March is Bleeding Disorders Awareness Month (BDAM), and patients, caregivers, advocates, and supporters are working together to call attention to and provide resources for coping with the bleeding disorders estimated to affect more than 3 million people in the U.S. Bleeding disorders such as hemophilia, von Willebrand disease, and…
News
Pfizer said it will stop development and commercialization of hemophilia B treatment Beqvez (fidanacogene elaparvovec-dzkt). “Pfizer has made the decision to cease further development and commercialization activities with respect to Beqvez for several reasons, including the limited interest patients and their doctors have demonstrated in hemophilia gene therapies…
The Loma Linda University Health (LLUH) Hemophilia Treatment Center has become the first clinic in California offering Hympavzi (marstacimab) for people with hemophilia B. “For the first time, patients with hemophilia B in Southern California have access to a therapy that is not just effective, but also drastically…
Ixinity (trenonacog alfa) can effectively prevent and control bleeds in young children with severe or moderately severe hemophilia B, according to data from a Phase 3/4 clinical trial. “This study showed that [Ixinity] was safe and effective as prophylaxis and for controlling bleeds in previously treated persons with…
The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved efanesoctocog alfa — to be sold under the brand name Altuvoct — as a treatment for moderate or severe hemophilia A in adults and children, ages 2 and older. Approved in the European Union in June…
Four years after treatment with the gene therapy Hemgenix (etranacogene dezaparvovec), most people with hemophilia B aren’t taking preventive therapies and annual bleeding rates remain substantially reduced, new data from the Phase 3 HOPE-B trial shows. CSL Behring, which markets Hemgenix, presented the results at the 18th…
Antibody therapy Mim8 (denecimig) was well tolerated and provided effective bleed control in children with hemophilia A regardless of inhibitor status. That’s according to interim data from the now-complete Phase 3 FRONTIER3 study (NCT05306418), which enrolled children ages 1-11. These findings were consistent with recent data from…
People with severe hemophilia A who have two or more self-described problem joints — a person-centric characterization of joints based on underlying joint damage, pain, and mobility impairment, regardless of bleeding — have worse clinical outcomes, including more chronic pain, a new study from researchers in Europe has found.
Hemlibra (emicizumab), a preventive treatment for hemophilia A, reduced pain and improved quality of life for adults and adolescents with the disease, including those with recurrent joint bleeds, a study found. Significant improvements in pain-related quality of life were seen 13 weeks, or about three months, after starting…
An experimental gene therapy using modified blood stem cells helped five adults with severe hemophilia A attain therapeutic levels of clotting factor VIII (FVIII) to prevent and control bleeding, according to a small first-in-human study. Over the course of the Phase 1 clinical study (NCT05265767), none of the…
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