New drug approved for inherited clotting disorder, offering simpler dosing and enhanced quality of life.
NICE publishes final draft guidance recommending efanesoctocog alfa as an option for treating and preventing bleeding in people aged 2 and over with severe haemophilia A.
Efanesoctocog alfa (also called Altuvoct and made by Swedish Orphan Biovitrum) can be given as a once-weekly injection instead of some current treatments that are given several times a week.
Affecting mostly males, haemophilia A is a rare, inherited and incurable condition that stops blood from clotting properly. People with haemophilia A do not have enough of a clotting factor called factor VIII in their blood, or it isn’t working properly. This means they cannot form strong clots and so they bleed for longer than usual. Bleeds can be the result of an injury and, when the condition is severe, bleeding into joints and muscles can happen without any injury.
Bleeds can be prevented by injections of factor VIII given every 2 to 3 days. A non-factor VIII treatment, emicizumab, is also available for people of all ages.
Around 7,700 people in England have haemophilia A with an estimated 1,900 who have severe haemophilia A.
Today’s decision, which comes just a week after efanesoctocog alfa received its UK licence, shows how, by collaborating with system partners we can quickly recommend treatments that deliver tangible benefits for patients and the NHS alongside value-for-money for taxpayers.
Helen continues, “Severe haemophilia A is a lifelong 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.
A Department of Health and Social Care spokesperson said: “We know that those living with rare diseases and their families face immense everyday challenges, and through our Rare Diseases Action Plan we aim to centre their voices to address the difficulties they face.
“We have committed to 3 new actions to improve coordination of care, access to specialised treatments and bolster research into innovative therapies for these rare conditions.
“More widely, our Plan for Change will transform the NHS by driving down waiting lists and investing in quality facilities to ensure all patients – including those with rare diseases - receive the care and treatment they deserve.”
Professor James Palmer, NHS England's Medical Director for Specialised Services 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’ commitment to providing innovative treatments at value for the taxpayer.”