Lindsey Shapiro, PhD, science writer —

A small percentage of young people with hemophilia A who are on preventive treatment with Hemlibra (emicizumab) experience severe muscle bleeds that require prolonged factor replacement therapy. These are the findings of a real-world, multicenter study in the U.S. which also show that most of these severe muscle…

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,…

The Hemophilia Federation of America (HFA) is now offering a “clinical trial finder” to help people with hemophilia and other bleeding disorders more easily connect with the studies they are eligible for. The platform enables visitors to search across all clinical trials, regardless of sponsor, for key…

Electrically stimulating the vagus nerve — the main set of nerve fibers that control involuntary bodily functions — significantly reduced blood loss and sped up clot formation after injury in a mouse model of hemophilia A, according to a recent study. Controlling bleeding by stimulating the vagus nerve was…

Weekly treatment with the experimental therapy marstacimab (PF-06741086) was found to significantly reduce bleed rates for adult and adolescent hemophilia A and B patients without inhibitors relative to standard therapies. In so doing, it met the main goal of the pivotal Phase 3 BASIS clinical trial, designed to…

Switching to Rebinyn (nonacog beta pegol) from other standard or extended half-life replacement therapies led to “robust” reductions in annual treatment costs among hemophilia B patients in Canada, a study based on real-world data reported. Its findings add to an earlier and real-world analysis of patients who…

About one-third of moderate-to-severely affected people with hemophilia perceived declines in their daily functional abilities over a period of three to five years, a Dutch study found. While no strong risk factors for such declines were identified, patients more likely to be affected were those who were obese, had…

SerpinPC, an investigational treatment that Centessa Pharmaceuticals is developing for hemophilia, will advance to interventional Phase 2 clinical testing later this year. The studies will support Centessa’s registrational program for hemophilia B. Such a program involves clinical trials that ultimately will lead to the company seeking regulatory approval…

People with moderate-to-severe hemophilia and ankle joint disease reported significant impairments in life quality and foot and ankle function, according to a recent U.K. study. Chronic pain emerged as a significant predictor of these patient-reported outcomes, as did the presence of inhibitors — neutralizing antibodies that can lower the…

Note: This story was updated Feb. 24, 2023, to correct the secondary headline since there are several other recombinant factor VIII therapies available.  Altuviiio is a first-in-class therapy, but not the first. Efanesoctocog alfa, Sobi and Sanofi’s first-in-class, long-lasting recombinant or man-made factor…