FDA Issues Complete Response Letter for PTC’s Vatiquinone NDA

PTC Therapeutics, Inc. announced that the U.S. FDA has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich’s ataxia.

The FDA stated in the CRL that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission.

“We are of course disappointed by the FDA’s decision to not approve vatiquinone,” said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. “We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich’s ataxia. We plan to meet with the FDA to discuss potential steps to address the issues raised in the CRL.”

The application had been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of Aug. 19, 2025.