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

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) ...

PTC Therapeutics has not provided substantial evidence that vatiquinone is effective in treating children and adults with Friedreich’s ataxia, the FDA told the company in a complete response letter, ...

Lexeo Therapeutics Inc. (NASDAQ:LXEO) is one of the best IPO stocks to buy according to Wall Street analysts. Earlier on July ...

The regulatory body determined that PTC Therapeutics’ New Drug Application for vatiquinone did not provide sufficient ...

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

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.

It looks like Biogen Inc.’s Nrf2 activator, Skyclarys (omaveloxolone), will maintain its status as the sole therapy approved for treating patients with Friedreich’s ataxia (FA), at least for now. The ...

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

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.