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…

Combining immune tolerance induction, used to prevent the development of anti-factor VIII inhibitors, with Hemlibra (emicizumab) is a feasible and safe way of treating children with severe hemophilia A, a study shows. Published in the journal Haemophilia, the report covers clinical outcomes in the first seven patients to be…

Marstacimab, an experimental treatment in clinical testing for hemophilia A and B, was able to restore clotting in hemophilic blood and plasma tested in laboratory assays, according to a study. If proven safe and effective in patients, the investigational therapy could provide an alternative preventive solution to…

Preventive treatment with Hemlibra (emicizumab) is superior to replacement therapy with factor VIII infusions for reducing bleed rates in people with hemophilia A without inhibitors, a pooled analysis of trials shows. The study “Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A…

Genetic variations in certain immune-related genes, namely the HLA and IL-10 genes, are linked with an increased risk for developing inhibitors against factor VIII replacement therapies in patients with severe hemophilia A. The study that determined that finding, “Risk stratification integrating genetic data for factor VIII inhibitor development…

Mitsubishi Tanabe Pharma is joining forces with researchers from Jichi Medical University to launch a program in Japan focused on developing a gene therapy product to treat patients with hemophilia B, the company announced. The project, titled “R&D of gene 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…

Cell therapies may be a feasible way of achieving a long-term correction of bleeding disorders, preclinical data shared by Sigilon Therapeutics show. A single implant of Sigilon’s engineered cells given to mice enabled durable production of therapeutic levels of blood clotting factors and normalized bleeding time. The technology uses the…

Investigational gene therapy SB-525 can safely induce durable clotting factor VIII (FVIII) activity in patients with severe hemophilia A, preliminary data from the Phase 1/2 Alta clinical trial show. The trial’s most recent results were discussed at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress in…

Bayer’s Jivi, an FVIII replacement therapy for hemophilia A, has a longer half-life and a slower clearance from blood circulation than Eloctate, and may protect against bleeding for longer periods, results of a Phase 1 trial in people with severe disease suggests. The study “Direct comparison of…