EMA review first novel gene therapy candidate for haemophilia B

Gene therapy

CSL Behring has today announced that the EMA has accepted the Marketing Authorising Application for etranacogene dezaparvovec (EtranaDez), under its accelerated assessment procedure. If approved, etranacogene dezaparvovec will provide people living with haemophilia B in the EU and European Economic Area (EEA) with the first-ever gene therapy treatment option that significantly reduces the rate of annual bleeds after a single infusion.

read more