PTC Therapeutics Faces FDA Rejection On Rare Disease Treatment Over Efficacy Concerns

1. FDA issued Complete Response Letter for PTC's vatiquinone NDA. 2. CEO expressed disappointment but believes vatiquinone is effective for Friedreich's ataxia. 3. Significant evidence of efficacy was not demonstrated, requiring further study. 4. Long-term results indicate 50% slowing in disease progression over three years. 5. PDUFA target action date for resubmitted application is August 19, 2025.