Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…

Note: This article was updated June 26, 2024, to clarify that the analysis of fitusiran in surgery was conducted in patients receiving prophylactic treatment with the therapy. The U.S. Food and Drug Administration (FDA) is reviewing a request to approve fitusiran, an under-the-skin injection therapy, as a treatment for adults…

Efanesoctocog alfa, a long-lasting factor VIII (FVIII) replacement therapy, has now been approved in the European Union for hemophilia A. It will be marketed under the brand name Altuvoct. Altuvoct can be used as an on-demand treatment for bleeding episodes, a once-weekly prophylactic (preventive) therapy to reduce the…

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…

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…

Immune tolerance induction (ITI), a type of treatment that’s designed to eliminate neutralizing antibodies, or inhibitors, against clotting factors for people with hemophilia A, may work just as well across different racial and ethnic groups, a study finds. The findings don’t “support the hypothesis that ITI response varies according…

Ixinity (trenonacog alfa) has been approved by the U.S. Food and Drug Administration (FDA) for use in children younger than 12 with hemophilia B. This label expansion by the FDA means that the factor IX (FIX) replacement therapy, sold by Medexus Pharmaceuticals, can now be used in…

TiumBio is preparing to launch a Phase 1b clinical trial to test TU7710, its experimental treatment to manage bleeding in people with hemophilia who have inhibitors, that is, neutralizing antibodies that can lower standard therapies’ effectiveness. “Our team has been developing TU7710 as an innovative hemophilia treatment option,”…

In observance of Bleeding Disorders Awareness Month, the integrated infusion therapy provider BioTek reMEDys is calling attention to the importance of safe treatment use and education among people with conditions such as hemophilia and von Willebrand disease. Each March, the bleeding disorders community and supporters seek to heighten…