PTC Therapeutics announced on August 19, 2025, that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding its New Drug Application (NDA) for vatiquinone. The application sought approval for the treatment of children and adults living with Friedreich's ataxia.
The FDA stated in the CRL that substantial evidence of efficacy was not demonstrated for vatiquinone. The agency requested an additional adequate and well-controlled study to support any future NDA resubmission.
PTC Therapeutics expressed disappointment with the FDA's decision and plans to meet with the agency to discuss potential steps to address the issues raised in the CRL. This decision significantly impacts the near-term commercialization prospects for vatiquinone.
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