Positive CHMP opinion for etranacogene dezaparvovec – gene therapy for adults with hemophilia B.- CSL Behring
Global biotechnology leader CSL announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) of etranacogene dezaparvovec.
Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds with a single infusion.If the marketing authorization for which CSL applied under the brand name Hemgenix is approved by the European Commission, etranacogene dezaparvovec would be the first gene therapy for people living with hemophilia B in the European Union (EU) and European Economic Area (EEA).
The positive CHMP opinion is based on findings from the pivotal HOPE-B trial, the largest gene therapy trial in hemophilia B to date. These findings showed that hemophilia B patients treated with etranacogene dezaparvovec demonstrated stable and durable increases in mean Factor IX (FIX) activity levels (with a mean FIX activity of 36.9%) which led to an adjusted annualized bleed rate (ABR) reduction of 64%. Following infusion of etranacogene dezaparvovec, 96% of patients discontinued routine FIX prophylaxis.
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