News

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The U.K.’s National Institute for Health and Care Excellence (NICE) has issued draft guidance recommending that England’s National Health Service (NHS) should not cover the gene therapy Hemgenix (etranacogene dezaparvovec) for adults with hemophilia B.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…

Nuwiq (simoctocog alfa) effectively prevented and controlled bleeds in previously untreated people with severe hemophilia A, according to data from a Phase 3 clinical trial. Full trial results were reported in the study, “Simoctocog alfa (Nuwiq) in previously untreated patients with severe haemophilia A — Final efficacy…

The approval by the U.S. Food and Drug Administration (FDA) of BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox) late last month means that eligible adults with severe hemophilia A will soon have access to a gene therapy for the first time. “Adults with severe hemophilia A live with several limitations,…

Preventive treatment with Hemlibra (emicizumab) was not considered cost-effective at its current price compared with standard replacement therapies in adults with mild or moderate hemophilia A without inhibitors, according to a modeling study. The cost of Hemlibra would need to drop by at least 35% to become cost-effective…