Health Canada has approved Esperoct, an engineered formulation of clotting factor VIII, for the treatment of hemophilia A in people of all ages. The approval covers prophylactic, or preventive treatment, on-demand use for controlling bleeding episodes, and use in reducing bleeding during surgery. Esperoct received similar approval…

The cost of hemophilia treatment doubled for patients who switched from standard half-life (SHL) to extended half-life (EHL) products, according to a recent study by Prime Therapeutics. The findings of the study were presented at the Academy of Managed Care Pharmacy’s (AMCP) Managed Care & Specialty…

Starting Hemlibra (emicizumab) early is safe and helps to prevent bleeding in babies with severe hemophilia A who are untreated or minimally treated, though doctors should still monitor for self-reactive antibodies that can make other treatments less effective, a study suggested. The study, “Emicizumab in Previously Untreated…

The U.S. Food and Drug Administration (FDA) has accepted Bioverativ’s investigational new drug application (IND) for BIVV001, an investigational Factor VIII therapy designed to extend protection from bleeds in hemophilia A patients. The FDA will now review the IND application for safety. The Waltham, Massachusetts-based company plans to initiate a Phase…

Dosing has begun in a Phase 2b clinical trial evaluating the safety and efficacy of SerpinPC, Centessa Pharmaceuticals’ investigational therapy for hemophilia. The open-label study, called PRESent-2 (NCT05789524), is part of the company’s registrational program for hemophilia B, which includes multiple clinical trials aiming to…

Nearly all adults with acquired hemophilia A (AHA) safely achieved complete remission with either standard or low doses of rituximab when combined with corticosteroids and cyclophosphamide, a small study shows. Key features of disease remission, including an increase in clotting factor levels and a reduction in inhibitor levels,…

In boys and men with severe hemophilia A, starting prophylaxis — preventive treatment given regularly to reduce bleeding — early and maintaining regular physical activity were each linked to a lower risk of hemophilic arthropathy, a progressive form of joint damage caused by repeated bleeding, according to a real-world…

BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec continues to safely and effectively prevent bleeding episodes and the need for prophylactic clotting factor VIII in adults with severe hemophilia A, four-year data from a Phase 1/2 clinical trial show. The results were submitted for presentation at the World Federation…

Roctavian, an investigational gene therapy, significantly reduced bleeding rates and the need for other treatments in men with severe hemophilia A over at least one year, top-line data from a Phase 3 clinical trial show. The therapy’s durability in sustaining factor VIII production at levels necessary to…