EMA review first novel gene therapy candidate for haemophilia B
pharmafile | March 28, 2022 | News story | Sales and Marketing |
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.
Etranacogene dezaparvovec is an investigational adeno-associated virus five (AAV5)-based gene therapy administered as a one-time treatment for haemophilia B patients with a severe bleeding phenotype.
“As the first gene therapy candidate for haemophilia 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.”
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. MAAs pertain particularly to therapeutic innovations.
“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 haemophilia 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.
Haemophilia B is an inherited disorder in which the blood does not clot due to insufficient clotting factors. It is a life-threatening degenerative disease which makes people particularly vulnerable to bleeds in their joints, muscles, and internal organs. This can lead to pain, swelling, and joint damage, alongside tightness of joints. Haemophilia B can also cause blood in the urine or stool, and nose bleed.
Treatments can help to manage the condition, but there is no known cure.
Current treatment for haemophilia B includes life-long prophylactic infusions of FIX to temporarily replace or supplement low levels of the blood-clotting factor.
Ana Ovey
