PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for ...

The U.S. Food and Drug Administration has denied approval for PTC Therapeutics' oral medication for a rare genetic disorder ...

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 U.S. Food and Drug Administration has turned away PTC Therapeutics' proposed vatiquinone treatment for the rare, inherited neurodegenerative disorder Friedreich's ataxia over concerns about the ...

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

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.

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

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