Posted by Michael Wonder on 31 Jan 2017
uniQure announces FDA breakthrough therapy designation for AMT-060 in haemophilia B
30 January 2017 - uniQure today announced that AMT-060, its proprietary, investigational gene therapy in patients with severe haemophilia B, has received breakthrough therapy designation by the U.S. FDA.
This designation is based on results from the ongoing, dose-ranging Phase 1-2 study that show sustained increases in Factor IX (FIX), reductions in FIX replacement usage and a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up.
“We are very pleased the FDA has designated AMT-060 a breakthrough therapy for patients with haemophilia B,” stated Matthew Kapusta, chief executive officer of uniQure. “The FDA’s decision to prioritize and expedite the review of AMT-060 is an important milestone for uniQure and we are committed to working closely with the FDA to rapidly advance our haemophilia B program into late-stage development.”
