Hemgenix shows efficacy among haemophilia B patients

CSL Behring’s study reveals significant long-term benefits

New data from CSL Behring’s HOPE-B study demonstrates that Hemgenix (etranacogene dezaparvovec-drlb) gene therapy provides sustained efficacy and safety in adults with hemophilia B.

The study shows that 94% of patients eliminated factor IX prophylaxis and maintained near-normal factor IX levels through four years. This suggests that Hemgenix offers a viable long-term treatment option for haemophilia B patients.

The phase 3 study, which included 54 adult male participants, found that the mean factor IX activity levels were sustained at 37% through four years post-treatment.

Additionally, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by approximately 90% from the lead-in period to year four.

These results were presented at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders.

Professor Steven Pipe, a paediatric medical director at the University of Michigan, highlighted the significance of the findings. He said, “These results underscore the ability of Hemgenix to offer long-term bleed protection with a one-time treatment, resulting in dramatic decreases in all annual bleed rates, including joint bleeds, and sustained independence from regular prophylactic infusions.”

The study also confirmed that Hemgenix is generally well-tolerated with no serious adverse events related to the treatment.

CSL Behring’s Andres Brainsky expressed confidence in the clinical benefits of Hemgenix. He said, “These data continue to instill confidence in the clinical benefits of Hemgenix, highlighting the remarkable impact of this one-time treatment to reduce the frequency of bleeds in people with hemophilia B and improve quality of life by alleviating the burden of ongoing factor IX prophylactic treatment.”

CSL Behring plans to continue ongoing data analyses and has established a post-marketing registry to generate additional evidence on the long-term safety, efficacy and durability of Hemgenix.

The gene therapy has received various approvals, including from the US Food and Drug Administration and the European Medicines Agency.