CSL Behring has announced the treatment of two patients with HEMGENIX (etranacogene dezaparvovec) gene therapy for haemophilia B, an inherited bleeding disorder.
The patients were treated at haemophilia treatment centres in France.
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HEMGENIX’s multi-year clinical development was spearheaded by uniQure, with the trials’ sponsorship transitioning to CSL Behring after it acquired the global rights to commercialise the asset.
This gene therapy received conditional marketing authorisation from the European Commission for the European Union and European Economic Area came in February last year.
A one-time treatment, gene therapy is approved in Europe for treating adult patients with severe and moderately severe haemophilia B who do not have a history of Factor IX inhibitors.
Subsequent to the approval by the European Commission, HEMGENIX was granted Direct Access in France.
Existing therapies for haemophilia B can be burdensome, requiring frequent administration and having a significant impact on patients’ lives.
HEMGENIX offers a one-time treatment option that enables individuals with haemophilia B to produce their own Factor IX, potentially reducing the risk of bleeding episodes.
CSL Behring Commercial Operations Europe senior vice-president and general manager Dr Lutz Bonacker said: “Only a few decades ago, gene therapy for haemophilia was a distant concept, which has now become a reality. Accordingly, the first two patients treated with HEMGENIX since receiving European approval is a major accomplishment and a testament to the joint commitment of the haemophilia B community and the access and reimbursement authorities in bringing innovative therapies to patients.
“This milestone has been made possible by the innovative Direct Access scheme adopted in France, allowing patients to benefit from early access to pioneering treatments. We are encouraged to see increasing access to gene therapies in European countries and are fully committed to ensuring that access to potentially life-changing treatment continues.”
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