Data from ongoing Phase 3 clinical trials show that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis — or preventive treatment — greatly outperforms current standard-of-care therapies, leading to significant reductions in the number of bleeds in hemophilia A patients regardless of inhibitor status. Promising results from these pivotal studies may lead…
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Intensive treatment with factor VIII, the clotting protein missing or defective in patients with hemophilia A, is associated with a greater risk of inhibitor development among patients with non-severe disease, a study has found. The findings highlight the importance of closely monitoring these patients in order to prevent or…
Single or combination therapy with bypassing agents used for hemophilia A patients with inhibitors can be suitable treatment options for children with hemophilia B with factor IX inhibitors as well, a case series suggests. The report, “Alternative treatment options…
Once-daily preventive treatment with Novo Nordisk’s experimental therapy concizumab significantly reduced bleeding rates in people with both hemophilia A and B with inhibitors, according to new data from the Phase 3 explorer7 trial. In fact, bleeding rates in patients receiving concizumab in trial decreased by more than 85%. Based…
The U.S. Food and Drug administration (FDA) has approved Genentech’s Hemlibra (emicizumab-kxwh) for routine prophylactic (preventive) treatment of patients with hemophilia A without factor VIII inhibitors. Hemlibra is now the sole available prophylactic treatment for patients with hemophilia A with and without factor VIII inhibitors that can…
A 48-year-old man with severe hemophilia A developed a rare intramural hematoma with blood leaking into one of the layers of the intestine wall that was successfully treated with an early infusion of the missing blood clotting factor, a recent case report described. Prophylactic treatment prevented its recurrence. A…
The U.S. Food and Drug Administration (FDA) has cleared for use CRYOcheck Chromogenic Factor VIII, Precision BioLogic‘s lab test to monitor factor VIII activity in the blood of hemophilia A patients ages 2 and older and help in disease management, the company said in a…
Note: This story was updated Feb. 24, 2023, to correct the secondary headline since there are several other recombinant factor VIII therapies available. Altuviiio is a first-in-class therapy, but not the first. Efanesoctocog alfa, Sobi and Sanofi’s first-in-class, long-lasting recombinant or man-made factor…
Switching from on-demand to preventive treatment — or prophylaxis — significantly reduces bleeding in adults with hemophilia A, including the elderly, a real-life study shows. The study, “Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A,”…
The U.S. Food and Drug Administration (FDA) has given orphan drug and rare pediatric disease designations to Sernova’s experimental and cell-based hemophilia A treatment program using the Cell Pouch System, the company’s novel medical device. Orphan drug status is awarded to therapies aiming to treat…