Historical times! First-ever hemophilia B gene therapy to apply for a license.

The Coalition for Hemophilia B is excited to report that on March 28, 2022, CSL Behring announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for their drug etranacogene dezaparvovec (EtranaDez) EtranaDez is an investigational adeno-associated virus five (AAV5)-based gene therapy administered as a one-time treatment for people with hemophilia B patients with a severe bleeding phenotype. If approved, it will provide people living with hemophilia B in the European Union (EU) and European Economic Area (EEA) with their first-ever gene therapy treatment option. The review will take place under accelerated assessment which will potentially reduce the timeline for approval.

The application is supported by encouraging findings from the HOPE-B trial, the largest gene therapy trial in hemophilia B to date. Hemophilia B patients classified as having a severe bleeding phenotype treated with EtranaDez demonstrated reduced adjusted annualized bleed rate (ABR) by 64% and demonstrated superiority to prophylaxis treatment at 18 months post-treatment compared to a 6-month run-in period. Additionally, there were stable and durable increases in mean Factor IX (FIX) activity levels.

CSL Behring’s announcement with more information on the application is available here:

https://www.prnewswire.com/news-releases/european-medicines-agency-commences-review-of-novel-gene-therapy-candidate-etranacogene-dezaparvovec-for-people-with-hemophilia-b-301511443.html

The Coalition for Hemophilia B will continue to keep the community informed of this and other developments in new therapies for hemophilia B through our website, in publications, and on social media.