A significant proportion of bleeds in people with hemophilia A are left untreated, according to an analysis of data from an observational study. Because only treated bleeds are typically reported in clinical trials, these findings suggested the full disease burden is not adequately captured in many studies. As such,…

Treatment with marstacimab (PF-06741086), an experimental antibody-based therapy developed by Pfizer, reduced the number of bleeds in patients with severe hemophilia who took part in a Phase 1b/2 clinical study. This held true regardless of whether patients had hemophilia A or hemophilia B, or had inhibitors…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SerpinPC for treating hemophilia B. Orphan drug status is awarded to therapeutics intended to treat rare conditions, defined as those affecting fewer than 200,000 people in the U.S. The designation provides financial incentives to support clinical…

Hemophilia A patients who develop inhibitors — neutralizing antibodies made by immune cells against clotting factors — have more expression, or activity, of genes involved in activating the immune system, a small study reports. “The results of our study reveal that there is an upregulation of genes involved with…

The U.S. Food and Drug Administration (FDA) has granted priority review for the approval of efanesoctocog alfa, with a decision on the potentially longer-lasting hemophilia A treatment due early next year. The regulatory agency accepted an application from developers Sobi and Sanofi requesting approval of the…

Adults with hemophilia and ankle joint damage taking part in a yearlong pilot study largely reported that two injections of hyaluronic acid, which acts to lubricate the joint, eased ankle pain and improved movement. The treatment, given six months apart in this U.K. trial, also enabled most of these people…

Switching to Hemlibra (emicizumab) can add more than $100,000 a year to healthcare costs for people with hemophilia A in the U.S. who were previously using preventive replacement therapies, according to a new analysis. “Increased use of [Hemlibra] may cause an increase in economic burden for payers without…

The U.S. Food and Drug Administration has approved Novo Nordisk’s Rebinyn (nonacog beta pegol) as a routine preventive treatment for bleeding episodes in adults and children with hemophilia B. Rebinyn, sold under the brand name Refixia in the EU, is a replacement therapy that had originally…

The extended half-life replacement therapies Eloctate and Alprolix can be used to effectively manage bleeding during surgery in people with hemophilia, according to a new study based on Phase 3 clinical trial data. These findings highlight the “efficacy and safety of these products in a broad age range of…

Most people with hemophilia A or B who switched from an approved prophylactic (preventive) therapy regimen to once-monthly treatment with fitusiran in the ATLAS-PPX clinical trial had no bleeds over seven months on the investigational therapy, new data show. Fitusiran also reduced bleeding rates regardless of the presence…