EtranaDez’s Efficacy in Hemophilia B: Wolfgang Miesbach, MD, PhD


The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed new efficacy findings from the HOPE-B study.

“Significant improvements were seen in 4key domains, participants’ feelings, their work and school life, their treatments and in their future domains with reductions between 6and nearly 15 points.”

Etranacogene dezaparvovec (EtranaDez, AMT-061; CSL Behring, UniQure) reduced annualized bleeding rate (ABR) in patients with hemophilia B compared to superior to Factor IX (FIX) prophylactic therapy in findings from the final analysis of the phase 3 HOPE-B study (NCT03569891).

Investigator Wolfgang Miesbach, MD, PhD, presented the final analysis at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting, February 2-4, 2022. Miesbach is a professor and head of coagulation disorders at the Comprehensive Care Centre at University Hospital of Frankfurt, Germany. Miesbach and colleagues found that adjusted ABR was reduced by 64% (P = .0002) and FIX-treated bleeds reduced by 77% (P <.0001) over months 7 to 18 after the 6-month lead-in period. Most participants (98%) treated with the full dose of EtranaDez were able to discontinue prophylaxis.

CGTLive spoke with Miesbach to learn more about the efficacy findings seen in the HOPE-B study. He stressed that significant improvements were seen in bleeding events as well as in quality-of-life scores.

Final analysis of pivotal HOPE-B study demonstrates durable and sustained therapeutic effect of etranacogenedezaparvovec gene therapy in hemophilia B – Data presented at EAHAD 2022. News release. CSL Behring. February 4, 2022.
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