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Final draft guidance from the National Institute for Health and Care Excellence (NICE) has recommended a new treatment for patients aged two years and above with severe haemophilia A.
Published on 28 February 2025, the draft guidance approves efanesoctocog alfa (Altuvoct; Swedish Orphan Biovitrum), a replacement factor VIII protein administered as a once-weekly injection, for patients with the blood clotting condition.
Haemophilia A is a rare, inherited and incurable condition, which mainly affects males. Around 7,700 people in England have haemophilia A, with 1,900 people affected by severe haemophilia A.
Patients with the condition do not have enough blood clotting factor VIII, or it does not work properly, meaning they cannot form strong clots and bleed for longer than usual after an injury.
However, patients with severe haemophilia A can experience bleeding into joints and muscles without any injury. The condition is classed as severe in patients with a factor VIII activity level of less than 1%.
Currently, other factor VIII replacement therapies must be administered two to three times per week.
Helen Knight, director of medicines evaluation at NICE, said: “Severe haemophilia A is a life-long inherited condition that can be life-threatening. It can impact a person’s ability to carry out normal daily activities because of the risk of suffering a bleed.
“Current factor VIII replacements can be difficult to manage due to the need for frequent dosing to prevent potentially life-threatening and debilitating bleeding episodes. Efanesoctocog alfa only has to be taken once a week. Combined with its effective bleeding control, it has the potential to have a significant positive impact for some people with severe haemophilia A.”
James Palmer, medical director for specialised services at NHS England, said: “This is an important step forward in treatment for patients with severe haemophilia A and ensures they continue to have access to the latest advances in care on the NHS to help prevent bleeding episodes.
“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, reinforcing the NHS’s commitment to providing innovative treatments at value for the taxpayer.”
Kate Burt, chief executive of the Haemophilia Society, commented: “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.”
Final NICE guidance is due to be published on 26 March 2025.
