CSL Behring said that two patients with haemophilia B received its gene therapy Hemgenix (etranacogene dezaparvovec) at Hemophilia Treatment Centers in France.
This marks the first instance of Hemgenix being administered as a treatment for haemophilia B in a real-world setting in Europe.
CSL Behring SVP and commercial operations Europe general manager Lutz Bonacker said: “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 Hemgenix 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.
“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.”
In February 2023, the European Commission granted conditional marketing authorisation (CMA) for Hemgenix to treat severe and moderately severe haemophilia B in adults without a history of Factor IX inhibitors.
Hemgenix is the first one-time gene therapy approved in Europe for this indication, addressing an inherited bleeding disorder caused by a deficiency in Factor IX, a crucial protein for blood clotting.
The gene therapy utilises an adeno-associated virus five (AAV5)-based gene therapy approach, delivering the Factor IX-Padua gene variant. This variant produces Factor IX proteins that are significantly more active, five to eight times higher than normal levels.
Hemgenix was previously known as CSL222 and AMT-061. It was developed by uniQure and later transitioned to CSL Behring for commercialisation rights.
Approved by the US Food and Drug Administration (FDA) in November 2022, Hemgenix has also received approvals from Health Canada, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), Swissmedic, and Australia’s Therapeutic Goods Administration (TGA).
