NHS Broadens Access to Hemlibra Drug
Hemlibra Now Available for All Moderate haemophilia A Patients in England
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NHS England has expanded access to Roche/chugai’s subcutaneous treatment, Hemlibra (emicizumab), making it an option for all patients with moderate haemophilia A, irrespective of severity of bleeding phenotype.
A Significant Step forward in Haemophilia Treatment
Hemlibra (emicizumab) is now available as a prophylactic treatment for patients of all ages with moderate haemophilia A in England. This decision by NHS England marks a significant expansion from its previous restriction to patients at the severe end of the spectrum. Crucially, Hemlibra is the only treatment for haemophilia A currently available on the NHS that does not require intravenous infusions, offering a more convenient subcutaneous administration.
The MHRA initially licensed Hemlibra for individuals of all ages with moderate haemophilia A, who exhibit a severe bleeding phenotype and do not have factor VIII inhibitors, in May 2023. Following this, Roche and Chugai collaborated with NHS England’s Clinical Policy Lead and the Specialised Blood Disorder Clinical Reference group (CRG) to establish a commissioning policy that would facilitate reimbursement for this expanded patient group.
Impact and Patient Benefits
Estimates suggest that approximately 9,700 people in the UK live with haemophilia A, with around 800 of these individuals diagnosed with moderate haemophilia A. This new policy is expected to make Hemlibra accessible to approximately 100 people with moderate haemophilia in England.
Kate Burt, chief executive of the Haemophilia Society, welcomed the development, stating, “Extending the availability of emicizumab means more people with haemophilia A will have more treatment choices, which is a very positive development.” She also expressed anticipation that Scotland,Wales,and Northern Ireland will follow suit.
“Having a treatment which can be administered through an under-the-skin injection is an option that we know our members value,” Burt added. “We’re very pleased that more people will now be eligible for this product and can discuss its suitability with their clinician.”
Hemlibra’s Journey and Future Prospects
Hemlibra first received NHS commissioning approval in 2018 for severe haemophilia A with inhibitors. Its use was subsequently broadened to patients without inhibitors in 2019. The treatment functions by mimicking the activity of the blood protein factor VIII, and its administration as a fortnightly injection eliminates the need for frequent, time-consuming intravenous infusions. This profile has contributed to its widespread adoption and its status as a blockbuster product generating $5 billion annually.
The positive impact of this expanded access is already being felt by families. Andrew Varley, whose two sons and father-in-law have moderate haemophilia, shared his optimism: “This will be a game changer for my family. The boys’ grandad is in his mid-70s and cannot administer his factor anymore as of severe joint damage. Being able to use emicizumab will be a godsend for him. My youngest son is 10 and uses a port-a-Cath,so I’m hoping he can transfer directly onto ’emi’.”
Looking ahead, Roche recently presented promising phase 1/2 results for NXT007, a bispecific antibody derived from emicizumab. This potential successor aims to bring together factors IXa and X, offering the possibility of enhanced potency and less frequent dosing for haemophilia patients.