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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 (NDA) for vatiquinone, a treatment for Friedreich’s ataxia.
The FDA stated in the CRL that PTC (NASDAQ:PTC) failed to demonstrate "substantial evidence of efficacy" for vatiquinone and that an additional "adequate and well-controlled study" would be needed before the company could resubmit its application.
"We are of course disappointed by the FDA’s decision to not approve vatiquinone," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich’s ataxia."
The company plans to meet with the FDA to discuss potential steps to address the issues raised in the regulatory agency’s response.
Vatiquinone is described as a first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that regulates energetic and oxidative stress pathways disrupted in Friedreich’s ataxia. The drug has been evaluated in several clinical studies, particularly in pediatric patients, where the company reports it demonstrated impact on mortality risk and various neurological and neuromuscular disease symptoms.
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