PTC Receives Positive CHMP Opinion For Upstaza For Treatment Of AADC Deficiency

(RTTNews) - PTC Therapeutics Inc. (PTCT) said that Upstaza received a positive opinion from the Committee for Medicinal Products for Human Use or CHMP of the European Medicines Agency or EMA.

Once ratified by the European Commission, Upstaza will be the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency for patients 18 months and older and the first marketed gene therapy directly infused into the brain, the company said in a statement.

PTC expects the European Commission to ratify the marketing authorization for Upstaza under exceptional circumstances in about two months. The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.