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…
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Several treatments for hemophilia A have shown promise in clinical trials. SCT800 and Esperoct have been found safe and effective in preventing and treating bleeds. Emicizumab also shows promise as a preventive treatment for all ages. Additionally, Novo Nordisk acquired a hemophilia A program focusing on gene-editing technology.
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Results from two Phase 3 trials support the use of emicizumab for the preventive treatment of hemophilia A in children, adolescents, and adults, Genentech announced. These results will be presented at the 26th International Society on Thrombosis and Haemostasis (ISTH) Meeting July 8-13 in Berlin, Germany. Emicizumab is an…
Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…
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…
Interim results of Phase 3 clinical studies investigating two major challenges in hemophilia — the development of inhibitors (antibodies to the drug used to treat bleeding episodes) and the need for repeated venous injection of blood clotting protein Factor VIII (FVIII) — were recently released. The results were presented by…
Hemophilia News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to hemophilia throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read stories of 2019. No.
Continuous into-the-vein (intravenous) infusions of Advate immediately before and after major orthopedic surgery is as safe and effective as standard, short, bolus infusions at preventing bleeds in men with moderate-to-severe hemophilia A. That finding from a Phase 3/4 clinical trial — the first controlled trial to compare the two types…
After receiving guidance from both the U.S. Food and Drug Administration and the European Medicines Agency, Catalyst Biosciences is preparing to launch a pivotal Phase 3 clinical trial assessing marzeptacog alfa activated (MarzAA) for the treatment of bleeding episodes in people hemophilia A or B with…
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…
Clinical trials of Hemlibra (emicizumab-kxwh) continue to show significant reductions in bleeding episodes in patients with hemophilia A with inhibitors, Genentech recently announced. Data from Hemlibra’s clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. The results show that in hemophilia A patients with inhibitors…