PTC Therapeutics yesterday revealed that the US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL ...

"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' ...

Morgan Stanley lowered the firm’s price target on PTC Therapeutics (PTCT) to $71 from $76 and keeps an Overweight rating on the shares after the ...

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

Wells Fargo lowered the firm’s price target on PTC Therapeutics (PTCT) to $73 from $79 and keeps an Overweight rating on the shares. Expectations ...

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.

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.

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

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

The FDA said data for PTC's drug did not prove "substantial evidence of efficacy." Elsewhere, the FDA delayed its decision on ...

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 denied approval for PTC Therapeutics' oral medication for a rare genetic disorder ...