The U.S. Food and Drug Administration (FDA) has approved fitusiran as a routine prophylactic therapy to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older, with or without inhibitors, or antibodies that can make treatment less effective.
The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the…
Antibody therapy Mim8 (denecimig) was well tolerated and provided effective bleed control in children with hemophilia A regardless of inhibitor status. That’s according to interim data from the now-complete Phase 3 FRONTIER3 study (NCT05306418), which enrolled children ages 1-11. These findings were consistent with recent data from…
The U.S. Food and Drug Administration (FDA) has approved Novo Nordisk’s concizumab, which will be sold under the brand name Alhemo, as a daily treatment to prevent or reduce the frequency of bleeding episodes in certain adults and adolescents with hemophilia. Eligible patients are those with hemophilia…
Preventive treatment with Altuviiio (efanesoctocog alfa) provided better bleed prevention than Hemlibra (emicizumab) in adults and adolescents with hemophilia A, according to an indirect comparison of Phase 3 clinical trial data. “While it should be borne in mind that this is an indirect comparison and, as such,…
Nearly two-thirds of hemophilia A patients given a single infusion of the investigational gene therapy giroctocogene fitelparvovec were free of bleeding episodes a median of almost three years after being treated, according to full data from the Phase 3 AFFINE clinical trial. These new data add to top-line…
Real-world use of the extended half-life factor replacement therapy Elocta (efmoroctocog alfa) was associated with better bleed control than standard half-life products among people with hemophilia A, according to findings from an observational study in Europe. Elocta, sold as Eloctate in the U.S., was also associated with fewer…
A gene therapy designed to provide hemophilia A patients with a highly functional version of factor VIII (FVIII), the blood clotting protein they lack, could offer more sustained bleed control than existing gene therapies, preclinical research suggests. The current approach in hemophilia A gene therapy is to provide a…
A team of U.S. researchers has developed a new gene-editing strategy that could make the approach safer and more efficient for treating certain diseases, such as hemophilia. In preclinical studies, the gene-editing technique was shown to enable fast and efficient delivery of healthy genetic material to cells, while bypassing…
The incidence of acquired hemophilia A (AHA) was nearly twice as high in Hong Kong than in Caucasian populations previously observed, a study found. Patients in Hong Kong tended to be older and have co-existing medical conditions, with age being a significant predictor of mortality and failure to achieve…
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