News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B. This designation is given to therapies that are meant to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. It offers…

A European Medicines Agency (EMA) committee has issued a positive opinion recommending conditional marketing authorization of gene therapy Durveqtix (fidanacogene elaparvovec), developed by Pfizer, to treat adults with severe and moderately severe hemophilia B. The European Commission will review the recommendation from the EMA’s Committee for Medicinal…

In Brazil, hemophilia markedly impairs patients’ quality of life and ability to work, with up to 82.6% of people with the disorder being unemployed, a review study finds. Barriers to treatment include long distances to centers, lack of coordination of specialized and emergency care, and restricted access to rehabilitation.

The U.S. Food and Drug Administration (FDA) has cleared clinical testing in adults of BE-101, a B-cell treatment candidate for hemophilia B being developed by Be Biopharma. The Phase 1/2 trial, called BeCoMe-9, will be a first-in-human study designed to assess the safety and preliminary efficacy of BE-101…

In real-world settings, Hemlibra (emicizumab-KXWH) can safely control bleeds in children with hemophilia A better than previous treatment regimens, a study reports. Hemlibra’s improved efficacy was more pronounced among patients with inhibitors, or neutralizing antibodies targeting factor VIII (FVIII), which is the clotting protein missing in people with…

Weekly and monthly treatment with Mim8, an investigational antibody therapy, was seen to be better than on-demand or standard preventive treatments at reducing bleeding episodes in patients with hemophilia A, regardless of inhibitor status, who took part in a clinical trial. Top-line results from the global Phase 3…

Pfizer is preparing to launch its hemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt) after securing a U.S. approval for the treatment last month. Beqvez is the second gene therapy to be approved for adults with moderate to severe hemophilia B in the U.S., following the clearance of…

The U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information for Altuviiio (efanesoctocog alfa), a long-lasting factor VIII (FVIII) replacement therapy for hemophilia A, to include the full results from a clinical trial, the XTEND-Kids study. As requested by Sanofi, which markets…

Artificial intelligence (AI) can comb through ultrasound scans to detect joint bleeding and inflammation occurring as a result of hemophilia, a study has found, suggesting it may help doctors make quicker and more accurate diagnoses. “AI models have the potential to aid diagnosis and enable earlier therapeutic interventions, helping…

Gentle electrical stimulation may help liver cells uptake gene therapies, such as those used to treat hemophilia, according to new research by a team of U.S. scientists. In lab studies, the team found that liver cells exposed to a brief electrical field more easily took up a viral carrier,…