Posted by Michael Wonder on 27 Apr 2017
uniQure receives European Medicines Agency Priority Medicines (PRIME) designation for AMT-060 in haemophilia B
25 April 2017 - uniQure today announced that AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B, has received PRIME designation by the EMA.
This designation is based on results from the ongoing, dose-ranging Phase 1-2 study that show a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up, clinically significant and sustained increases in Factor IX (FIX) and substantial reductions in FIX replacement usage.
