19 August 2025 - PTC Therapeutics announced today that the US FDA has issued a complete response letter related to the new drug application for vatiquinone for the treatment of children and adults living with Friedreich's ataxia.

The FDA stated in the complete response letter that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support new drug application resubmission.

Read PTC Therapeutics press release