PTC Therapeutics, Inc. (PTCT) announced Tuesday that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia.
The CRL states that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission.
PTC said it believes 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 now plans to meet with the FDA to discuss potential steps to address the issues raised in the CRL.
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