The protein missing or defective in people with hemophilia A, called blood coagulation factor VIII (FVIII), is able on its own to stimulate immune responses in a mouse model, a study suggests. This immune response to FVIII is dependent on a set of distinct immune cells within the spleen,…
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The National Hemophilia Foundation (NHF) has changed its name to the National Bleeding Disorders Foundation (NBDF) to reflect its support for people with inheritable blood and bleeding disorders other than hemophilia. The new name includes a new visual identity and logo. The foundation also unveiled a new tagline…
Treatment with Ixinity (trenonacog alfa) can safely control bleeds in children younger than 12 with severe or moderately severe hemophilia B. That’s according to data from a Phase 3/4 clinical trial that Medexus Pharmaceuticals, the company that markets Ixinity, presented at the National Hemophilia Foundation’s recent Bleeding…
More bleeding episodes may mean a lower quality of life and less productivity at work for men with hemophilia A living in Europe, according to an analysis of data from the CHESS II retrospective study. The analysis, “The impact of bleeding event frequency on health-related quality of life…
When given as a prophylactic, or preventive therapy, or on an on-demand basis, FEIBA (factor eight inhibitor bypassing activity) was deemed safe and effective at lessening the number of bleeds in people with severe hemophilia and high levels of inhibitors. That’s according to four-year data from a real-world…
Novo Nordisk’s Alhemo (concizumab injection) has been approved in Canada for hemophilia A patients, 12 years or older, who test positive for factor VIII inhibitors. The under-the-skin injectable therapy was recently approved by Health Canada for hemophilia B patients in the same age group with factor…
A small percentage of young people with hemophilia A who are on preventive treatment with Hemlibra (emicizumab) experience severe muscle bleeds that require prolonged factor replacement therapy. These are the findings of a real-world, multicenter study in the U.S. which also show that most of these severe muscle…
The U.K.’s National Institute for Health and Care Excellence (NICE) has issued draft guidance recommending that England’s National Health Service (NHS) should not cover the gene therapy Hemgenix (etranacogene dezaparvovec) for adults with hemophilia B. The recommendation is based on uncertainty surrounding the long-term efficacy of the…
Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy approved for adults with severe hemophilia A, is expected to be widely available in the U.S. this month, according to its developer, BioMarin. BioMarin also is working with authorities in Germany, Italy, and France to secure market access to…
Roche is initiating a Phase 3 clinical trial to test the experimental gene therapy SPK-8011 in people with hemophilia A. The company announced the move in its 2023 half-year results. At the same time, however, Roche is discontinuing the development of another investigational hemophilia A gene therapy…
