PTC Therapeutics, Inc. announced that Upstaza received a positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA). Once ratified by the European Commission, Upstaza will be the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase deficiency for patients 18 months and older and the first marketed gene therapy directly infused into the brain. The CHMP opinion is based on the findings of clinical studies conducted in Taiwan.

In addition, data from the compassionate use treatment of patients in Europe were included in the application. In the clinical studies, patients went from no display of any motor milestone development to developing clinically meaningful motor skills and neuromuscular function from as early as three months following treatment, with transformational improvements shown to continue up to nine years after treatment.1 Cognitive and communication skills improved in all treated patients. PTC expects the European Commission to ratify the marketing authorization for Upstaza under exceptional circumstances in approximately two months.

The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.