FDA Denies Approval of Vatiquinone for Friedreich Ataxia

The Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to PTC Therapeutics regarding the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich ataxia (FA).

Vatiquinone is a 15-lipoxygenase (15-LO) inhibitor designed to reduce cellular inflammation and oxidative stress in patients with FA. The original NDA submission included data from the registration-directed phase 3 MOVE-FA trial (ClinicalTrials.gov Identifier: NCT04577352), as well as 2 long-term studies evaluating vatiquinone in FA. 

Findings from MOVE-FA showed that while vatiquinone did not meet statistical significance in the modified Friedreich Ataxia Rating Scale (mFARS) score at week 72 (primary endpoint) vs placebo, the 15-LO inhibitor did show significant benefit in key secondary endpoints (eg, bulbar subscale, upright stability subscale, Modified Fatigue Scale). Additionally, in both long-term studies, vatiquinone demonstrated a statistically significant improvement on the mFARS relative to a matched natural history population. 

According to the CRL, vatiquinone could not be approved due to a lack of substantial evidence of efficacy. The FDA requested that an additional adequate and well-controlled study be conducted to support NDA resubmission. 

“We are of course disappointed by the FDA’s decision to not approve vatiquinone,” said Matthew B. Klein, MD, 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 ataxia. We plan to meet with the FDA to discuss potential steps to address the issues raised in the CRL.”

This article originally appeared on MPR