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CHMP recommends Upstaza for aromatic L-amino acid decarboxylase (AADC) deficiency.- PTC Therapeutics

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Published:22nd May 2022

PTC Therapeutics, Inc. announced that Upstaza (eladocagene exuparvovec; PTC-AADC) received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

Once ratified by the European Commission, Upstaza will be the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency for patients 18 months and older and the first marketed gene therapy directly infused into the brain.

The CHMP opinion is based on the findings of clinical studies conducted in Taiwan. In addition, data from the compassionate use treatment of patients in Europe were included in the application. In the clinical studies, patients went from no display of any motor milestone development to developing clinically meaningful motor skills and neuromuscular function from as early as three months following treatment, with transformational improvements shown to continue up to nine years after treatment. Cognitive and communication skills improved in all treated patients.

Condition: Aromatic L-Amino Acid Decarboxylase Deficiency
Type: drug

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