FDA Rejects Vatiquinone as Potential Friedreich Ataxia Treatment

The Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the New Drug Application (NDA) for vatiquinone (PTC Therapeutics, Warren, NJ), denying approval of the use of the drug for the treatment of people with Friedreich ataxia (FA). According to an announcement from PTC Therapeutics, the FDA concluded that substantial evidence of efficacy was not demonstrated and that an additional adequate and well-controlled study would be required before resubmission.

Vatiquinone is an oral small-molecule inhibitor of 15-lipoxygenase, an enzyme implicated in mitochondrial dysfunction and oxidative stress—key contributors to FA pathophysiology. According to PTC Therapeutics, the drug is designed to promote neuronal survival and decrease oxidative stress and inflammation.

Vatiquinone’s NDA was primarily supported by data from the phase 3 MOVE-FA clinical trial (NCT04577352), which included 146 participants with FA aged ≥7 years. While MOVE-FA did not meet its primary end point on overall modified Friedreich Ataxia Rating Scale (mFARS) scores, treatment with vatiquinone was associated with statistically significant benefits on prespecified subscales, including upright stability and bulbar function. The FDA determined that these data did not meet the threshold for approval.

Source: A study to assess the efficacy and safety of vatiquinone for the treatment of participants with Friedreich ataxia (MOVE-FA). ClinicalTrials.gov. Updated March 27, 2025. Accessed August 28, 2025. https://clinicaltrials.gov/study/NCT04577352

Kang J. FDA Denies approval of Vatiquinone for Friedreich ataxia. eMPR.com. August 19, 2025. Accessed August 28, 2025. https://www.empr.com/news/fda-denies-approval-of-vatiquinone-for-friedreich-ataxia

PTC Therapeutics Receives Complete Response Letter for vatiquinone NDA. PRNewswire.com. Published August 19, 2025. Accessed August 28, 2025. https://www.prnewswire.com/news-releases/ptc-therapeutics-receives-complete-response-letter-for-vatiquinone-nda-302533332.html