The Danish Medicines Council recommends CSL Behring’s gene therapy HEMGENIX® for adults with severe or moderately severe haemophilia B

COPENHAGEN, DENMARK – June 24, 2024 – Global biotechnology leader CSL Behring 

(ASX: CSL) today announced that a positive recommendation for reimbursement of  HEMGENIX® (etranacogene dezaparvovec) has been released by the Danish Medicines Council for the treatment of patients with rare blood clotting disorder, haemophilia B opening the way to finalize a commercial contract with Amgros, the Danish procurement authority.1 Denmark is the first country in the Nordics to reimburse HEMGENIX®. 

HEMGENIX® is the first one-time gene therapy approved in Denmark for the treatment of severe and moderately severe haemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors.2 This approval will advance the shift away from lifelong intravenous factor IX infusions, which can significantly impact the quality of life and wellbeing of patients.3  

“Patients with haemophilia B are vulnerable to bleeds in joints, muscles, and soft tissues. Until now, patients with severe and moderately severe haemophilia B have required lifelong bleeding prophylaxis and treatment of breakthrough bleeds with repeated intravenous Factor IX infusions at regular intervals of 3 to 14 days. The bleeding tendency is sufficiently managed on this treatment schedule, but the repeated intravenous self-administration has a negative impact on patients’ quality of life and sense of identity. The one-dose gene therapy regimen has proven itself as steady long-term prophylaxis and will be an interesting kit in our toolbox over the next years,” said MD Peter Kampmann, Head of Department Hematology at Rigshospitalet, Copenhagen. “The approval of a gene therapy product in Denmark is a milestone. To the haemophilia society, the one-dose long term prophylaxis is a potential step away from the identity and the burden of chronic disease. To healthcare professionals, gene therapy in haemophilia is a herald to development and approval of other ATMPs to come.” 

The Danish Medicines Council, Amgros and CSL Behring collaborated to develop an innovative agreement, suitable with the newly created Danish ATMP approvals process, and paving the way for access to future ground-breaking therapies for Danish patients. CSL Behring is working diligently with relevant stakeholders to continue the expansion of access to HEMGENIX® in other Nordic countries and across Europe, following other milestone access pathways in France and Austria.4    

“At CSL Behring, we are dedicated to developing and delivering innovative medicines and treatment options that improve the lives of people with bleeding disorders. This agreement is an important milestone for Danish people living with haemophilia B, as well as their friends, families and caregivers,” said Lise Grove, General Manager Denmark and Iceland, CSL Behring. “The Danish Medicines Council’s recommendation of HEMGENIX® means that this groundbreaking gene therapy can now be provided to eligible patients, and we are hopeful that its value will continue to be recognised in other Nordic countries.”