Today, December 19, 2024, PTC Therapeutics announced their vatiquinone New Drug Application (NDA) submission to FDA for the treatment of children and adults living with Friedreich’s ataxia (FA). The vatiquinone NDA is based on data from the placebo-controlled MOVE-FA study as well as results from two long-term studies including pediatric and adult FA patients. MOVE-FA was a global registration-directed trial of vatiquinone that enrolled 146 pediatric, adolescent and adult FA patients, the majority of whom were under 18 years of age.
“While the primary endpoint of change from baseline in the overall mFARS score did not reach statistical significance (p=0.14), a statistically significant effect (p=0.021) was recorded on the mFARS upright stability subscale, which was a pre-specified endpoint, and the portion of the mFARS now understood to be the most sensitive and relevant for the enrolled primary analysis population. In addition, the effect on upright stability was concordant with favorable treatment effect on the 1-minute walk distance test and the functional component of the Modified Fatigue Rating Scale. The study included a 72-week placebo-controlled phase and a long-term open-label extension. Following completion of MOVE-FA, subjects were eligible to enroll in a long-term, open-label extension study which is ongoing.”
The FDA has 60 days to accept the New Drug Application submission. If the submission for vatiquinone is accepted, the FDA will begin its review of the full data package.