Early Access for patients with inhibitors to Emicizumab
The MHRA has announced that Emicizumab (previously known as Ace910 and now marketed in the USA as Hemlibra) can be provided to Haemophilia A patients with current inhibitors under the Early Access to Medicines Scheme.
As of Tuesday, the 2nd January 2018, it is 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 most likely be patients who have unsuccessfully had ITI (immune tolerance induction) treatment or who are still experiencing frequent bleeds due to their inhibitor.
If you think you or your child would benefit from this treatment you should discuss it with your haemophilia centre. There is a short window in the next few months to apply for the early access prior to any decisions on commissioning. This is a new treatment which will require close monitoring and evaluation and may not be suitable for everyone.
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. It is currently only being considered in the UK for treatment of people with current inhibitors.
Emicizumab is produced by Chugai, a subsidiary of Roche Pharmaceuticals.
More information is available here which includes information for healthcare professionals on how to register patients for the early access scheme.