Wolfgang Miesbach, MD, PhD, on Future Research with EtranaDez


The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed future integration of EtranaDez into hemophilia B treatment.

“In the future, I think hemophilia centers should be able to offer gene therapy after it has been approved. And one possibility, for example, would be to initiate a hub and spoke model in which there would be a close cooperation between experienced and non-experienced centers to enable patients to be treated with gene therapy.”

The phase 3 HOPE-B study (NCT03569891) met its primary endpoint, with etranacogene dezaparvovec (EtranaDez, AMT-061; CSL Behring, UniQure) significantly reducing annualized bleeding rate (ABR) and demonstrating superiority to Factor IX (FIX) prophylactic therapy. The therapy demonstrated efficacy in patients both with and without neutralizing adeno-associated virus vector antibodies.

The final analysis of HOPE-B were presented by investigator Wolfgang Miesbach, MD, PhD, at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting, February 2-4, 2022. Miesbach serves as 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% (= .0002) and FIX-treated bleeds reduced by 77% (<.0001) after a 6-month lead-in period over months 7 to 18. Almost all participants (98%) treated with the full dose of EtranaDez were able to discontinue prophylaxis.

CGTLive spoke with Miesbach to learn more about research that remains to be conducted with EtranaDez. He discussed potential ways the therapy could be integrated into treatment for patients with hemophilia B.

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. https://www.cslbehring.com/newsroom/2022/hope-b-data-presented-at-eahad2022
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