The vatiquinone NDA is supported by data from the placebo-controlled MOVE-FA study, as well as results from two long-term studies involving pediatric and adult FA patients. These studies collectively demonstrated significant, durable, and clinically meaningful evidence of slowing disease progression on key aspects of the disease.
Vatiquinone was also shown to be safe and well-tolerated across all age groups studied. This submission represents PTC's fourth approval application filed with the FDA in 2024, highlighting the company's active pipeline development.
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