Delaying the start of immunosuppressive treatment in adults diagnosed with acquired hemophilia A who are receiving Hemlibra (emicizumab-kxwh) to prevent bleeding may reduce the risk of infections and extend survival, a clinical study found. Patients in the Phase 2 study (NCT04188639 received Hemlibra for 12 weeks while immunosuppression…

Combining gene therapy with minimally invasive, robot-assisted hip replacement surgery safely and effectively treated severe joint damage for a 27-year-old man in China with hemophilia A, according to a case report. The approach significantly reduced the need for replacement therapy…

BioMarin Pharmaceuticals is planning to submit marketing authorization applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) seeking approval of valoctocogene roxaparvovec, its investigational gene therapy for the treatment of adults with hemophilia A, before the end of the…

Bleeding episodes in people with severe hemophilia A receiving prophylactic (preventive) treatment have the potential to impair patients’ health-related quality of life, according to data from a survey conducted in Europe. Even a single bleed was linked to marked medication-related and non-related health costs. In fact, in terms of…

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended Hemlibra’s (emicizumab) approval be expanded to include people with moderate hemophilia A without inhibitors. “We’re very pleased that the CHMP’s recommendation brings us closer to potentially transforming the day-to-day lives of people in the…

A multi-institute research collaboration has received a $12 million grant from the National Heart Lung and Blood Institute to understand what’s behind limitations in gene therapies currently being tested in people with hemophilia A. The goal is to use this information to develop safer and long-lasting gene therapies…

Artificial intelligence (AI) can comb through ultrasound scans to detect joint bleeding and inflammation occurring as a result of hemophilia, a study has found, suggesting it may help doctors make quicker and more accurate diagnoses. “AI models have the potential to aid diagnosis and enable earlier therapeutic interventions, helping…

Simultaneous use of NovoSeven (recombinant factor VIIa, rFVIIa) and Hemlibra (emicizumab) in patients with hemophilia A is not associated with higher risk of thrombosis, new analysis of the Phase 3 HAVEN program shows. The trials’ findings were reported in the study, “Safety analysis of…

The approval by the U.S. Food and Drug Administration (FDA) of BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox) late last month means that eligible adults with severe hemophilia A will soon have access to a gene therapy for the first time. “Adults with severe hemophilia A live with several limitations,…

More bleeding episodes may mean a lower quality of life and less productivity at work for men with hemophilia A living in Europe, according to an analysis of data from the CHESS II retrospective study. The analysis, “The impact of bleeding event frequency on health-related quality of life…