PTC Therapeutics, Inc. (NASDAQ:PTCT) saw its stock slide 5% after the U.S. Food and Drug Administration (FDA) declined to approve its New Drug Application (NDA) for vatiquinone, a treatment candidate for Friedreich’s ataxia. The decision came in the form of a Complete Response Letter (CRL).
In its letter, the FDA said that PTC had not provided “substantial evidence of efficacy” to support approval and that an additional “adequate and well-controlled study” would be required before the application could be reconsidered.
“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.”
PTC noted that it intends to meet with the FDA to discuss possible next steps to resolve the agency’s concerns.
Vatiquinone is a first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme involved in managing energy and oxidative stress pathways disrupted in patients with Friedreich’s ataxia. The experimental therapy has been tested in multiple clinical trials, particularly among pediatric populations, where the company said it showed an impact on mortality risk as well as neurological and neuromuscular symptoms.
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