UK regulator backs first gene therapy for hemophilia B

Having gained US approval with much fanfare in December 2022 and in Europe in February this year, Hemgenix (etranacogene dezaparvovec), from Australian biotech CSL Limited (ASX: CSL), has now received regulatory clearance in the UK

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization to Hemgenix, as the first and only single infusion gene therapy for the treatment of severe and moderately severe hemophilia B (congenital FIX deficiency) in adults without a history of Factor IX (FIX) inhibitors in Great Britain (England, Scotland and Wales).

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