Global biotechnology leader CSL Behring  announced that two hemophilia B patients were treated with the gene therapy HEMGENIX^® (etranacogene dezaparvovec) at Hemophilia Treatment Centers in France. This milestone achievement makes HEMGENIX^® the first gene therapy administered as a treatment in a real-world setting for hemophilia B in Europe.

HEMGENIX^® is the first one-time gene therapy approved in Europe for the treatment of adults with severe and moderately severe hemophilia B, an inherited bleeding disorder caused by the lack of Factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of Factor IX inhibitors.¹

Following European Commission approval, HEMGENIX^® was the first ever therapy to be granted Direct Access in France, thus enabling the first patients to be treated in Europe outside of the clinical program.

Though effective, current therapies can be time intensive and require regular treatment that can have a substantial impact on a patient’s daily life. HEMGENIX^® offers a one-time treatment, allowing people living with hemophilia B to produce their own Factor IX, which can lower the risk of bleeding.

“Only a few decades ago, gene therapy for hemophilia 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 hemophilia B community, as well as the access and reimbursement authorities, in bringing innovative therapies to patients,” said Dr Lutz Bonacker SVP and General Manager, CSL Behring Commercial Operations Europe. “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.”

HEMGENIX^® was granted conditional marketing authorisation by the European Commission (EC) for the European Union and European Economic Area in February 2023, following approval from the U.S. Food and Drug Administration (FDA) in November 2022. It has also been approved by Health Canada, the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA), Switzerland’s Swissmedic and Australia’s Therapeutic Goods Administration (TGA).

The multi-year clinical development of HEMGENIX^® was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment.