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PTC Updates On EMA Regulatory Review Timeline For AADC Deficiency Gene Therapy Program

PTC Therapeutics Inc. (PTCT), on Thursday, announced that it has completed the Scientific Advisory Group and Oral Explanation meetings for its gene therapy treatment for AADC deficiency or PTC-AADC with the Committee for Advanced Therapies or 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 or CHMP in May.

If approved, PTC's AADC gene therapy would be the first marketed gene therapy directly infused into the brain, the company said.

"We are looking forward to treating patients with our gene therapy treatment for AADC deficiency," stated Stuart Peltz, Chief Executive Officer, PTC Therapeutics. "AADC deficiency is such a terrible disease and we believe that this treatment has the potential to be transformational for patients with this disorder."

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