CSL Behring Announces First Two Patients Treated with HEMGENIX (etranacogene dezaparvovec) Gene Therapy for Haemophilia B in Europe
July 4, 2024 – Pharmaceutical – CSL, haemophilia B, treatment
4 July 2024, Marburg, Germany– Global biotechnology leader CSL Behring (ASX: CSL) today announced that two haemophilia B patients were treated with the gene therapy HEMGENIX (etranacogene dezaparvovec) at Haemophilia Treatment Centers in France. This milestone achievement makes HEMGENIX the first gene therapy administered as a treatment in a real-world setting for haemophilia B in Europe.
HEMGENIX is the first one-time gene therapy approved in Europe for the treatment of adults with severe and moderately severe haemophilia B, an inherited bleeding disorder caused by the lack of factor 9 (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of factor 9 inhibitors.
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 haemophilia B to produce their own factor 9, 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 haemophilia 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.
About CSL: CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSL.com/Vita. Visit CSL.com.
