Fitusiran prophylaxis in severe haemophilia without inhibitors

Haemophilia is a congenital X-linked bleeding disorder caused by deficiency of coagulation factors VIII (haemophilia A) or IX (haemophilia B). Recurrent joint bleeding, leading to the development of arthropathy, is the hallmark of haemophilia and is more pronounced in people with moderate to severe forms of the disease (<1–5% of normal factor VIII or IX concentrations).1 Prophylaxis is the mainstay therapy for enhancing haemostasis sufficiently to prevent and control bleeding events in people with moderate to severe haemophilia.