PTC Therapeutics Receives Positive Opinion for Upstaza

PTC Therapeutics announced on May 20, 2022 that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted a positive opinion of Upstaza, a treatment for aromatic L-amino acid decarboxylase (AADC) deficiency for patients 18 months and older. The treatment will be the first marketed gene therapy directly administered into the brain.

EMA has recommended the treatment for regulatory approval by the European Commission (EC). PTC anticipates the EC 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.

“We are thrilled with the positive opinion from the CHMP, and are eager to bring Upstaza to patients living with AADC deficiency," said Stuart W. Peltz, PhD, CEO of PTC Therapeutics. "Upstaza will be the first marketed gene therapy that is directly administered into the brain, the first gene therapy approved in a major market in several years, the third gene therapy that is on the market now, and only the fourth in vivo gene therapy ever approved. It's important for the biotech community to have gene therapy products achieving approvals at regulatory bodies, as well as it being an important milestone for PTC that will help us build the gene therapy franchise and grow our revenue base.”