The US Food and Drug Administration (FDA) has rejected PTC Therapeutics’ new drug application (NDA) for vatiquinone in ...

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

PTC Therapeutics, Inc. (NASDAQ:PTCT) saw its stock slide 5% after the U.S. Food and Drug Administration (FDA) declined to ...

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

"We are of course disappointed by the FDA's decision to not approve vatiquinone," said Matthew Klein, PTC's chief executive, who noted that the FDA is seeking another 'adequate and well-controlled' ...

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.

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

FDA states substantial evidence of efficacy was not demonstrated.

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 ...

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 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.

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