The first patient in the UK has been treated by the NHS with CSL Behring’s haemophilia B gene therapy Hemgenix (etranacogene dezaparvovec).
The patient from the North East of England was treated at Guys & St Thomas’s NHS Foundation Trust in London and may no longer require regular infusions to help their blood clot.
Affecting more than 2,000 people in the UK, haemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of clotting factor IX (FIX).
Patients with severe cases of the disease currently require lifelong treatment with intravenous FIX, which can still leave them vulnerable to breakthrough bleeds and pain in the days before infusions.
CSL’s Hemgenix addresses the underlying genetic cause of haemophilia B by enabling the body to continuously produce FIX, and is the only one-time gene therapy to be approved in the UK for adults with severe or moderately severe haemophilia B without a history of FIX inhibitors.
Pu-Lin Luo, consultant haematologist at Guy’s and St Thomas’, said: “This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients. It is also a testament to the advancement of cell and gene therapies in the UK and these are exciting times.”
CSL Behring said it will continue to work “closely and collaboratively” with the selected gene therapy centres to ensure that further eligible patients across the country will be able to access Hemgenix.
The National Institute for Health and Care Excellence’s decision to recommend Hemgenix for use on the NHS in June 2024 was supported by positive results from the late-stage HOPE-B trial, in which a single infusion of Hemgenix demonstrated significant increases in mean FIX activity levels of 36.9% at 18 months.
These increases were sustained at 36.7% at 24 months and 38.6% at 36 months and led to an adjusted annualised bleed rate reduction of 64% at 24 and 36 months post-dose compared to the six-month lead-in period.
CSL Behring also presented updated results from the ongoing trial in February this year.
Data presented at this year’s Annual Congress of the European Association for Haemophilia and Allied Disorders showed that mean factor IX activity levels remained at near normal levels of 37% through four years post-treatment, and mean adjusted annualised bleeding rate for all bleeds was reduced by approximately 90% from lead-in to year four.