PTC Therapeutics provides regulatory updates

PTC Therapeutics submitted a BLA to the FDA for Upstaza, a gene therapy for the treatment of aromatic L-amino acid decarboxylase deficiency, a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life. In addition, based on recent feedback from the FDA, PTC plans to re-submit an NDA for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy, a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20’s. PTC expects to make the NDA resubmission by mid-year. In addition, the sepiapterin MAA for PKU remains on schedule for submission to the EMA by the end of this month. The efficacy and safety profile of Upstaza has been demonstrated across clinical trials and compassionate use programs. The first patient was dosed in 2010. Upstaza has received marketing authorization in Europe, Great Britain and Israel.