CSL

CSL’s Hemgenix (etranacogene dezaparvovec) has been authorised as the first gene therapy for adults with severe and moderately severe haemophilia B (congenital Factor IX (FIX) deficiency) in Great Britain. It has been granted a marketing authorisation for patients without a history of FIX inhibitors by the UK Medicines and Healthcare products Regulatory Agency (MHRA). The […]

The European Commission has granted conditional marketing authorisation for CSL’s Hemgenix (etranacogene dezaparvovec), the first one-time gene therapy for severe and moderately severe haemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors.   In an ongoing clinical trial, the drug reduced the rate of annual bleeds with a single infusion […]

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP).  The CHMP has recommended conditional marketing authorisation of CSL’s etranacogene dezaparvovec, a one-time gene therapy for the treatment of appropriate adults with the disease.  The therapy […]