The introduction of prescription medication, emicuzimab, for haemophilia A has transformed the treatment of patients with the disorder, improving their quality of life and reducing the need for additional treatments and hospitalisations. To tie in with World Haemophilia Day which took place on 17 April, Associate Professor Chris Barnes, consultant haematologist at the Royal Children’s Hospital Precinct in Melbourne, explains why emicuzimab has become an attractive option for people with haemophilia A. patients.

“Haemophilia A is a genetic disorder which is caused by having low levels of a protein called factor VIII, which is needed to form blood clots. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. Until now, the primary therapy for haemophilia A has been replacement therapy, which involves replacing the missing blood clotting factor intravenously. However, this procedure can be invasive and painful. Young people in particular, require regular infusions of clotting factor concentrates to prevent bleeding episodes, which can be a significant burden and adversely affect their quality of life. Frequent factor administration can also lead to vein damage and scarring.

“The recent emergence of emicuzimab has revolutionised haemophilia A care. Unlike traditional clotting factor concentrates, emicuzimab is administered subcutaneously, into the fatty tissue rather than straight into the vein, and has a longer half-life, allowing people to receive less frequent injections. Subcutaneous injections of emicuzimab may be given weekly, fortnightly or monthly, unlike replacement therapy which is typically required two or three times a week. This can significantly reduce the burden of treatment and improve a patient’s quality of life,” said A/Prof Barnes.

Emicuzimab is a bispecific monoclonal antibody, an artificial protein which have two binding sites and can bind to two different antigens at the same time. By binding to both factor IXa and factor X, it can mimic the activity of factor VIII. It has been shown to be highly effective in reducing bleeding episodes and consequently, the need for additional treatments and hospitalisations.

“Some patients with haemophilia develop antibodies to factor VIII or IX, which is a significant complication of traditional haemophilia A treatment. Patients with these inhibitors require high doses of clotting factor concentrates, which can be expensive and require frequent monitoring. The bispecific antibody structure of emicuzimab means that it can be effective in the management of patients with inhibitors to factor VIII.

“It is clear that emicuzimab has the potential to become the standard of care for haemophilia A patients, and its widespread use is likely to have a significant impact on healthcare systems and the lives of patients worldwide,” said A/Prof Barnes.