Marta Figueiredo, PhD, managing science editor —

Hemlibra (emicizumab) continues to safely and effectively prevent bleedings in children and adults with hemophilia A, regardless of developing neutralizing inhibitors (antibodies) against synthetic factor VIII (FVIII), according to nearly three years of data from four Phase 3 clinical trials. These and preliminary real-world safety findings from a European…

Prophylactic (preventive) treatment with Adynovate safely and effectively lowers the number of bleedings, including those in target joints, in children and adults with severe hemophilia A, according to data from two Phase 3 clinical trials. Notably, personalized Adynovate treatment targeting higher minimum blood levels of factor VIII (FVIII) —…

Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively lowers bleeding rates in children and adolescents with severe hemophilia A, according to a real-life study from Israel. The therapy’s effectiveness was independent of the presence of inhibitors (antibodies) against synthetic formulations of factor VIII (FVIII) — the missing…

Valoctocogene roxaparvovec, BioMarin Pharmaceutical‘s investigational gene therapy, results in a more than 90% drop in annual bleeding rates and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, updated data from a Phase 1/2 clinical trial show. It also precluded the need…

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…

Novo Nordisk has paused three clinical trials evaluating concizumab, its experimental treatment candidate for hemophilia A and B, due to safety concerns. The three Explorer studies — the Phase 2 Explorer 5 (NCT03196297), and the global Phase 3 Explorer 7 (NCT04083781) and Explorer 8 (NCT04082429)…

Researchers have developed a faster, less expensive, reliable method for non-invasive prenatal diagnosis (NIPD) of hemophilia A. Future, larger studies are required to confirm the utility of the method, which could be used for the NIPD of hemophilia A and type B, as well as other X-linked…

Specific genetic changes may account for a larger proportion than previously thought of children with severe hemophilia B who develop inhibitors against coagulation factor IX replacement therapy, according to an international study. The study, “Inhibitor Incidence In An Unselected Cohort Of Previously Untreated Patients With…

ASC Therapeutics is partnering with Vigene Biosciences to support the manufacturing process of its current and future gene therapy clinical platforms — including its ongoing hemophilia A program. “We are proud to welcome Vigene, a global leader in gene therapy process development and GMP [Good…

BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec safely and significantly reduced bleeding events — including in target joints — and the use of prophylactic clotting factor VIII in adults with severe hemophilia A, data from a Phase 1/2 study show. These results, along with an interim analysis…