News

Using Hemlibra (emicizumab) as prophylaxis lowers treatment costs considerably for people with hemophilia A compared to preventative factor (FVIII) treatment, according to an economic model study. The model predicts $47,814 in savings to U.S. payers in year one, and a potential for more $3 billion in savings over 20 years…

Many hemophilia patients experience undiagnosed symptoms of depression and anxiety that can impact their ability to cope with the disease and to adhere to treatment, a U.S. survey says. These findings shed light on psychological factors that affect the course of the disease, but with dedicated management and follow-up, depression…

Problematic thrombotic events are fairly rare in people using NovoSeven, among all types of bleeding disorders for which it is approved, including hemophilia, a review study shows. The study, “Thrombotic events with recombinant activated factor VII (rFVIIa) in approved indications are rare and associated with older age,…

Simultaneous use of NovoSeven (recombinant factor VIIa, rFVIIa) and Hemlibra (emicizumab) in patients with hemophilia A is not associated with higher risk of thrombosis, new analysis of the Phase 3 HAVEN program shows. The trials’ findings were reported in the study, “Safety analysis of…

Hemlibra (emicizumab) is safe and effective to treat children younger than 12 who have hemophilia type A and are negative for antibodies against synthetic factor VIII, results from a Japanese clinical study show. The trial findings were reported in the study, “A multicentre, open‐label study of emicizumab given…

Data from the first two patients treated with FLT180a, Freeline’s single-infusion gene therapy for patients with hemophilia B, showed a rise in levels of clotting factor IX to 40%, with levels remaining stable for over a year. Pratima Chowdary, principal investigator for the FLT180a program, presented the data…

Scientists report that hemophilia is three times more prevalent in men worldwide than previously thought and still associated with a poorer life expectancy, especially in low-income countries. Findings of the study, “Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries,”…

Combining immune tolerance induction, used to prevent the development of anti-factor VIII inhibitors, with Hemlibra (emicizumab) is a feasible and safe way of treating children with severe hemophilia A, a study shows. Published in the journal Haemophilia, the report covers clinical outcomes in the first seven patients to be…

Hemophilia A patients undergoing total hip or knee replacement/reconstruction surgeries have lesser blood loss and require a lower transfusion amount if treated with Cyklokapron (tranexamic acid), a study reports. They also are likely to experience less joint pain or swelling and better joint function, and have lower levels…

uniQure has achieved its target patient enrollment goal ahead of schedule for its ongoing Phase 3 HOPE-B trial. The trial is assessing the safety and efficacy of AMT-061 (etranacogene dezaparvovec), the company’s investigational gene therapy for the treatment of patients with moderate and severe…