Benzinga | Sep 29, 2021 12:30PM EDT

PTC Therapeutics' Rare Genetic Disease Gene Therapy Shows Durable Improvement Even At 5 Years

* PTC Therapeutics Inc (NASDAQ:PTCT) presented a new analysis of five-year results of gene therapy, PTC-AADC.

* The data exhibited improvements in children with aromatic L-Amino acid decarboxylase (AADC) deficiency.

* AADC deficiency is a rare genetic disorder that causes severe disability.

* Children treated with PTC-AADC developed motor function and cognitive skills not previously seen, such as holding up their head, sitting or standing with support, and communicating. These persisted for up to 10 years.

* In addition, the rate of respiratory infection declined from an average of 2.4 episodes per year at 12 months to 0.6 episodes per year at 2 years and 0.3 episodes per year at 5 years.

* Almost all treated children went from a baseline weight below the third percentile to making age-appropriate weight gains by 12 months following gene therapy treatment.

* PTC-AADC is currently under review by the European Medicines Agency's Committee for Medicinal Products for Human Use, with an opinion expected in Q4 of 2021.

* A Biologics License Application is expected to be submitted to the FDA by the end of 2021.

* Related Link: PTC Therapeutics/Roche's Evrysdi Approved In Europe For Muscular Atrophy.

* Price Action: PTCT stock is down 1.37% at $37.77 during the market session on the last check Wednesday.