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Using a combination of genetic and functional approaches, scientists have identified new mutations in parts of the F8 gene that do not code for factor VIII (FVIII) in a small group of French and Canadian patients with hemophilia A. These results were described in the study, “The…

Hemlibra (emicizumab) continues to safely and effectively prevent bleedings in children and adults with hemophilia A, regardless of developing neutralizing inhibitors (antibodies) against synthetic factor VIII (FVIII), according to nearly three years of data from four Phase 3 clinical trials. These and preliminary real-world safety findings from a European…

The full range of hemophilia treatments should be made available to patients without consideration of cost or other barriers, leaders of patient associations and others argued at a public meeting hosted by the Institute for Clinical and Economic Review (ICER) to consider its recent analysis of hemophilia A therapies. The…

People living with bleeding disorders such as hemophilia and von Willebrand disease report consistently high levels of satisfaction with the care they receive at U.S. hemophilia treatment centers (HTCs), according to the results of the U.S. HTC Network’s first national survey. Most respondents said they were satisfied with…

Preventive treatment with Hemlibra (emicizumab) improves health-related quality of life (HQRoL) in children with hemophilia A who develop inhibitors, or neutralizing antibodies, against engineered factor VIII (FVIII), an essential blood-clotting protein, a new analysis of a Phase 3 trial shows. Such Hemlibra therapy also reduces the burden on…

At standard U.S. doses, Hemlibra (emicizumab) provides comparable or even superior clinical benefits than prophylactic factor VIII (FVIII) replacement therapy among adults with severe hemophilia A and no inhibitors, according to a report from the Institute for Clinical and Economic Review (ICER). Hemlibra is also cost-saving, due to…