For the first time, CSL Behring has treated two haemophilia B patients with its proprietary gene therapy Hemigenix (etranacogene dezaparvovec) at specialist haemophilia treatment centre in France.
This is the first gene therapy to ever be administered in a real-world setting for adult patients with severe haemophilia B in Europe.
To gain approval for the administration of Hemigenix, CSL Behring applied through the European Commission, receiving limited marketing approval in February 2023. This allowed the company to administer the immunotherapy outside of clinical trials.
Patient challenges associated with haemophilia
Haemophilia B is a congenital, inherited disease that impacts the body’s ability to respond to bleeding by clotting due to a lack of Factor IX.
It can have a significant impact on the a patient’s daily life, with current therapies proving time intensive to those who receive it.
Hemigenix could offer as a solution to this issue, as it is a one-time treatment that will stimulate the production of Factor IX in patients, reducing the risks associated with bleeding.
“Only a few decades ago, gene therapy for haemophilia was a distant concept, which has now become reality. Accordingly, the first two patients treated with Hemigenix since receiving European approval is a major accomplishment and a testament to the joint commitment of the haemophilia B community, as well as the access and reimbursement authorities, in bringing innovative therapies to patients,” said SVP and General Manager, CSL Behring Commercial Operations Europe, Dr Lutz Bonacker.