NHS England has announced that it will commission a life-changing prophylatic therapy for individuals with severe congenital haemophilia A without factor VIII inhibitors. The new treatment offered is a part of the measures outlined in the NHS Long Term Plan.
The new drug emicizumab (Hemlibra) mimicks the action of the blood protein factor VIII. It binds to the factor X and activated factor IX which, bringing them close to each other and activates the clotting system even in the absence of factor VIII.
The dose of emicizumab is calculated based on the patient’s weight. The loading dose is administered intravenously once a week for the first 4 weeks, followed by a maintenance dosing schedule of once every 2 weeks or once every 4 weeks.
Evidence from the HAVEN 3 trial of patients with severe haemophilia previously treated with on-demand factor VIII showed significantly lower annualised bleeding rates in individuals receiving emicizumab compared with those receiving no prophylaxis. None of the serious adverse events in the trial were found to have a causal link with emicizumab.
Around 2000 individuals in the country afflicted with this condition are expected to receive the drug through the NHS. These include several young children whose parents often find it challenging to administer multiple infusions every month.
Simon Stevens, Chief Executive of NHS England, said: "This treatment has the potential to significantly improve the lives of people with haemophilia, especially children – reducing treatment time and even ending the dangerous bleeds which can lead to life-threatening cuts and life-changing damage."