PTC Therapeutics announces 2024 potential key clinical, regulatory events

Submission of a MAA to the EMA for sepiapterin for the treatment of PKU is expected in the first quarter. Submission of an NDA to the FDA for sepiapterin for the treatment of PKU is expected no later than the third quarter. Results from 12-month interim data from the PIVOT-HD trial of PTC518 in HD patients are expected in the second quarter. Submission of a BLA to the FDA for Upstaza for the treatment of AADC deficiency is expected in the first quarter. Topline results from the CardinALS trial of utreloxastat in ALS are expected in the fourth quarter. PTC expects the CHMP opinion from the re-examination procedure of the negative opinion on the Translarna conditional marketing authorization renewal in late January 2024, with ratification of that opinion by the European Commission 67 days later. FDA meeting for Translarna to align on the specific contents of a potential NDA resubmission is scheduled for the first quarter. FDA meeting for vatiquinone to discuss how the MOVE-FA data along with additional clinical and preclinical data could support an NDA submission in FA is scheduled for the first quarter. Scientific advice feedback from the EMA on a potential submission of vatiquinone for conditional marketing authorization for Friedreich ataxia is expected in the first quarter.