FDA Accepts New Drug Application for Vatiquinone to Treat Friedreich Ataxia

February 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. 

"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.”