Around 1,900 people suffer from haemophilia A in England (Image: Getty)
Patients with an incurable, inherited condition are set to benefit from a £510,000-a-year treatment that can prevent bleeding episodes.
Haemophilia A is a rare disorder that means sufferers do not have enough of the clotting factor VIII in their blood or it does not work properly.
This means they cannot form strong clots and bleed for longer than usual. The , efanesoctocog alfa, is given as a weekly injection.
The National Institute for Health and Care Excellence (NICE) has recommended it is offered on the to patients aged two and over with severe haemophilia A, which affects an estimated 1,900 people in the UK.
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.
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“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.”
Dosing is based on the person’s weight and the treatment at list price would cost around £510,000 per year for an adult weighing 81.8 kg.
However, manufacturer Swedish Orphan Biovitrum has agreed a confidential discount with NHS England.
Helen Knight, director of medicines evaluation at NICE, said: “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.
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“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.
“Today’s decision, which comes just a week after efanesoctocog alfa received its UK licence, shows how, by collaborating with system partners NICE can quickly recommend treatments that deliver tangible benefits for patients and the NHS alongside value-for-money for taxpayers.”