Jivi (damoctocog alfa pegol) is an approved lab-made replacement therapy designed to prevent or treat bleeding episodes in previously treated children and adults with hemophilia A. It is administered via into-the-vein (intravenous) infusions.
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PEGylation is one of the molecular technologies that has been used to create extended half-life replacement therapies for hemophilia. These longer-lasting therapies don’t need to be administered as often, reducing the treatment burden for patients.
The European Medicines Agency has recommended marketing authorization for Shire’s Adynovi (BAX-855) as a treatment for adults and adolescents with hemophilia A. Approval would mean the therapy could be used twice a week to prevent bleeding episodes and to counter bleeding when episodes do occur. The U.S. Food and Drug Administration…
A modified version of the coagulation protein factor X was able to significantly reduce bleeding in a mouse model of hemophilia A, and may offer a way of treating people with this disease due to factor VIII deficiency, a study suggests. Lab work also showed a potential for treating people…
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Professional Help Essential for Parents Telling Daughters of Hemophilia Carrier Risk, Study Reports
An interview-based study from Japan found professional support was necessary when mothers inform their daughters they might be carriers for hemophilia, as the women found themselves “not knowing what to do” and expressing “anxiety due to uncertainty.” In Japan, parents are often responsible for telling a child that a genetic…
Two presentations at the 2016 American Society of Hematology Annual Meeting (ASH) highlight ongoing treatment advances for hemophilia B. The data suggest that life with hemophilia could soon become easier to manage. Long-acting replacement therapy The ASH meeting, held in San Diego on Dec. 3-6, was a platform for CSL Behring to…
Female hemophilia AÂ patients and carriers have more frequent bleeding than males, yet a much smaller proportion receive replacement therapy to prevent or stop them, a U.S. study finds. The medical and pharmacy costs for women with hemophilia A or carriers were up to about 14 times lower…
A team led by Lurie Children’s Hospital of Chicago researchers reported a case study of a newborn with combined spontaneous splenic rupture and severe hemophilia A. This baby, whose case is believed to be the first documented, was successfully treated without surgical intervention. Results from the study, “Successful medical management of a…
Preventive treatment with Hemlibra (emicizumab) improves health-related quality of life (HQRoL) in children with hemophilia A who develop inhibitors, or neutralizing antibodies, against engineered factor VIII (FVIII), an essential blood-clotting protein, a new analysis of a Phase 3 trial shows. Such Hemlibra therapy also reduces the burden on…