New haemophilia A treatment could be available this summer

Written by Sam Wilson, February 28, 2025

A new product for severe haemophilia A which reduces the number of treatments needed while increasing the length of factor cover should be available in England in July.

Today NICE, the body that considers clinical and cost effectiveness of new treatments, has published draft guidelines which recommend Sobi’s efanesoctocog alfa, also known as Altuvoct, for use in the NHS in England. We expect this to be confirmed in NICE’s final guidelines at the end of March which would mean it could start to be prescribed 90 days from that point. Availability of this product through the NHS in Wales, Scotland and Northern Ireland should follow. 

Efanesoctocog alfa can be prescribed to people with severe haemophilia A from the age of two upwards.

Helen Crawley, whose nine-year-old son Charlie has been on clinical trials for efanesoctocog alfa for three years is delighted it will be available to others. She said: ‘Previously, Charlie was treated every two days and I never felt he was completely safe on the day he wasn’t treated as he didn’t hold the factor very well. Now, he’s treated once a week, just before his football training on a Friday night and then we don’t think about it for a week. It’s unbelievable, we forget he has haemophilia.’

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. 

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 maintain 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.  

Kate Burt, Chief Executive of the Haemophilia Society, said: ‘This product could significantly improve the lives of some adults and children with severe haemophilia A. It has the potential to offer better protection against bleeds with less frequent infusions which should help people focus on living the lives they choose, rather than managing their bleeding disorder.’

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.