New Haemophilia A Treatment Endorsed by NICE
The National Institute for Health and Care Excellence (NICE) has published a final draft guidance recommending efanesoctocog alfa for treating and preventing bleeding episodes in people aged 2 years and above with severe haemophilia A. The decision follows the treatment’s recent licensing in the United Kingdom.
An estimated 7700 people in England have haemophilia A, with around 1900 classified as severe cases where their factor VIII activity level is less than 1%. Current treatments involve factor VIII injections every 2-3 days to prevent bleeds.
“Current factor VIII replacements can be difficult to manage due to the need for frequent dosing,” highlighted Helen Knight, director of medicines evaluation at NICE, in a statement.
Efanesoctocog alfa is a fully recombinant fusion protein with a longer half-life than existing standard or extended half-life factor VIII products. It requires only a weekly injection, offering greater convenience and effective bleeding control. NICE emphasised that this simpler dosing regimen could significantly improve patients’ quality of life.
Kate Burt, chief executive of the Haemophilia Society, welcomed the decision. “This product could significantly improve the lives of some adults and children with severe haemophilia A,” she said. The treatment could provide “better protection against bleeds with less frequent infusions, helping people focus on living their lives rather than managing their bleeding disorder.”
Superior Bleeding Prevention
Current treatment options for severe haemophilia A include ongoing treatment with factor VIII replacement therapies — including standard half-life and extended half-life therapies — or emicizumab to prevent bleeding. On-demand factor VIII replacement therapies are used to treat bleeds.
NICE’s recommendation was based on clinical data from XTEND-1 , a phase 3 open-label non-randomised trial. The study involved patients aged 12 years and over with severe haemophilia A and no inhibitors to factor VIII. Researchers found that once-weekly efanesoctocog alfa provided superior bleeding prevention compared to pre-study prophylaxis, as well as near-normal factor VIII activity. Patients also reported improvements in physical health, pain, and joint health.
Cost and Access
The dose of efanesoctocog alfa is based on a person’s weight. For an adult weighing 81.8 kg, the annual treatment cost would be around £510,000 at its list price. However, the company had agreed a confidential discount.
The Haemophilia Society noted that the therapy could offer prolonged factor VIII activity compared to current treatments, reducing the “burden of treatment.” “Higher factor levels between doses and higher troughs should provide better protection from bleeds and make it easier to participate in sports and other activities,” the organisation stated.
Professor James Palmer, National Health Service England’s medical director for specialised services, welcomed the approval. “This condition can have really debilitating and painful impacts, and it’s fantastic news that this time-saving therapy will now be available to help improve patients’ lives,” he said.
Dr Rob Hicks is a retired NHS doctor. A well-known TV and radio broadcaster, he has written three books and has regularly contributed to national newspapers, magazines, and online. He is based in the UK.
