Health Canada has approved Hemlibra (emicizumab) as a treatment to prevent or reduce bleeding in Hemophilia A patients in Canada who have factor VIII inhibitors. Hemlibra is an antibody therapy designed to combine factors IX and X of the blood clotting series and promote clotting. Restoring the…

Healthcare costs, particularly related to disease-specific medications, are especially high for people with hemophilia A or hemophilia B on preventive treatments and/or for those with inhibitors. That’s according to a study in France, which also found that patients have a significant clinical burden, with a higher risk of…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to efanesoctocog alfa, an investigational factor VIII (FVIII) replacement therapy for treating hemophilia A, Sanofi, one of the therapy’s developers, announced in a press release. This designation is given to accelerate the development and review of medications…

Advate is an effective and well-tolerated treatment among patients with hemophilia A, according to an observational, long-term, Japanese study. The treatment showed better results when used as a preventive, rather than an on-demand, therapy. The findings were reported in an article titled “Inhibitor development, safety and efficacy of…

A gene therapy designed to provide hemophilia A patients with a highly functional version of factor VIII (FVIII), the blood clotting protein they lack, could offer more sustained bleed control than existing gene therapies, preclinical research suggests. The current approach in hemophilia A gene therapy is to provide a…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application…

An experimental cell-based therapy using Sernova’s innovative medical device, Cell Pouch, safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and reduced bleeds in a mouse model of the disease. These are the findings of a study, “…

The U.K.’s National Institute for Health and Care Excellence (NICE) has recommended Altuvoct (efanesoctocog alfa) be available through the National Health Service (NHS), England’s public healthcare system, as a treatment option to manage and prevent bleeds in people with severe hemophilia A, ages 2 and older. This decision…

The U.S. Food and Drug Administration has approved Bayer’s Jivi, previously known as BAY94-9027, as a preventive therapy for hemophilia A in patients ages 12 and older, the company announced. The recommended regimen is for Jivi to be administered intravenously (into the blood) two times a week (30-40 IU/kg),…

The blood-clotting protein factor VIII (FVIII) that’s missing or faulty in people with hemophilia A is flexible and adopts multiple shapes, some of which favor the binding of neutralizing antibodies that can reduce the effectiveness of FVIII replacement therapies, researchers have learned. “This helps explain why some people…