Hemophilia A is a condition of increased tendency to bleed due to an inherited deficiency of factor VIII, a protein that aids in blood-clotting. Hemophilia A has X-linked recessive inheritance, and so predominately affects males. It is the most common of the hemophilias with an incidence of one in 5,000 male births. Patients with hemophilia A, particularly those with severe disease, are at risk for life-threatening bleeding, including intracranial bleeding, but bleeding into a joint (hemarthrosis) or muscle is more common and can lead to substantial disability. Hemarthroses cause ongoing joint inflammation and damage and also increase the likelihood of further bleeding into the same joint.
Interventions of Interest:
- Factor VIII: Factor VIII concentrate is given intravenously, whether administered on-demand or prophylactically. Prophylaxis is administered multiple times per week, which is burdensome.
- Emicizumab (Hemlibra®, Genentech): a monoclonal antibody with dual targets that allow it to bridge activated factor IX and factor X, the role normally played by activated factor VIII in the clotting cascade. Emicizumab was approved by the US Food and Drug Administration (FDA) as a prophylactic treatment for hemophilia A in patients who have inhibitors to factor VIII in 2017 and in those without inhibitors in 2018.
- Valoctocogene roxaparvovec (Roctavian ™; BioMarin): gene therapy with valoctocogene roxaparvovec results in factor VIII production in the liver, but not in the cells in the liver that normally produce factor VIII.
Below you will find the final documents from the assessment review process: