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

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

"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' ...

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 is demonstrating robust growth and consistent profitability, and analyst sentiment has grown more favorable recently.

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

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 ...

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

FDA states substantial evidence of efficacy was not demonstrated.

The U.S. Food and Drug Administration has declined to approve PTC Therapeutics' drug to treat a rare genetic disorder with ...