Update from PTC Therapeutics
In a press release, PTC Therapeutics, Inc. announced this morning, August 19, 2025 that the U.S. Food and Drug Administration (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.
In a community statement, PTC shared that while they work with FDA to understand the next steps for vatiquinone, they will continue all ongoing vatiquinone studies and continue to provide vatiquinone for all those participating in the studies for the foreseeable future.
