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 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)…

Altuviiio (efanesoctocog alfa) reduced bleeding and was safe for boys and young men with severe hemophilia A, according to a recent study. Researchers also reported an increase in participants’ confidence in everyday activities and no return of neutralizing antibodies that may inhibit treatment, even in patients who are usually…

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…

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…

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…

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…

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…

Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively reduces bleeding rates in children younger than 12 with hemophilia type A, regardless of development of inhibitors (antibodies) against synthetic factor VIII (FVIII), data from two Phase 3 studies show. Hemlibra is a type of replacement therapy that mimics…