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…

The U.S. Food and Drug Administration granted breakthrough therapy designation to Genentech’s Hemlibra (emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Hemlibra is designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with…

Switching to Hemlibra (emicizumab) was safe and effective in children with hemophilia A, including in those who had been minimally treated before or who had not been treated at all, a real-world study reported. Researchers noted that studies involving a larger number of patients are still warranted to…

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…

Nuwiq, a fourth-generation genetically engineered version of blood-clotting factor VIII, is a safe and effective way of controlling bleeding in people with severe hemophilia A during and after surgery, a study reports. The research, “Efficacy and safety of Nuwiq® (human-cl rhFVIII) in patients with severe haemophilia A undergoing…

Hemlibra (emicizumab-kxwh) safely supports joint health in children with hemophilia A without inhibitors through three years of treatment, according to interim data from a post-marketing study in Japan. The ongoing study, AOZORA (jRCT1080224629), is evaluating the long-term safety of Hemlibra and its impact on joint health in children ages…

Severe hemophilia A patients using Esperoct (turoctocog alfa pegol) as a preventive treatment do well, with fewer bleeds or breakthrough bleeds, lower costs, and better life quality compared with other extended half-life (EHL) therapies, a modeling analysis suggested. The…

Long-term prophylactic (preventive) treatment with a fixed dose of Esperoct (turoctocog alfa pegol) — approved in the U.S. and the EU in 2019 — was effective in preventing and stopping bleeding in people with severe hemophilia A, according to data from two Phase 3 clinical trials. Findings from…

Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia A. The therapy is designed to allow for dosing at intervals as long as once every seven days. The…

At standard U.S. doses, Hemlibra (emicizumab) provides comparable or even superior clinical benefits than prophylactic factor VIII (FVIII) replacement therapy among adults with severe hemophilia A and no inhibitors, according to a report from the Institute for Clinical and Economic Review (ICER). Hemlibra is also cost-saving, due to…