NHSE has announced that Emicizumab (marketed as Hemlibra) can be provided to Haemophilia A patients with current inhibitors under the Framework Agreement for the supply of products for the treatment of bleeding disorders as an in year service development.
From 1st July it will be available for routine prophylaxis to Haemophilia A patients over the age of one with factor VIII inhibitors who may have unmet need from their current treatment. This will be patients who have unsuccessfully had ITI (immune tolerance induction) treatment or who are still experiencing frequent bleeds due to their inhibitor.
Liz Carroll, Chief Executive of The Haemophilia Society said:
“NHS England’s decision is a great step forward for people with haemophilia A. The development of inhibitors is a very serious and challenging complication of haemophilia treatment and there has been an urgent need for new options. This news means people with haemophilia A who have inhibitors will now have access on the NHS to a new treatment that could improve their quality of life.”
Careen, Inhibitor Ambassador at the Haemophilia Society described her experience of her son’s inhibitor and how it didn’t respond to treatment:
“For over 5 years we gained the reputation of a “compliant family”, following all the protocols and injecting our son daily, sometimes twice, three times a day with huge doses of medication that were ineffective within minutes. We hoped and prayed that one day his immune system would accept the vital medication required to clot his blood. As each medication failed, the bleeds increased and so did the need for a wheelchair, crutches, slings etc. More and more he watched his friends play by the sidelines. We tried all treatments available to us, some with the possibility of terrifying side effects. Finally we reached a time when no one had any more answers on how to stop a bleed, lets hope its not a serious one.”
“In March 2017 a new drug became available, which gave Henry the option of being involved in a trial. Once again we had to make a frightening decision for our beautiful boy who we love with all our heart. A protein that would cause his blood to clot correctly. Within three weeks of taking the medication, we were told his blood was clotting. Within six months he said he was no longer in constant pain.”
“From daily huge intravenous injections to weekly less than 3ml subcutaneous injections. Our lives have changed. We are all grateful and our son regularly rails about the magical scientists and researchers that made a medicine to make him normal and whose just been picked to play football for his school.”
If you think you or your child would benefit from this treatment you should discuss it with your haemophilia centre.
We have also set up a new email address firstname.lastname@example.org that will be managed by volunteers who have an inhibitor, or care for a child who has one, so you can contact them for support. If you are looking for medical information, please do contact your centre, but we know that being able to speak to someone else who fully understands what you are living with day to day is incredibly valuable.
Emicizumab is a novel bypassing agent delivered under the skin that replicates the role of factor VIII in clotting by binding to activated factor IX and factor X precipitating the activation of factor X.
Emicizumab is produced by Chugai, a subsidiary of Roche Pharmaceuticals.