Pharma Focus Asia

PTC Therapeutics Receives Positive CHMP Opinion for Upstaza™ for the Treatment of AADC Deficiency

Monday, May 23, 2022

PTC Therapeutics, Inc. announced that Upstaza™ (eladocagene exuparvovec; PTC-AADC) received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) 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 (AADC) deficiency for patients 18 months and older and the first marketed gene therapy directly infused into the brain.

"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, Ph.D., Chief Executive Officer, 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."

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.1,2

"The difference Upstaza, a one-time gene therapy, can make is life-changing," said Paul Wuh-Liang Hwu, M.D., Ph.D., Lead Investigator, National Taiwan University Hospital. "AADC deficiency is a devastating neurological disorder with no effective treatment. Before therapy, affected children couldn't even lift their head, but now many can sit, stand with help, feed themselves and some can walk and talk."

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.

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