Iqra Mumal, MSc,  —

The U.S. Food and Drug Administration has approved a larger vial size — 3,500 IU — of CSL Behring’s Idelvion (albutrepenonacog alfa) for the treatment of hemophilia B patients. Idelvion is supplied as a powder for intravenous use and must be reconstituted, or mixed with a liquid (called…

Hemophilia A patients who do not refrigerate their bleeding prevention therapy are more satisfied with it and report fewer restrictions on their daily activities, a study finds. Researchers also discovered that most of the patients were unaware that they could keep their recombinant factor VIII clotting therapy at room temperature.

Workers with hemophilia are at a much higher risk of stroke, developing a joint disease, or needing a knee or hip replacement than the general population, Taiwanese researchers report. The study, “Risk of major comorbidities among workers with hemophilia: A 14-year population-based study,” was published in the journal…

Researchers report that the Medtep Hemophilia online platform helped hemophilia patients improve their adherence to preventive treatment, while increasing their quality of life and their perception of their illness. The study titled, “Effectiveness of the Medtep Hemophilia online platform for adherence to prophylactic treatment…

New research shows that bilateral total knee arthroplasty — or reconstruction of the joint — is a safe and cost-effective treatment for patients with hemophilic arthropathy (a hemophilia degenerative joint disease). The study, titled “Simultaneous bilateral total knee arthroplasty in patients…

Nuwiq, a fourth-generation genetically engineered version of blood-clotting factor VIII, is a safe and effective way of controlling bleeding in people with severe hemophilia A during and after surgery, a study reports. The research, “Efficacy and safety of Nuwiq® (human-cl rhFVIII) in patients with severe haemophilia A undergoing…

The U.S. Food and Drug Administration (FDA) has approved Hemlibra (emicizumab-kxwh) for routine prophylaxis to prevent or lessen bleeding episodes in children and adults with hemophilia A with factor VIII inhibitors, Genentech announced. Hemlibra is first newly approved treatment for this patient group in almost 20 years, the…

uniQure plans to advance its investigational gene therapy AMT-061 into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B. Gene therapies are being develop to treat diseases caused by genetic mutations. It involves replacing the faulty gene in patients with a healthy gene.

Shire has acquired worldwide rights to the antibody-based therapy that Novimmune developed as a treatment for hemophilia A. Geneva-based Novimmune creates therapies for inflammatory diseases, autoimmune diseases and cancer. It uses antibodies that target tumor-associated proteins and the immune checkpoint protein CD47. Shire, which is based in Dublin, is a…

European regulators have granted orphan drug status to CB 2679d, a hemophilia B therapy that Catalyst Biosciences developed to prevent bleeding episodes. The therapy, known as ISU304 in South Korea, prevents bleeding by increasing hemophilia B patients’ level of a blood protein called Factor IX, which is deficient in the disease.