Treatment with an antibody designed to specifically block the anti-blood clotting, or anticoagulant, activity of an enzyme called activated protein C (APC) significantly reduced bleeding and related mortality in hemophilia A and hemophilia B mouse models, a study shows. The antibody, called HAPC1573, was also found to improve…
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The U.S. Food and Drug Administration has approved new vial strengths for Octopharma’s hemophilia A therapy Nuwiq, which means patients will need fewer vials per treatment session. Octapharma USA said single-dose strengths of 2,500, 3,000 and 4,000 International Units will be available in the United States in September. The vials are already…
Prophylactic (preventive) treatment with Adynovate safely and effectively lowers the number of bleedings, including those in target joints, in children and adults with severe hemophilia A, according to data from two Phase 3 clinical trials. Notably, personalized Adynovate treatment targeting higher minimum blood levels of factor VIII (FVIII) —…
BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec safely and significantly reduced bleeding events — including in target joints — and the use of prophylactic clotting factor VIII in adults with severe hemophilia A, data from a Phase 1/2 study show. These results, along with an interim analysis of…
A committee of the European Medicines Agency (EMA) has recommended that the approval of Alhemo (concizumab-mtci) be expanded to cover its use to reduce the risk of bleeding in people with severe hemophilia A and moderate or severe hemophilia B who don’t have inhibitors. The recommendation from…
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Living With Hemophilia: Brandston’s Story
To help prevent bleeds in hemophilia patients, many people living with the disease need to have infusions of clotting factor. In this video from infuzrTV, we meet super cute 5-year-old Brandston. Brandston has severe hemophilia A and needs to have clotting factor infusions on alternate days. MORE: …
Afstyla (lonoctocog alfa) is an injectable factor replacement therapy used to prevent and treat bleeding episodes, including those occurring during surgery, in people with hemophilia A.
Dimension Therapeutics recently revealed positive preclinical results from a novel adeno-associated virus (AAV) that delivers Factor VIII as a therapeutic approach for hemophilia A patients. The results were presented in a poster, titled “Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques” at the 19th American Society of…
Prime Therapeutics and Takeda will work together to establish the value of Advate, relative to its costs and benefits, as a treatment for hemophilia A. According to Prime, work comparing factors like total health care costs and emergency hospital visits allow the company to better evaluate and distinguish…