Children and adults with hemophilia A in the United States may soon be treated with N8-GP (turoctocog alfa pegol), an engineered formulation of clotting factor VIII developed by Novo Nordisk. The therapy will be available under the brand name Esperoct. The U.S. Food and Drug Administration (FDA) approved N8-GP, or…

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…

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…

Japan’s Ministry of Health, Labor and Welfare has extended Hemlibra (emicizumab)’s approval to include routine prophylaxis to prevent or lower the frequency of bleeds in people with acquired hemophilia A. Its decision comes seven months after Hemlibra’s maker, Chugai Pharmaceutical, applied for extended use. Hemlibra was previously approved in…

As a long-term preventive treatment, fitusiran leads to sustained reductions in bleed frequency in people with moderate-to-severe hemophilia A or B regardless of inhibitor status, early results from an extension study show. The findings were presented at the recent World Federation of Hemophilia Virtual Summit and…

A combination of Cyklokapron and Feiba — usually avoided due to a perceived risk of blood clots — appears to be safe and effective for treating patients with acquired hemophilia A (AHA), though these early study results need to be confirmed in clinical trials, researchers in Italy say. The study, “…

Last week, I was on Facebook and a post I had been dreaming about appeared on my timeline. The U.S. Food and Drug Administration has approved a new therapy, Hemlibra (emicizumab-kxwh), for patients with hemophilia A and inhibitors. It’s the first new medicine for inhibitors in almost…

Patient treatment for hemophilia B has come a long way since the introduction of factor IX (FIX) concentrates. In a recent commentary published in the journal Blood and titled “A new era for hemophilia B treatment,” researchers from Oregon Health and Science University and Bloodworks Northwest highlighted recent advances in…