Obizur (susoctocog alfa) is an effective and safe therapeutic option for the treatment of severe bleeding episodes in people with acquired hemophilia A, a study in Japanese patients showed. The study “supports the use of [Obizur] as a novel therapy in the clinical management of patients with [acquired…

A major complication in scientists’ efforts to treat hemophilia A by replacing a missing or defective clotting factor is an immune response to the treatment. Researchers have discovered a connection between the body’s production of pro-inflammatory molecules known as cytokines and its immune response to clotting factor VIII therapy. They used…

The U.S. Food and Drug Administration (FDA) has approved Wilate as a prophylactic (preventive) and on-demand treatment for bleeding episodes in adults and adolescents with hemophilia A. Wilate, developed and marketed by Octapharma, is an injectable medication that contains human plasma-derived von Willebrand Factor (VWF)…

Hemophilia is a blood disorder that’s usually genetically inherited from parents. People born with hemophilia have little or no clotting factor – a protein needed for normal blood clotting. Hemophilia can also be acquired, meaning patients don’t inherit the disease from their parents but instead develop it during their lifetime. This…

A new type of gene therapy that uses RNA instead of DNA led to rapid and prolonged production of therapeutic levels of factor VIII (FVIII) in a mouse model of hemophilia A, a study reported. The study, “Treatment of…

The once-weekly injection therapy marstacimab is being considered for approval in both the U.S. and Europe for people with hemophilia A and hemophilia B who don’t have inhibitors. The U.S. Food and Drug Administration (FDA) is expected to decide on it in late 2024, while a decision from the European…

Treatment with Eloctate (efmoroctocog alfa) was well-tolerated and effective at preventing and treating bleeding episodes in children with severe hemophilia A who had never received any form of therapy for their disease, a Phase 3 trial shows. The study had enrolled boys with severe hemophilia A younger than…

The risk of spontaneous bleeding episodes in patients with severe hemophilia A receiving preventive treatment changes over time and can be influenced by treatment history and other patient-related factors, a study has found. As such, the choice of an individual preventive — or prophylactic — regimen should take…

Preventive treatment with factor VIII (FVIII) — the missing blood clotting protein in hemophilia A — was not fully effective at preventing MRI-detected joint changes in boys with severe disease, according to a Canadian study. Regular evaluation of joints by MRI or ultrasounds, however, may help in identifying those…

Prime Therapeutics and Takeda will work together to establish the value of Advate, relative to its costs and benefits, as a treatment for hemophilia A. According to Prime, work comparing factors like total health care costs and emergency hospital visits allow the company to better evaluate and distinguish…