The FDA issued a complete response letter for PTC Therapeutics’ 15-lipoxygenase inhibitor, vatiquinone, declining approval for Friedreich’s ataxia due to insufficient evidence of efficacy. Despite positive secondary endpoint data related to upright stability, the primary endpoint in the Phase III Move-FA study was not met statistically. PTC plans to consult with the FDA on next steps for resubmission, potentially involving additional studies. The rejection underscores the difficulties in securing regulatory approval for rare neurodegenerative disease treatments and follows a previous clinical trial failure with vatiquinone in a seizure disorder indication.