The European Medicines Agency (EMA) has granted orphan medicinal product status to SB-525, a clinical stage gene therapy candidate under development for the treatment of hemophilia A, according to Sangamo Therapeutics and Pfizer. The announcement coincides with the opening of patient enrollment for a Phase 1/2…

Treatment with Esperoct led to stabilization and/or improvement of health-related quality of life (HRQoL) in patients with severe hemophilia A from all age groups, a study suggests. The study, “Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant…

A programmable mini-pump able to provide regular doses after surgery of FVIIa to hemophilia A patients with inhibitors — those resistant to standard FVIII replacement therapy — successfully maintained expected levels of blood clotting factors, a small study reports. Attending surgical nurses also voiced satisfaction with the pump’s use, reporting a…

The National Institute for Health and Care Excellence (NICE) has issued a final guidance recommending the use of Altuvoct (efanesoctocog alfa) in England and Wales as an option for people with severe hemophilia A, ages 2 and older. The National Health Service, the U.K.’s public healthcare system, is…

Swedish Orphan Biovitrum AB recently announced that Eloctate (efmoroctocog alfa; brand name Elocta in Europe), a treatment for hemophilia A, has been approved for reimbursement in the United Kingdom, Italy, and France. These countries join others in the European Union — Germany, Sweden, Denmark, Norway, Switzerland, the Netherlands,…

The Royal College of Surgeons in Ireland (RCSI) and Bayer will collaborate to develop improved treatments for patients with severe hemophilia. The research collaboration will focus on personalized treatments adapted to the severity of each patient’s condition to achieve safer and more effective blood clotting in hemophilia…

Researchers have found  that N8-GP (turoctocog alfa pegol) treatment improved the quality of life and satisfaction of children with severe hemophilia A. The study titled “Health-Related Quality of Life and Satisfaction of Children with Severe Hemophilia a Treated with Turoctocog Alfa Pegol (N8-GP),” appeared in the journal Blood.

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…

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…

The U.S. Food and Drug Administration (FDA) has accepted Bayer’s Biologics License Application (BLA) filing for BAY94-9027 to treat hemophilia A in adolescents starting at age 12, and in adults. A BLA is a marketing application that covers biological products, as opposed to chemically synthesized ones, and its…