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.
“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.”
The 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 compared to a 6-month run in period. Additionally, there was stable and durable increases in mean Factor IX (FIX) activity levels. Etranacogene dezaparvovec was specifically designed to make near-normal blood-clotting ability possible by addressing the underlying cause of the condition: a faulty F9 gene that causes a deficiency in clotting Factor IX (FIX).
“The acceptance of etranacogene dezaparvovec for review by the EMA furthers our relentless pursuit to improve the lives and well-being of those living with hemophilia B and other rare and serious medical disorders,” said Bill Mezzanotte, Executive Vice President, Head of R&D and Chief Medical Officer for CSL Limited. “We are proud to work with uniQure to be at the forefront of this scientific advancement which aims to make hemophilia B a secondary part of a patient’s life instead of a constant concern.”