Medexus Pharmaceuticals has completed patient enrollment in its clinical trial investigating Ixinity as a preventive treatment for children younger than 12 with hemophilia B.  The open-label Phase 4 trial (NCT03855280) achieved its target enrollment goal of 22 participants and is being conducted in 15 centers in Brazil,…

SCT800, a replacement therapy containing a man-made form of clotting factor VIII (FVIII), appears to be safe and effective at treating and preventing bleeds in adolescents and adults with severe hemophilia A, a study has found. The study, “Pharmacokinetic, efficacy and safety evaluation of B-domain-deleted…

As a parent, it is my responsibility to help my sons become productive, compassionate, caring individuals. My oldest son, Julian, is 25 and working to complete his college degree. He is on the cusp of transitioning to a new phase in life. In less than a year, he will…

Two years of preventive treatment with Genentech’s Hemlibra (emicizumab) safely and effectively lowered the number of bleeds in people with hemophilia A and clotting factor VIII (FVIII) inhibitors, according to final data from the Phase 3b STASEY trial. Development of antibodies against the therapy (anti-drug antibodies) was…

The European Medicines Agency (EMA) has validated BioMarin Pharmaceutical’s new application seeking approval of its investigational one-time gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. The application was resubmitted to EMA following a request for longer follow-up data from the Phase 3 GENEr8-1 clinical trial (NCT03370913),…

Voncento — a highly concentrated mixture of Von Willebrand factor (VWF) and clotting factor VIII (FVIII) — was seen to safely and effectively treat or prevent bleeds in children, age 12 and younger, with severe hemophilia A, according to data from a Phase 3 trial. Findings were reported in the…

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…

Spending on treatments for hemophilia by Medicaid, a U.S. government health insurance program for select groups, more than tripled from 2005 to 2019, an analysis reported. Its researchers expect this finding will help state agencies with decisions regarding treatment coverage based on their Medicaid budget, given that the program…

A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of understanding how treatment decisions affect patients. The investigator-led MOTIVATE study (NCT04023019; EudraCT No. 2019-003427-38) is recruiting…

Severe hemophilia B patients receiving prophylactic, or preventive treatment with factor IX face a significant financial and personal burden, according to data from two U.S. population-based medical databases. These findings were reported in a study, “Clinical, humanistic, and economic burden of severe hemophilia B in the United States:…