The U.S. Food and Drug Administration (FDA) has accepted — under priority review — a marketing application for Valrox (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s investigational gene therapy for hemophilia A. This is the first time the FDA has accepted an application for a gene therapy specifically…

AscellaHealth and Audaire Health together are offering on a hemophilia management program that aims to containing prescription costs and help patients better manage their treatment needs. The program leverages AscellaHealth’s network of specialty pharmacy partners and Audaire’s healthcare technology to offer pharmacy network design, therapy utilization management, and clinical…

Light Chain Bioscience, a unit of Swiss biotech Novimmune, has completed the discovery phase of its collaborative program with Takeda for developing bispecific antibodies — protective proteins produced by the immune system — that mimic the activity of blood clotting factor VIII as a treatment for…

When administered at a high dose, a gene therapy for hemophilia B known as FLT180a seems able to restore the activity of clotting factor IX (FIX) within a normal range in men with moderate-to-severe disease, early data from Phase 1/2 trial show. The findings were announced in an oral presentation,…

Our baby girl turned one earlier this year. Watching Cittie mature makes me reflect on the innocent phase of childhood. Life was so simple back then. I believed games and toys to be major issues of the utmost importance. I stayed late after school and played with my friends.

Esperoct (turoctocog alfa pegol) is now available in the U.S. to treat and control bleeding in adults and children with hemophilia A, the therapy’s manufacturer, Novo Nordisk, announced. The treatment’s use was approved by the U.S. Food and Drug Administration (FDA) in February 2019, but its availability…

Researchers have developed a faster, less expensive, reliable method for non-invasive prenatal diagnosis (NIPD) of hemophilia A. Future, larger studies are required to confirm the utility of the method, which could be used for the NIPD of hemophilia A and type B, as well as other X-linked…

Inheritance patterns in hemophilia are a common discussion topic for many reasons. Among the reasons that people seek information about hemophilia inheritance is their desire to better understand if their future children might be at risk for inheriting the disorder. Not only do people express concern regarding their children, but…

Aptevo Therapeutics announced the dosing of a first patient in a Phase 4 study aiming to extend use of its prophylactic (preventive) treatment Ixinity (coagulation factor IX [recombinant]) to children with hemophilia B  younger than 12 years old. “Initiation of the clinical study of Ixinity in…

Specific genetic changes may account for a larger proportion than previously thought of children with severe hemophilia B who develop inhibitors against coagulation factor IX replacement therapy, according to an international study. The study, “Inhibitor Incidence In An Unselected Cohort Of Previously Untreated Patients With…