A therapy designed to promote the growth of regulatory T-cells (Tregs) in the body prevented inhibitor development in a mouse model of hemophilia A that was treated with factor replacement therapy, a study showed. Inhibitors, a type of neutralizing antibody some patients develop that can make replacement therapies…
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A gene therapy designed to provide hemophilia A patients with a highly functional version of factor VIII (FVIII), the blood clotting protein they lack, could offer more sustained bleed control than existing gene therapies, preclinical research suggests. The current approach in hemophilia A gene therapy is to provide a…
A single dose of Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for severe hemophilia A from BioMarin Pharmaceutical, maintains factor VIII (FVIII) levels at near-normal or normal levels for up to five years, with most patients staying off prophylaxis, or preventive treatment. These lasting benefits were observed in GENEr8-1…
Prophylactic replacement therapy to prevent bleeds was more cost-effective than on-demand treatment of active bleeds for children and adolescents in China with moderate or severe hemophilia A without inhibitors, a study reported. The superior cost-effectiveness of prophylaxis over on-demand treatment was mainly driven by the on-demand costs of treating…
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…
The World Federation of Hemophilia (WFH) has created a universal case report form for the collection of core data on hemophilic patients in a standardized format that will include relevant diagnosis, therapies, and outcome. Previous WFH forms provided only an overview of the number of patients and access to treatment.
Treatment with Hemlibra (emicizumab) can help to effectively manage bleeding in individuals with acquired hemophilia A (AHA), according to a new study. While clinical trials will need to be done to fully assess Hemlibra as an AHA therapy, the researchers said this medication also has the potential to…
Biogen and Sobi announced that they will be presenting updated and long-term data on two approved hemophilia treatments, Eloctate  (marketed as Elocta in Europe) for hemophilia A, and Alprolix for hemophilia B, at the World Federation of Hemophilia (WFH)’s 2016 World Congress next week. Specifically, data from separate Phase 3 extension studies evaluating each treatment’s safety and…
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Why Hemophilia Is Called ‘A Royal Disease’
Hemophilia is a rare blood condition where people do not have the clotting factor which enables their blood to clot when bleeding. It’s an inherited disease that’s usually passed from mother to son. It’s also a disease that’s been prevalent in European royal families. MORE:Â A brief history of hemophilia…
People with severe hemophilia A who have two or more self-described problem joints — a person-centric characterization of joints based on underlying joint damage, pain, and mobility impairment, regardless of bleeding — have worse clinical outcomes, including more chronic pain, a new study from researchers in Europe has found.