FDA Accepts New Drug Application for Vatiquinone to Treat Friedreich Ataxia
The FDA also granted priority review, with a Prescription Drug User Fee Act target action date of August 19, 2025.
The FDA accepted a new drug application (NDA) for vatiquinone for the treatment of children and adults with Friedreich ataxia (FA) and granted it priority review. The drug has a Prescription Drug User Fee Act target action date of August 19, 2025.1
Data from the MOVE-FA (NCT04577352) supported the NDA, which established significant, durable, and clinically meaningful findings of slowing disease progression. | Image Credit: monticellllo | stock.adobe.com
"We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich's ataxia," Matthew B. Klein, MD, CEO of PTC Therapeutics, said in a news release.1 "If approved, vatiquinone would be the first therapy for pediatric patients with FA and provide a potential safe, well-tolerated, and effective treatment alternative for adults. The granting of priority review by FDA reflects the significant unmet need for younger patients with FA. We look forward to working collaboratively with FDA during the review process."
Data from the MOVE-FA (NCT04577352) supported the NDA, which established significant, durable, and clinically meaningful findings of slowing disease progression. Patients included in the trial had a modified Friedreich Ataxia Rating Scale (mFARS) of 20 to 70 at baseline, were able to ambulate at least 10 feet in 1 minute (with or without assistance), and had a diagnosis confirmed by clinical testing.1,2
Patients received randomized treatment of either vatiquinone or placebo orally 3 times a day for 72 weeks in the placebo-controlled phase and 24 weeks for the open-label extension phase. There were 146 pediatric and adult patients included in the study. The primary end point included change from baseline in mFARS score at week 72. Secondary end points included change from baseline in Friedreich Ataxia Rating Scale of Activities of Daily Living Score at week 72, change from baseline in 1-minute walk test at week 72, and number of falls through week 72, according to the clinical trial data.2,3
In the topline results, the study did not meet its primary end point, but it showed significant benefit on key disease subscales and secondary end points. Further, for individuals that completed the study, there was a significance reached for the primary end point as well as secondary end points. The mean placebo-corrected change of mFARS score was 1.6.3
In the long-term extension, the pre-specified end points were met, showing a clinically meaningful 50% slowing of disease progression over 3 years. After 24-months of treatment, individuals also had a 4.8-point benefit on mFARs relative to matching natural history populations, according to the results.4
"The results of the extension studies provide further evidence of the potential benefit of vatiquinone in slowing disease progression," Klein said in a news release.4 "In addition, the strong safety profile of vatiquinone positions it to be a potentially meaningful therapy for all Friedreich ataxia patients, particularly children and adolescents for whom there are no approved therapies.”
