PTC Therapeutics Receives Positive Opinion for Upstaza

News
Article

PTC Therapeutics has received a positive opinion from EMA for Upstaza for the treatment of AADC deficiency.

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.”

Source: PTC Therapeutics

Recent Videos
Miguel Forte from ISCT and Kiji Therapeutics talks about the potential impact of a changing European political landscape.
Miguel Forte from ISCT and Kiji Therapeutics provides his insights into the changing political landscape in the US as well as legislative and regulatory adjustments
Miguel Forte from ISCT and Kiji Therapeutics chats about expectations for 2025 and the future technology agenda for industry.
Sheryl Johnson from Orbia Fluor & Energy Materials chats about gender diversity, how women are helping to advance innovation, sustainability challenges, and progress in the field of inhaled drugs.
Mike Baird from Schlafender Hase gives his predictions for how AI and ML may find use in the industry moving forwards and provides some predictions about M&A and the changing US government administration.
Mike Baird from Schlafender Haser discusses industry trends from 2024 and those expected to have an impact in 2025 from the perspective of a software developer.
Preeya Beczek from Beczek.COM gives her thoughts on the areas to watch with the new US administration and how Europe might be finalizing preparations for previous legislative changes