The original NDA submission included data from the registration-directed phase 3 MOVE-FA trial as well as 2 long-term studies evaluating vatiquinone in FA.

FDA states substantial evidence of efficacy was not demonstrated.

Today, the FDA issued a complete response letter (CRL) for vatiquinone (PTC Therapeutics), delaying its potential approval as a treatment for Friedreich ataxia amid efficacy concerns.

PTC Therapeutics has long been trying to workshop its vatiquinone into a marketable treatment for Friedreich's ataxia (FA) after a phase 3 fail in 2023. | Despite missing its primary endpoint in a ...

The small molecule, vatiquinone, had already flunked a Phase III trial, but the company pushed ahead with an approval bid ...

PTC Therapeutics (PTCT) announced that the U.S. FDA has issued a Complete Response Letter related to the New Drug Application for vatiquinone for ...

PTC Therapeutics ( ($PTCT) ) has issued an update. On August 19, 2025, PTC Therapeutics announced that the FDA issued a Complete Response Letter ...

FDA rejected PTC Therapeutics' vatiquinone for Friedreich's ataxia, citing insufficient efficacy data and requiring another ...

Investing.com -- PTC Therapeutics, Inc. (NASDAQ: PTCT) stock fell 5% after the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the company’s New Drug Application ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has issued a ...