PTC Therapeutics, Inc. announced that it has completed the Scientific Advisory Group and Oral Explanation meetings for its gene therapy treatment for AADC deficiency (PTC-AADC) with the Committee for Advanced Therapies (CAT). The CAT is responsible for the scientific assessment of advanced therapies based on its expert member's assessment of the quality, safety and efficacy of the product. The CAT is scheduled to provide a draft opinion for adoption to the Committee for Medicinal Products for Human Use (CHMP) in May 2022.

If approved, PTC's AADC gene therapy would be the first marketed gene therapy directly infused into the brain. The AADC gene therapy Marketing Authorization Application is supported by the findings of three clinical studies conducted in Taiwan. In addition, data from the compassionate use treatment of two patients in Europe were included in the application.

PTC-AADC administration occurs through a stereotactic surgical procedure, a minimally invasive neurosurgical procedure used for the treatment of a number of pediatric and adult neurological disorders. PTC-AADC, is a one-time gene replacement therapy currently under regulatory review in Europe for the treatment of AADC deficiency. It is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, containing the human DDC gene.i It is designed to correct the underlying genetic defect, by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production.