Hemlibra (emicizumab) was able to prevent excessive bleeding in patients with severe hemophilia A without inhibitors, according to results from a Phase 3 trial. Trial findings were published in the study, “Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors,” in The New England Journal of…
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The U.S. Food and Drug Administration has approved Bayer’s Jivi, previously known as BAY94-9027, as a preventive therapy for hemophilia A in patients ages 12 and older, the company announced. The recommended regimen is for Jivi to be administered intravenously (into the blood) two times a week (30-40 IU/kg),…
uniQure has treated the first patient in a Phase 2b dose-confirmation study of its investigational gene therapy, AMT-061, for people with severe and moderately severe hemophilia B. The trial is still recruiting participants. AMT-061 uses a viral vector, AAV5, to deliver the gene for a mutated clotting factor…
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…
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…
Health Canada has approved Hemlibra (emicizumab) as a treatment to prevent or reduce bleeding in Hemophilia A patients in Canada who have factor VIII inhibitors. Hemlibra is an antibody therapy designed to combine factors IX and X of the blood clotting series and promote clotting. Restoring the…
Several genetic and environmental factors can play a role in the development of inhibitors against treatment with factor VIII in hemophilia A patients, according to a review study. The review, “Risk factors for inhibitor development in severe hemophilia A,” was published in the journal Thrombosis Research. Severe…
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Employment Status, Pain, Low Physical Activity Linked to Anxiety, Depression in Hemophilia Patients
A study that examined the role of emotional distress in the lives of hemophilia patients found that a patient’s employment status, pain interference levels, perception of the detrimental consequences of hemophilia, and physical activity levels were all independently associated with symptoms of anxiety and depression. The study, “Emotional distress…
The Hemophilia Federation of America (HFA) is seeking a variety of historical artifacts and records to add to its archival project in honor of its upcoming 25th year of helping the bleeding disorders community. The nonprofit is collecting vintage medical equipment, supplies, rally posters, newsletters, documents, personal journals, diaries, and…
A gene therapy that can treat patients with severe hemophilia A is likely to be more cost-effective over the long term than reliance on prophylactic (preventive) therapy using factor VII (FVIII), a model-based analysis of the two treatment approaches reports. The study, “Gene therapy in hemophilia A: a cost-effectiveness…
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