Real-world use of the extended half-life factor replacement therapy Elocta (efmoroctocog alfa) was associated with better bleed control than standard half-life products among people with hemophilia A, according to findings from an observational study in Europe. Elocta, sold as Eloctate in the U.S., was also associated with fewer…

The U.S. Food and Drug Administration has approved Novo Nordisk’s Rebinyn (nonacog beta pegol) as a routine preventive treatment for bleeding episodes in adults and children with hemophilia B. Rebinyn, sold under the brand name Refixia in the EU, is a replacement therapy that had originally…

The gene therapy BBM-H901 increased factor IX activity in 10 men with moderate to severe hemophilia B, while reducing bleeds and the use of FIX replacement therapy over one year, according to results of a pilot Phase 1 trial. “The study was the first clinical trial conducted by Belief BioMed…

Hemophilia A patients who receive replacement therapies of factor VIII often develop inhibitors against factor concentrates which hinders the effectiveness of the therapy, increases disability, and reduces quality of life. Although immune tolerance induction (ITI) can reduce inhibitors against factor VIII concentrates, the study “Long-term course of…

A hemophilia diagnosis takes a toll on the social and mental health of men, who often feel misunderstood and excluded from activities such as sports, a Canadian interview-based study reports. While blood-clotting replacement therapies have improved quality of life, they can also be costly. Study participants highlighted the need…

Elevated levels of the immune signaling protein BAFF were found in the bloodstream of boys who developed neutralizing antibodies against FVIII replacement therapy, a standard treatment in hemophilia A, a study reported for the first time. Changes in the gene that encodes the BAFF protein, involved in the growth…

Researchers have developed new versions of clotting factor IX (FIX) that may be used to create personalized treatments for people with hemophilia B — specifically, new replacement therapies designed to help more effectively control bleeding. According to the team, the development of these new FIX versions may serve to create both short-…

Efanesoctocog alfa, a long-lasting factor VIII (FVIII) replacement therapy, has now been approved in the European Union for hemophilia A. It will be marketed under the brand name Altuvoct. Altuvoct can be used as an on-demand treatment for bleeding episodes, a once-weekly prophylactic (preventive) therapy to reduce the…