FDA Rejects PTC’s Vatiquinone Application for Friedreich’s Ataxia, Requests Further Studies
The FDA has issued a complete response letter (CRL) to PTC Therapeutics, denying approval of vatiquinone for Friedreich's ataxia due to insufficient evidence of efficacy135.
The agency stated that an additional adequate and well-controlled study is required before the application can be resubmitted135.
PTC Therapeutics plans to meet with the FDA to discuss potential next steps and continues all ongoing vatiquinone clinical studies for participants15.
Vatiquinone was intended as a treatment for both children and adults with Friedreich's ataxia, potentially being the first approved therapy for the pediatric population24.
The Phase 2/3 MOVE-FA trial previously supported the application, but the FDA has found the efficacy data insufficient at this time2.
Shares of PTC Therapeutics were halted after the FDA's decision was announced1.
Sources:
1. https://www.morningstar.com/news/dow-jones/202508194249/fda-rejects-ptc-therapeutics-friedreichs-ataxia-drug
2. https://friedreichsataxianews.com/news/fda-grants-priority-review-vatiquinone-application-fa/
3. https://www.neurologylive.com/view/fda-denies-approval-friedreich-ataxia-agent-vatiquinone-citing-more-efficacy-needed
4. https://www.prnewswire.com/news-releases/ptc-therapeutics-announces-vatiquinone-nda-submission-to-fda-for-the-treatment-of-children-and-adults-living-with-friedreich-ataxia-302336134.html
5. https://www.curefa.org/news/ptc-therapeutics-receives-complete-response-letter-for-vatiquinone-nda/