A drug that can be given as a weekly injection is set to be approved as a treatment for severe haemophilia A in England.
Efanesoctocog alfa (also called Altuvoct) has won backing in draft guidance from the National Institute for Health and Care Excellence (NICE).
NICE says the drug offers effective bleeding control and improved convenience for some patients. It received a licence in the UK three weeks ago from the Medicines and Healthcare products Regulatory Agency (MHRA).
The treatment costs £510,000 a year, but NICE said the manufacturers have agreed an undisclosed discount for the NHS. About 1,900 people in England have severe haemophilia A. The drug will be available to patients over the age of two, to treat and prevent bleeding.
Helen Knight, director of medicines evaluation at NICE, said: “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.”
NHS England medical director for specialised services Professor James Palmer 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.”
Approving the licence for efanesoctocog alfa, the MHRA referred to a study of 159 patients over the age of 12 receiving weekly injections of the drug. 65% reported no bleeding episodes during the year of the study and 35% reported much reduced bleeding episodes. The MHRA said a study of children under the age of 12 yielded similar findings.
Potential side effects include headaches and arthralgia.
Source: NICE/ MHRA
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