News

The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Hympavzi (marstacimab) for the prevention of bleeding episodes in people with both hemophilia A and hemophilia B, ages 12 and older. The therapy, already approved in the U.S. and the European Union, is designed to…

The first hemophilia B patient in Austria has been treated with the gene therapy Hemgenix (etranacogene dezaparvovec), according to its developer, CSL Behring. “We are proud and grateful to offer hemophilia B patients in Austria a treatment option that has the potential to transform their lives,” Beate Natmessnig,…

The National Institute for Health and Care Excellence (NICE) has issued a final guidance recommending the use of Altuvoct (efanesoctocog alfa) in England and Wales as an option for people with severe hemophilia A, ages 2 and older. The National Health Service, the U.K.’s public healthcare system, is…

World Hemophilia Day is April 17, and this year it will focus on the challenges of women and girls living with hemophilia and other bleeding disorders. The World Federation for Hemophilia (WFH) said the theme of its campaign is “Access for all: women and girls bleed too.” “Today,…

The gene therapy BBM-H901 (dalnacogene ponparvovec) has been approved in China for adults with moderate to severe hemophilia B, making it the first approved gene therapy for the disorder in the country. BBM-H901 was developed and will be manufactured by Belief Biomed (BBM), but it will be commercialized…

Health Canada has approved Altuviiio (efanesoctocog alfa) to treat children, adolescents, and adults with hemophilia A, with the aim of preventing and controlling bleeds, including those occurring around the time of surgery. “This approval represents important progress in the treatment of patients with hemophilia A in Canada,” Stephanie…

The cost of the one-time hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) will now be covered for eligible patients in Germany through the country’s public healthcare system. This comes through a reimbursement price agreement between developer CSL Behring and GKV-Spitzenverband, the German National Association of Statutory Health…

Despite high rates of bone health issues in hemophilia and von Willebrand disease (VWD), the routine use of DEXA scans remains low across hemophilia treatment centers (HTCs) in the U.S., a study reports. DEXA, or dual-energy X-ray absorptiometry, is a type of imaging test that uses low-dose X-rays to…

The U.S. Food and Drug Administration (FDA) has approved fitusiran as a routine prophylactic therapy to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older, with or without inhibitors, or antibodies that can make treatment less effective.

Prophylactic, or preventive, treatment with Alhemo (concizumab) is associated with better quality of life and a lower treatment burden for people with hemophilia A or B without inhibitors, according to patient-reported data from a Phase 3 trial. The ongoing study, called explorer8 (NCT04082429), is investigating the…