Healthcare
New haemophilia A treatment licensed in the UK
Written by Sam Wilson, February 14, 2025
A new product for severe haemophilia A which reduces the number of treatments needed while increasing the duration of factor cover has been licensed in the UK.
Sobi’s efanesoctocog alfa, known as Altuvoct or efa, is a new extended half-life factor VIII product for treatment of severe haemophilia A in anyone aged over two years. It is infused intravenously.
Extended half-life factor products last longer in the bloodstream which means that they can be injected less often, will maintain factor levels for longer and will have higher trough levels (the amount of factor that remains in your blood) when you take your next treatment. Efanesoctocog alfa has the potential to last far longer than the factor VIII products currently used in the UK.
Our fundraiser and member advocate, Scott McLean, has severe haemophilia A and has been using Altuvoct through clinical trials since October 2023. Previously, he treated himself every three days which reduced to once a week once he started using Altuvoct.
Scott said: ‘It’s been lifechanging. I only have to inject myself once and week and then I can forget all about treatment. I no longer feel the burden of haemophilia and can honestly say it’s the most normal I’ve felt in my entire life.’
Clive Smith, President of the Haemophilia Society, who also has severe haemophilia A, welcomed the decision. He said: ‘Altuvoct is a major leap forward in treatment for severe haemophilia A. The increased level of protection it potentially provides, along with less frequent infusions should give people with this condition far more peace of mind and, hopefully, increase their ability to live a full and active life.’
For most people with severe haemophilia A this product would mean that you would only need to infuse factor once a week. This could reduce your burden of treatment and make it easier to stay compliant with your or your child’s treatment plan.
Higher factor levels between doses and higher troughs should give better protection from bleeds and make it easier to take part in sport and other activities.
For people currently on emicizumab (Hemlibra) the decision of whether this treatment is right for you or your child will be more difficult. There have been no direct head-to-head trials between the two products, but people may want to consider how the different products suit their lifestyle. Emicizumab is delivered sub-cutaneously, meaning it is injected under the skin, which many people find more convenient. It is also able to be given once every one, two or four weeks which reduces the treatment burden compared to factor products.
In contrast to the peaks and troughs of factor replacement, emicizumab provides a steady state level of protection which is thought to be equivalent to levels of 10-15% of normal factor VIII activity, in the mild haemophilia range. Efanesoctocog alfa will allow for peaks in the normal range and average troughs of between 9-18% of normal factor VIII activity, depending on age. This could provide better bleed protection and allow people to be more active, if they are comfortable with weekly self-infusions of factor.
Efanesoctocog alfa is currently being considered for use in England by NICE, the body that considers clinical and cost effectiveness of new treatments. We are hopeful that NICE will make a positive decision to recommend the product for use in NHS in England, which could mean it is able to be prescribed later this year. Availability of efanesoctocog alfa through the NHS in Wales, Scotland and Northern Ireland should follow.
If you would like to know more about efanesoctocog alfa and are interested in considering it as a treatment option when it becomes available, you should talk to your consultant at your haemophilia centre.