The blood-clotting protein factor VIII (FVIII) that’s missing or faulty in people with hemophilia A is flexible and adopts multiple shapes, some of which favor the binding of neutralizing antibodies that can reduce the effectiveness of FVIII replacement therapies, researchers have learned. “This helps explain why some people…
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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…
A lower dose of regular, preventive therapy with factor VIII concentrates was better at reducing bleeding events and improving joint function than on-demand treatment in children with hemophilia in Thailand, a study shows. The study, “Prophylactic vs episodic treatment to prevent bleeds and preserve joint function…
The five adults with hemophilia A who died while using Hemlibra (emicizumab-kxwh) to treat their disease did not die of causes related to the treatment, manufacturer Genentech said in a statement. “In each of these cases, the assessment of the treating physician or investigator was that the cause of death was unrelated to…
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…
Jivi (BAY94-9027), a lab-made version of the blood clotting factor VIII (FVIII), has been approved by the European Commission for the prophylactic (preventative) treatment of hemophilia AÂ in patients older than 12, Bayer announced. The recommended prophylaxis for Jivi administration is every five days, every…
High levels of antibodies against clotting factor VIII (FVIII) were associated with a slower response to immunosuppressive treatments in a small group of people with acquired hemophilia A in new study in China. Of 34 patients, 29 achieved a complete response to their first-line immunosuppressive therapy. Still, a lower…
Infection and related complications pose a serious risk to people with acquired hemophilia A (AHA), accounting for more than half of the deaths in a registry study in Spain. About 15% of participants in the study experienced at least one infection requiring treatment or hospitalization, the study found. About…
Shire has asked the U.S. Food and Drug Administration to grant Investigational New Drug status to its hemophilia A treatment SHP654. The gene therapy helps generate a clotting protein known as factor VIII that is missing or defective in hemophilia A. Shire said an FDA approval of its request will…