Roche Products and Chugai Pharma UK have announced that NHS England has commissioned Hemlibra (emicizumab) for people with severe congenital haemophilia A without factor VIII inhibitors.
The decision follows NHS England’s decision to routinely fund the product for all haemophilia A patients with factor VIII inhibitors in July 2018.
Dr Gerry Dolan, consultant haematologist at St Thomas’ Hospital, said: “People with severe haemophilia A face many challenges in managing their condition. There is a need for additional treatment options for bleed prevention, so this is a major advance. Achieving zero bleeds should be the goal that patients work towards with their doctor. Access to this innovative, preventative treatment could help make that a reality for many.”
Richard Eaton, rare conditions lead at Roche Products UK, added: “We are delighted that NHS England will reimburse Hemlibra in patients with severe haemophilia A without factor VIII inhibitors. Hemlibra has been shown to effectively control bleeds and is the only prophylactic medicine that can be administered subcutaneously and maintains a sustained therapeutic level between doses. Roche and Chugai are also progressing with reimbursement discussions in Scotland, Wales and Northern Ireland.”
“This decision is fantastic news,” said Liz Carroll from The Haemophilia Society. “Current treatments can require intravenous infusions multiple times a week which can place a significant burden on people with haemophilia and their carers. We know the opportunity to have treatment less frequently without intravenous access could have a positive impact on many of our members, enabling them to live their lives to the full.” Hemlibra can be self-administered by injection under the skin at multiple dosing options (once weekly, every two weeks, or every four weeks).
In clinical trials, a high proportion of patients with Haemophilia A, both with and without inhibitors, treated with Hemlibra achieved zero treated bleeds. This increased over time, from 70.8% in week 1-24 (n=391) to 88.6% in weeks 73-96 (n=114) in the pooled study data.iii Hemlibra was generally well-tolerated.
Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, in severe cases leading to uncontrolled and often spontaneous bleeding.[vi] People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII. There is currently no cure for haemophilia A but prophylaxis with factor VIII has been recognised as the preferred treatment approach to help prevent and reduce the frequency of bleeds.
Hemlibra received marketing authorisation from the European Medicines Agency for the prevention of bleeding episodes in people with severe congenital haemophilia A without factor VIII inhibitors in March 2019 and was commissioned directly through NHS England Specialised Services.
