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European Medicines Agency Commences Review of Novel Gene Therapy Candidate Etranacogene Dezaparvovec for People with Hemophilia B
Marketing Authorization Application (MAA) for etranacogene dezaparvovec will be reviewed under accelerated assessment and has the potential to be the first
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[{"type":"text","content":"Marketing Authorization Application (MAA) for etranacogene dezaparvovec will be reviewed under accelerated assessment and has the potential to be the first gene therapy for patients living with hemophilia B\nMAA filing is supported by the phase 3 HOPE-B study which demonstrated durable and sustained therapeutic effect after a single infusion\nMilestone underscores CSL Behring's promise to develop and deliver a truly unique portfolio of patient-focused therapies\nMARBURG, Germany, March 28, 2022 /PRNewswire/ -- Global biotherapeutics leader CSL Behring today announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for etranacogene dezaparvovec (EtranaDez) under its accelerated assessment procedure. Etranacogene dezaparvovec is an investigational adeno-associated virus five (AAV5)-based gene therapy administered as a one-time treatment for hemophilia B patients with a severe bleeding phenotype. 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 that significantly reduces the rate of annual bleeds after a single infusion. Accelerated assessment potentially reduces the timeline once the MAA is accepted for review and is provided for a medicinal product when the therapy is expected to be of major public health interest, particularly pertaining to therapeutic innovation.\n\n \n \n \n \n \n \n\n \n\"As the first gene therapy candidate for hemophilia B, this pivotal regulatory milestone brings CSL Behring one step closer to delivering on the promise of gene therapy for the bleeding disorders community,\" said Emmanuelle Lecomte Brisset, Head of Global Regulatory Affairs at CSL Behring. \"We look forward to working with regulatory authorities to bring the transformative potential of gene therapy to people living with this debilitating, life-long condition.\"\nThe MAA is supported by positive findings from the pivotal HOPE-B trial, the largest gene therapy trial in hemophilia B to date. Hemophilia B patients classified as having a severe bleeding phenotype treated with etranacogene dezaparvovec demonstrated reduced adjusted annualized bleed rate (ABR) by 64% and demonstrated superiority to prophylaxis treatment at 18 months post-treatment compare...