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…

Hemlibra (emicizumab) is safe and effective to treat children younger than 12 who have hemophilia type A and are negative for antibodies against synthetic factor VIII, results from a Japanese clinical study show. The trial findings were reported in the study, “A multicentre, open‐label study of emicizumab given…

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…

People with hemophilia may benefit from mild exercise to minimize their risk of bleeding according to a study published in the scientific journal Haemophilia.

The once-weekly injection therapy marstacimab is being considered for approval in both the U.S. and Europe for people with hemophilia A and hemophilia B who don’t have inhibitors. The U.S. Food and Drug Administration (FDA) is expected to decide on it in late 2024, while a decision from the European…

The Medicines and Healthcare Products Regulatory Agency (MHRA) for the U.K. has approved the resumption of patient enrollment in BioMarin Pharmaceuticals’ Phase 1/2 clinical trial evaluating the investigational gene therapy BMN 270 as a treatment for severe hemophilia A. BioMarin suspended patient dosing in the trial in June because of increasing levels…

Approximately 30,000 males have hemophilia in the U.S., according to estimates from a recent study, which also found that patients with hemophilia are generally young and that the disease is more common in Northeastern and Midwestern states. The study, “Occurrence rates of haemophilia among males in…

The U.S. Food and Drug Administration (FDA) has approved Wilate as a prophylactic (preventive) and on-demand treatment for bleeding episodes in adults and adolescents with hemophilia A. Wilate, developed and marketed by Octapharma, is an injectable medication that contains human plasma-derived von Willebrand Factor (VWF)…

High adherence to prophylactic treatment not only reduces the number of spontaneous bleeding episodes in patients with hemophilia A, but it also improves their physical activity, according to a study. The study, “Physical activity improved by adherence to prophylaxis in an Italian population of children, adolescents and…

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…