FDA grants Breakthrough designation for AMT-060 in hemophilia B

Dutch human gene therapy specialist uniQure (Nasdaq: QURE) says that AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B, has received Breakthrough Therapy designation by the US Food and Drug Administration.

This designation is based on results from the ongoing, dose-ranging Phase I/II 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.

All 10 patients have shown improvement, with levels of clotting factors remaining constant for at least 31 weeks, and only one patient has suffered a spontaneous bleed. None has developed antibodies against clotting factor IX or the viral vector.