PTC Therapeutics received a complete response letter from the FDA rejecting its Friedreich’s ataxia drug vatiquinone, citing insufficient evidence of efficacy in the phase III Move-FA trial. Though vatiquinone demonstrated significant benefits on certain disease subscales, it missed the primary endpoint. The company plans to meet with the agency to discuss potential next steps and resubmission strategies. This setback maintains Biogen’s Skyclarys as the only approved therapy for this rare neurodegenerative disorder, highlighting ongoing challenges in developing effective treatments for Friedreich’s ataxia.