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 ...

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.

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

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's fourth approval application submitted to FDA in 2024 - WARREN, N.J., Dec. 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (PTCT) announced today the submission of the vatiquinone New Drug ...

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 ...