Marisa Wexler, MS,  senior science writer—

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The U.K.’s National Institute for Health and Care Excellence (NICE) has issued draft guidance recommending that England’s National Health Service (NHS) should not cover the gene therapy Hemgenix (etranacogene dezaparvovec) for adults with hemophilia B.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…

Nuwiq (simoctocog alfa) effectively prevented and controlled bleeds in previously untreated people with severe hemophilia A, according to data from a Phase 3 clinical trial. Full trial results were reported in the study, “Simoctocog alfa (Nuwiq) in previously untreated patients with severe haemophilia A — Final efficacy…

Orsini Specialty Pharmacy has been selected to distribute the new gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for use by individuals with hemophilia A, the company announced in a press release. The one-time treatment from BioMarin Pharmaceutical late last month became the first gene therapy ever approved by…

Nearly two-thirds of children with severe hemophilia A given once-weekly Altuviiio (efanesoctocog alfa) in a Phase 3 clinical trial were completely free of bleeds over about one year of treatment. That’s according to new findings from the recently-completed XTEND-Kids study (NCT04759131), which were presented as late-breaking…

A first adult with hemophilia B in the U.S. has been treated with commercially available Hemgenix (etranacogene dezaparvovec), a recently approved and first gene therapy for the disease, CSL Behring announced. “As part of our longtime promise to patients, CSL Behring is thrilled to mark this latest milestone…

The U.S. Food and Drug Administration (FDA) has agreed to review Medexus Pharmaceuticals’ request to approve Ixinity (trenonacog alfa) for children with hemophilia B younger than 12. Ixinity is already approved by the FDA for adults and adolescents 12 and older. The therapy is indicated for use…