Earlier today, the Scottish Medicines Consortium (SMC) confirmed that a new gene therapy treatment option for patients with moderately severe or severe haemophilia B has been provisionally accepted for use in Scotland.
The treatment, Etranacogene dezaparvovec—known by its brand name Hemgenix—will be the first gene therapy of its kind available on the NHS in Scotland. It is intended for adults with severe or moderate-severe haemophilia B who have no current or historic inhibitors.
Alan Martin, Director of Haemophilia Scotland, commented: “Today’s decision to recommend the use of etranacogene dezaparvovec marks a significant moment for the treatment of haemophilia B in Scotland. This new medicine has the potential to significantly improve the quality of life for those eligible by reducing the need for constant infusions. This could potentially enable them to live their lives and take part in activities previously denied to them with a reduced fear of bleeds or long-term joint damage.
This decision symbolises to the haemophilia community that the latest medical treatments and innovations will be made available to those who can benefit from them. We congratulate the Scottish Medicines Consortium and NHS Scotland for taking this positive step forward.”
Haemophilia B is a rare condition where the blood doesn’t clot properly. This happens because the body doesn’t have enough of a protein called Factor IX, which is needed to stop bleeding. People with haemophilia B can bleed more easily, even from small injuries, and may have bleeding inside their joints, causing pain and damage over time. Current treatment for the condition involves regular infusions of
There are just over 200 people in Scotland living with haemophilia B, and a proportion of them will be eligible for consideration for gene therapy. If the gene therapy treatment is successful, the patient’s factor levels will reduce to the point that they no longer need regular infusions to prevent bleeds for several years and potentially longer. This marks a significant advancement in managing haemophilia B, reducing the burden of treatment, offering greater freedom to take part in more social and physical activities, and improving overall quality of life.
Haemophilia Scotland will provide further updates on this and other new treatments expected to be under review later this year, as more information becomes available.