FDA Rejects PTC Therapeutics Friedreich’s Ataxia Drug Over Efficacy Concerns

The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter rejecting PTC Therapeutics' New Drug Application for vatiquinone as a treatment for Friedreich’s ataxia (FA), citing insufficient evidence of efficacy. Despite vatiquinone showing some benefits on secondary endpoints and long-term extension studies, the FDA found that the primary endpoint in the phase 3 MOVE-FA trial was not met, requiring an additional well-controlled study for reconsideration. PTC Therapeutics’ CEO expressed disappointment but remains hopeful, stating the company plans to meet with the FDA to discuss next steps. The regulatory setback has negatively impacted PTC's stock and investor sentiment, with concerns about the company’s financial and regulatory challenges. Friedreich’s ataxia is a rare neurodegenerative disorder affecting muscle coordination and neurological function, and vatiquinone was seen as a potential therapy for both children and adults with the disease. The FDA's decision highlights the need for more robust clinical evidence before approval can be granted.