PTC : Results Show Improvements In Children With AADC Deficiency Treated With PTC-AADC Gene Therapy

PTC Therapeutics Inc. (PTCT) presented a new analysis of five-year results that shows its gene therapy, PTC-AADC, leads to profound improvements in children with aromatic L-Amino acid decarboxylase or AADC deficiency. It is a previously intractable, fatal and devastating rare disorder of the central nervous system.

The company stated that 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, and these persisted for up to 10 years.

PTC-AADC is currently under review by the European Medicines Agency's Committee for Medicinal Products for Human Use with an opinion expected in the fourth quarter of 2021, and is an investigational new drug in the United States. A Biologics License Application is expected to be submitted to the U.S. Food and Drug Administration by the end of 2021.

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