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CSL’s first gene therapy for haemophilia B approved

Boost for effected patients as HEMGENIX receives green light from European Commission

CSL has revealed that the European Commission (EC) has granted conditional marketing authorisation for HEMGENIX.

It is a treatment for severe and moderately severe haemophilia B (congenital Factor IX deficiency) in adults who have not had a history of Factor IX inhibitors. It also becomes the first approved gene therapy for haemophilia B in the EU and European Economic Area.

During the ongoing clinical trial, HEMGENIX – also known as etranacogene dezaparvovec – successfully reduced the rate of annual bleeds with a single infusion by delivering a functional gene that acts as a blueprint for coagulation Factor IX, a protein important for blood clotting.

The EC’s decision follows a positive opinion from the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) last year, which was based on findings emerging from the pivotal HOPE-B trial – the largest gene therapy trial in haemophilia B to date.

Professor Wolfgang Miesbach, head of coagulation disorders at the Comprehensive Care Center, University Hospital of Frankfurt, was upbeat about the approval: “This approval marks an important step forward in the treatment of haemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints and internal organs, alleviating the burden of lifelong intravenous infusions of Factor IX products.”

He added: “Data from the HOPE-B study demonstrates the potential of HEMGENIX to remove the need for routine prophylaxis, by providing durable Factor IX activity, as well as improved bleeding outcomes and quality of life for people with haemophilia B.”

Dr Bill Mezzanotte, head of research and development and chief medical officer at CSL, reflected: “The approval of HEMGENIX in Europe is the essence of great science delivering a medicine that we believe can transform the treatment paradigm for both people living with haemophilia B and the healthcare professionals who treat them.”

At the moment, people living with haemophilia B require lifelong treatment of intravenous Factor IX infusions, which can have a significant impact on their quality of life and well-being.

Meanwhile, according to the CHMP, there is ‘an unmet medical need for new therapeutic approaches that might free patients from the burden of frequent infusions, or episodically at the time of a bleeding event’.

Haemophilia B is a rare, life-threatening disease. Patients with the condition are particularly vulnerable to bleeds in their joints, muscles and internal organs, leading to pain, swelling and joint damage.

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