PTC Therapeutics expects EMA committee opinion on AADC deficiency gene therapy in May
- Biopharmaceutical company PTC Therapeutics (NASDAQ:PTCT) on Thursday said it has completed procedural meetings for its gene therapy treatment for (AADC) deficiency with the European Union drug regulator's Committee for Advanced Therapies (CAT).
- The CAT will provide its opinion for adoption of the therapy to the European Union drug regulator's Committee for Medicinal Products for Human Use in May.
- PTCT's gene therapy treats aromatic L-amino acid decarboxylase (AADC) deficiency, a rare and fatal genetic disorder that typically causes severe disability from the first months and years of life.