Europeans with severe hemophilia are largely satisfied with available treatments and able to maintain active lives, according to a survey of healthcare professionals by the biopharmaceutical company Sobi. Results also showed that 23 countries across Africa, South America, and Asia rely entirely on the World Federation of Hemophilia…

Data from a completed Phase 2b trial indicate that dalcinonacog alfa (DalcA) is an effective and safe therapy to prevent bleeding in people with severe hemophilia B. Final results from this open-label study in six patients are expected by June. “We are pleased to have successfully completed the DalcA Phase…

The U.S. Food and Drug Administration (FDA) approved Sevenfact (coagulation factor VIIa [recombinant]-jncw) for the treatment and control of spontaneous bleeding episodes in people, ages 12 and older, with hemophilia A or B with inhibitors. Hemophilia, a genetic disorder, affects the body’s ability to make blood…

Ixinity remains readily available to treat people with hemophilia B despite the current COVID-19 outbreak, Medexus Pharmaceuticals has announced. The company, which acquired Ixinity from Aptevo Therapeutics, also said it will continue working to ensure continued supply throughout the pandemic. “We would like to reassure the patients…

Joint changes detected by magnetic resonance imaging (MRI), and often are missed on radiographs, are a strong predictor of damaging bleeds over the next five years in joints of people with hemophilia, a study suggests. A 10-fold increase in the five-year risk of joint bleeds was found by…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

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…

The likelihood that teenagers and young adults with hemophilia will use a prophylactic replacement therapy as prescribed is mostly influenced by their level of responsibility in taking their infusions, and their own estimates of risk for each activity, a study suggests. To improve treatment adherence, doctors should discuss its importance during…

Adults and children with severe hemophilia A who are receiving Nuwiq (recombinant human coagulation factor VIII) as their first replacement therapy have a similar risk of developing inhibitors as those treated with plasma-derived therapies containing von Willebrand factor, the final results of the NuProtect trial show.