Doctors discovered a rare combination of blood clotting disorders — acquired hemophilia A alongside an inherited disorder called Factor XII deficiency — in a 70-year-old man set to undergo a leg amputation due to foot gangrene. “This case highlights the importance of considering acquired hemophilia A in unexplained bleeding…

Most people with severe hemophilia A who were given once-weekly treatment with efanesoctocog alfa in the Phase 3 XTEND-1 trial were bleed-free over a year on treatment. The therapy also improved joint and overall physical health and eased pain intensity in the participants compared with previous treatments. Results…

A gene therapy being developed by ASC Therapeutics restored levels of the clotting factor missing in hemophilia A in a mouse model of the disease, and further tests in primates and human cells suggest the therapy might also work for people, the company announced. Results from…

A recall on Ferring Pharmaceuticals‘ intranasal desmopressin compounds — used to treat mild to moderate hemophilia A and von Willebrand disease type 1 — is expected to affect the availability of these products until at least mid- to late-2021, according to an advisory from the…

Regeneron Pharmaceuticals and Intellia Therapeutics are expanding their collaboration to develop hemophilia A and B treatments using the CRISPR/Cas9 gene editing technology. The two companies signed a six-year agreement in 2016 to develop, license, and commercialize gene editing-based therapies. “We’re pleased to expand our work…

More than half the adults with hemophilia A in the Phase 3 GENEr8-1 clinical trial remain completely free of bleeds needing treatment, four years after a single dose of the gene therapy Roctavian (valoctocogene roxaparvovec-rvox). Also, activity levels of factor VIII (FVIII), the faulty clotting protein in hemophilia…

Continuous into-the-vein (intravenous) infusions of Advate immediately before and after major orthopedic surgery is as safe and effective as standard, short, bolus infusions at preventing bleeds in men with moderate-to-severe hemophilia A. That finding from a Phase 3/4 clinical trial — the first controlled trial to compare the two types…

Eligible patients with severe hemophilia A in Germany should now have improved access to Roctavian (valoctocogene roxaparvovec-rvox) — a gene therapy intended to reduce bleed risk in certain adults with the bleeding disorder — given a new agreement between its developer BioMarin Pharmaceutical and the German National Association…

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…

Hemlibra (emicizumab) is an effective and less costly therapy for hemophilia A patients who have substances in their blood that prevent clotting factor therapies from working as well as they should, according to a therapy rating organization. The Institute for Clinical and Economic Review (ICER) updated an initial report on Hemlibra…