A team of U.S. scientists discovered a naturally occurring F8 gene mutation that leads to increased activity of the factor VIII (FVIII) coagulation factor and enhanced blood clotting, offering new insights into the treatment of hemophilia A. The mutation was identified in a young man susceptible to excessive blood…

Preventive treatment with Hemlibra (emicizumab) is associated with better bleeding control and lower medication costs compared with immune tolerance induction, or ITI, among children with severe hemophilia A with inhibitors, a new study in China found. The researchers noted that use of the injection therapy Hemlibra also led…

People with hemophilia A in the U.K. will soon have greater access to Elocta (efmoroctocog alfa) under a two-year agreement between Sobi and the National Health Service (NHS), the company announced. Elocta was approved by the European Commission for the treatment and prevention of bleeding in all…

Hemophilia A patients rack up high hospital bills, driving costs for the healthcare system higher, a study from Canada showed. “This study confirms that even with improved [treatment to reduce bleeding], hemophilia A has a non-negligible impact on healthcare system costs,” researchers wrote in the study, “Population-Level Real-World…

Shire has acquired worldwide rights to the antibody-based therapy that Novimmune developed as a treatment for hemophilia A. Geneva-based Novimmune creates therapies for inflammatory diseases, autoimmune diseases and cancer. It uses antibodies that target tumor-associated proteins and the immune checkpoint protein CD47. Shire, which is based in Dublin, is a global…

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 European Medicines Agency has recommended marketing authorization for Shire’s Adynovi (BAX-855) as a treatment for adults and adolescents with hemophilia A. Approval would mean the therapy could be used twice a week to prevent bleeding episodes and to counter bleeding when episodes do occur. The U.S. Food and Drug Administration…

Long-term prophylaxis, or preventive treatment with Adynovate continues to safely reduce bleeding events over multiple years in children and adults with severe hemophilia A without causing neutralizing antibodies, a Phase 3b trial found. The study, “Long‐term safety and efficacy results from the phase…

The U.S. Food and Drug Administration (FDA) approved Sevenfact (coagulation factor VIIa [recombinant]-jncw) for the treatment and control of spontaneous bleeding episodes in people, ages 12 and older, with hemophilia A or B with inhibitors. Hemophilia, a genetic disorder, affects the body’s ability to make blood…

Catalyst Biosciences recently announced updates on two of its lead compounds — Factor VIIa (FVIIa) marzeptacog alfa (activated) (MarzAA) and Factor IX (FIX) dalcinonacog alfa (DalcA) — for treatment of hemophilia A or B with inhibitors, and for hemophilia B. DalcA is a potent recombinant (artificial) Factor IX prophylactic therapy designed to prevent acute bleeding…