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

The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) on Tuesday related to PTC Therapeutics, Inc.'s PTCT New Drug Application (NDA) for vatiquinone for Friedreich's ataxia. Analysis of the MOVE-FA long-term extension study demonstrated that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit (p<0.0001, N=70) on the modified Friedreich Ataxia Rating Scale (mFARS) relative to a matched natural history cohort from the FACOMS (Friedreich Ataxia Clinical Outcome Meas ...