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 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 ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has issued a ...

The US Food and Drug Administration (FDA) has approved PTC Therapeutics’ Sephience (sepiapterin) to treat the rare metabolic ...

The FDA approved sepiapterin (Sephience) for the treatment of children and adults with phenylketonuria (PKU), PTC Therapeutics announced. The approval includes broad labeling for the treatment of ...

The U.S. Food and Drug Administration has approved Sephience (sepiapterin), an oral therapy by PTC Therapeutics, for the treatment of phenylketonuria (PKU), a rare inherited metabolic disorder that ...

The FDA has approved the once-weekly injectable lonapegsomatropin -tcgd (Skytrofa, Ascendis Pharma A/S) for replacing endogenous growth hormone in adults with growth hormone deficiency (GHD ...

By Dean Seal Shares of PTC Therapeutics climbed after the company said federal regulators approved its treatment for elevated levels of a certain amino acid in the blood.