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

This paper presents a new multilevel converter topology suitable for very high voltage applications, especially network interties in power generation and transmission. The fundamental concept and the ...

Pioneer Theatre Company will present a developmental reading in advance of its upcoming world premiere of Ten Brave Seconds ...

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 ...