First patients treated with HEMGENIX® (etranacogene dezaparvovec) gene therapy for haemophilia B in Denmark
Natalia Elliot | January 21, 2025 | News story | Sales and Marketing | CSL, Denmark, genetic, haemophilia, treatment
21 January 2025 – Copenhagen, Denmark – CSL Behring Denmark today announced that the first Danish patients with haemophilia B were treated with the gene therapy HEMGENIX® (etranacogene dezaparvovec).
HEMGENIX® is the first one-time gene therapy to be 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 IX (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of Factor IX inhibitors.
“The first patients in Denmark to receive HEMGENIX® (etranacogene dezaparvovec) mark a historic moment. Seventy-two years ago, it was discovered that inherited haemophilia B is caused by a genetic defect that deprives the liver of the capacity to produce enough functional Factor IX. Today, treating haemophilia B by restoring this capacity to the liver has become part of clinical practice in Denmark. This means that recurring factor-concentrate injections, repeated numerous times, can be replaced by a single infusion delivering the gene,” said Fredrik Sjöö, MD, PhD, Head of Medical Affairs, Nordic Region, CSL Behring. “This is what scientific progress in medicine is intended to achieve: improving the life of patients, in particular those with rare diseases, by providing innovative therapeutic options.”
Patients in Denmark can access HEMGENIX® through an innovative outcome-based agreement with Amgros, finalised in October 2024.2 This makes Denmark the first Nordic and European country to adopt a performance-based model, where costs are incurred only as long as the gene therapy proves effective over the agreed long-term period.
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 UK’s Medicines and Healthcare products Regulatory Agency (MHRA), Switzerland’s Swissmedic, and Australia’s Therapeutic Goods Administration (TGA).
Following these approvals, CSL Behring is working with relevant stakeholders to continue making HEMGENIX® available across Europe, building on milestone access decisions in Spain, Denmark, the UK (including Scotland), and Austria, so that more patients are able to receive 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.
Related Content
Novo Nordisk presents promising findings for haemophilia treatment
Novo Nordisk has presented data indicating that a direct switch to Mim8 (denecimig) prophylaxis treatment …
Novo Nordisk Foundation expands ambitious Challenge Programme internationally
The Novo Nordisk Foundation Challenge Programme, a leading open competition grant programme, invites researchers outside …
Qfitlia approved by FDA to treat haemophilia A or B
The US Food and Drug Administration (FDA) has approved Qfitlia (fitusiran) as the first therapy …
