Challenges in the management of Haemophilia A
Haemophilia A is an inherited bleeding disorder where a person’s blood does not clot properly due to a lack or insufficient levels of a blood clotting protein known as factor VIII.
Learn more about Roche’s involvement in haemophilia.
Prophylaxis with intravenous FVIII replacement has been the standard of care for severe hemophilia A (HA). Emicizumab, a humanized bispecific FIXa- and FX-directed antibody, is approved for treatment of HA with or without inhibitors in the US. This study aimed to assess preferences for treatment characteristics for HA therapies in patients without current FVIII inhibitors and their caregivers using a discrete-choice experiment (DCE) survey.
Emicizumab has demonstrated efficacy in bleed prevention when administered subcutaneously 1.5 mg/kg once weekly, 3 mg/kg every two weeks (Q2W), and 6 mg/kg every four weeks (Q4W) in persons with haemophilia A (PwHA) with or without FVIII inhibitors. These dosing regimens were selected based on an exposure-repeated time-to-event model developed from bleed data collected in a phase I/II study in a small group of PwHA receiving weekly emicizumab doses of either 0.3, 1 or 3 mg/kg. This analysis presents the updated exposure–response relationship for bleeding events using a much larger database including data from PwHA from phase I/II and phase III studies.
The safety and efficacy of emicizumab has been demonstrated in persons with haemophilia A (PwHA) with or without FVIII inhibitors across all age groups in the HAVEN 1–4 studies. This presentation details the long-term efficacy and safety of emicizumab across the HAVEN 1–4 studies.