Shots:

• The P-III (HOPE-B) trial evaluates the safety & efficacy of etranacogene dezaparvovec vs FIX prophylactic therapy in 54 patients with hemophilia B
• The trial achieved the pre-specified 1EPs of non-inferiority in ABR @18mos. over baseline FIX prophylactic therapy. The 2EPs showed a reduction in ABR, stable & durable FIX levels with mean FIX activity of 36.9% in the study population as measured by one-stage APTT-based clotting assay @18mos. post-infusion compared to FIX activity of 39.0% @6mos., was well-tolerated
• Etranacogene dezaparvovec received the BTF from the US FDA & access to a PRIME regulatory initiative by EMA. CSL Behring plans for regulatory submissions in the US & EU in H1’22

Ref: Globe Newswire | Image: uniQure