Marisa Wexler, MS,  senior science writer—

Treatment with platelets stripped of their natural sugar molecule coating helped to prevent inhibitors, or neutralizing antibodies, from forming against clotting factor VIII (FVIII) replacement therapy in a mouse model of hemophilia A, a study reported. Platelets promote blood clotting, and they are generally thought to boost inflammation…

Altuviiio (efanesoctocog alfa) has been approved in Japan and Taiwan as a treatment to prevent and control bleeding in people with hemophilia A. The Japanese Ministry of Health, Labor, and Welfare (MHLW) granted Altuviiio marketing authorization on Sept. 25, according to Sanofi, the company that markets…

Treatment with Idelvion (albutrepenonacog alfa) was effective in a real-world setting at controlling bleeds for five people with hemophilia B at a center in Spain. That’s according to data reported in the study, “Benefits of rIX-FP prophylaxis in patients with Haemophilia B: real-world evidence from…

The U.S. Food and Drug Administration (FDA) is considering updating the prescribing information for Altuviiio (efanesoctocog alfa) to include final data on a clinical trial that tested the now-approved therapy in children with hemophilia A. Sanofi, which markets Altuviiio in the U.S., submitted a supplemental biologics license…

To help advance new treatments for hemophilia and other blood disorders, Pathway to Cures (P2C) has invested $250,000 in Anvesana, a company working to develop therapies based on RNA biology. “Anvesana is delighted to be working with Pathway to Cures to develop therapeutics that will bring life altering…

A person in Germany with severe hemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), the first time the gene therapy has been used commercially in Europe, according to BioMarin Pharmaceutical, its developer. “Today represents an important milestone for the hemophilia community and for patients and…

Treatment with Ixinity (trenonacog alfa) can safely control bleeds in children younger than 12 with severe or moderately severe hemophilia B. That’s according to data from a Phase 3/4 clinical trial that Medexus Pharmaceuticals, the company that markets Ixinity, presented at the National Hemophilia Foundation’s recent Bleeding…

The U.K.’s National Institute for Health and Care Excellence (NICE) has issued draft guidance recommending that England’s National Health Service (NHS) should not cover the gene therapy Hemgenix (etranacogene dezaparvovec) for adults with hemophilia B. The recommendation is based on uncertainty surrounding the long-term efficacy of the…

Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy approved for adults with severe hemophilia A, is expected to be widely available in the U.S. this month, according to its developer, BioMarin. BioMarin also is working with authorities in Germany, Italy, and France to secure market access to…

Roche is initiating a Phase 3 clinical trial to test the experimental gene therapy SPK-8011 in people with hemophilia A. The company announced the move in its 2023 half-year results. At the same time, however, Roche is discontinuing the development of another investigational hemophilia A gene therapy…