Europe's CHMP Backs Approval Of PTC Therapeutics' Gene Therapy In Rare Neurometabolic Disorder

  • The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has recommended the approval of PTC Therapeutics Inc's PTCT Upstaza (eladocagene exuparvovec, PTC-AADC).
  • 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.
  • Related: PTC Therapeutics' Rare Genetic Disease Gene Therapy Shows Durable Improvement Even At 5 Years.
  • AADC deficiency is an inherited disorder that affects how signals are passed between certain cells in the nervous system. Signs and symptoms of AADC deficiency generally appear in the first year of life.
  • The CHMP opinion is based on clinical studies conducted in Taiwan. In addition, data from the compassionate use treatment of patients in Europe were included in the application. 
  • Price Action: PTCT shares are down 1.69% at $31.95 during the market session on the last check Friday.
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