News

US FDA declines to approve PTC Therapeutics' oral drug for rare genetic disorder

The U.S. Food and Drug Administration has denied approval for PTC Therapeutics' oral medication for a rare genetic disorder ...
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FDA approves PTC Therapeutics’ Sephience to treat rare metabolic disorder PKU

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

The small molecule, vatiquinone, had already flunked a Phase III trial, but the company pushed ahead with an approval bid ...
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FDA rejection derails PTC Therapeutics' rare disease hopeful

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.: PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA

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

PTC Therapeutics Faces FDA Rejection On Rare Disease Treatment Over Efficacy Concerns

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

US FDA declines to approve PTC Therapeutics' rare genetic disorder drug

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

PTC Therapeutics rare disease therapy rejected by FDA

PTC Therapeutics (NASDAQ:PTCT) stock drops as the FDA declines to approve vatiquinone, its treatment for Friedreich's ataxia, ...
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PTC Therapeutics Stock In The Spotlight After FDA Refuses Approval For Drug To Treat Neurodegenerative Disease, Analysts Are Divided

The FDA stated that substantial evidence of efficacy was not demonstrated for Vatiquinone and that an additional adequate and ...