A study presented at American Society of Hematology’s (ASH) annual meeting and exposition shows that Adynovate is effective for the prevention and treatment of severe pediatric Hemophilia A (HA). Eric Mullins, MD and his colleagues from the Cancer and Blood Diseases Institute at Cincinnati Children’s Hospital Medical Center presented their work…
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The recommended treatment regimen for patients with severe hemophilia to prevent bleeding episodes is to replace the deficient clotting factor. However, currently available replacement clotting factors are limited by their relatively short half-lives and require intravenous injections up to three times a week to maintain protective levels. This can have…
When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and effectively lowered the number of bleeding episodes in patients with severe hemophilia A, according to data from a Phase 1 clinical trial and its long-term extension study. Importantly, reductions in the number of bleeds…
The U.S. Food and Drug Administration (FDA) has expanded its approval of Alhemo (concizumab-mtci), a daily therapy injected under the skin for preventing or reducing the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older. Sold by Novo Nordisk, Alhemo…
The blood-clotting protein factor VIII (FVIII) that’s missing or faulty in people with hemophilia A is flexible and adopts multiple shapes, some of which favor the binding of neutralizing antibodies that can reduce the effectiveness of FVIII replacement therapies, researchers have learned. “This helps explain why some people…
Total joint replacement (TJR) is more common in severe hemophilia patients requiring more frequent treatment, according to a population-based study in Taiwan. The findings also revealed that the incidence of TJR does not differ between hemophilia A and B. The study, “Comparison of Total Joint…
With a new label expansion cleared by the U.S. Food and Drug Administration (FDA), Hympavzi (marstacimab-hncq) is now approved for preventing or reducing the frequency of bleeds in all people with hemophilia A or hemophilia B, ages 6 and older, regardless of inhibitor status. The injectable therapy…
Roctavian, an investigational gene therapy, significantly reduced bleeding rates and the need for other treatments in men with severe hemophilia A over at least one year, top-line data from a Phase 3 clinical trial show. The therapy’s durability in sustaining factor VIII production at levels necessary to…
A team of U.S. scientists discovered a naturally occurring F8 gene mutation that leads to increased activity of the factor VIII (FVIII) coagulation factor and enhanced blood clotting, offering new insights into the treatment of hemophilia A. The mutation was identified in a young man susceptible to excessive blood…
Light Chain Bioscience, a unit of Swiss biotech Novimmune, has completed the discovery phase of its collaborative program with Takeda for developing bispecific antibodies — protective proteins produced by the immune system — that mimic the activity of blood clotting factor VIII as a treatment for…