EMA accepts first gene therapy for hemophilia B for accelerated assessment

The European Medicines Agency 0has accepted the Marketing Authorization Application (MAA) under accelerated assessment for etranacogene dezaparvovec (EtranaDez), a novel gene therapy candidate for the treatment of hemophilia B, developed by Dutch gene therapy company uniQure (Nasdaq: QURE) and CSL Behring, part of Australia’s CSL Limited (ASX: CSL),

This MAA is supported by the Phase III HOPE-B study, where hemophilia B patients treated with etranacogene dezaparvovec demonstrated reduced adjusted annualized bleed rate, with durable and sustained therapeutic effect after a single infusion.

If approved, etranacogene dezaparvovec will provide people living with hemophilia B in the European Union (EU) and European Economic Area (EEA) with the first-ever gene therapy treatment option, and has the potential to transform the lives of those living with the condition by eliminating the need for regular infusions.