Catalyst Biosciences announced positive interim results from a Phase 2/3 clinical trial (NCT03407651) of the Factor VIIa variant marzeptacog alfa (activated) (MarzAA) in the preventative treatment of patients with hemophilia A or B with inhibitors. The study, aiming to include up to 12 patients, is recruiting in Russia…

Kovaltry (octocog alfa) is effective and safe as a preventive treatment for children with hemophilia A, a European real-world study reports. The study, “Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a…

More than 21 years ago, when my first child was born and diagnosed with severe hemophilia A, he was very fortunate to have first-generation clotting factors available. These were concentrates stabilized with albumin that were very safe. When the factor was reconstituted with sterile water, a seemingly…

A major complication in scientists’ efforts to treat hemophilia A by replacing a missing or defective clotting factor is an immune response to the treatment. Researchers have discovered a connection between the body’s production of pro-inflammatory molecules known as cytokines and its immune response to clotting factor VIII therapy. They used…

People living with bleeding disorders such as hemophilia and von Willebrand disease report consistently high levels of satisfaction with the care they receive at U.S. hemophilia treatment centers (HTCs), according to the results of the U.S. HTC Network’s first national survey. Most respondents said they were satisfied with…

The European Commission has approved Hemlibra (emicizumab) for routine preventive treatment of bleeding episodes in people who have hemophilia A with factor VIII inhibitors. The antibody therapy is designed to restore the factor VIII, which is deficient in hemophilia A. Hemlibra is being co-developed by Japan’s Chugai Pharmaceuticals, Switzerland’s…

The advancements in modern medicine have meant that people with hemophilia are living longer than ever, however, this brings its own set of problems for the medical profession. People living with the disease are just as likely to have other health issues when they get older as those without hemophilia,…

The full range of hemophilia treatments should be made available to patients without consideration of cost or other barriers, leaders of patient associations and others argued at a public meeting hosted by the Institute for Clinical and Economic Review (ICER) to consider its recent analysis of hemophilia A therapies. The…

Under-the-skin, preventive treatment with Dalcinonacog alfa (DalcA) led to sustained and protective levels of factor IX (FIX) in people with severe hemophilia B, final data from a Phase 2b trial show. The findings, Phase 2b Trial to Evaluate the Safety and Factor IX Levels of a Daily Subcutaneous Prophylaxis…