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

PTC Therapeutics Inc 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.