Switching to Rebinyn (nonacog beta pegol) from other standard or extended half-life replacement therapies led to “robust” reductions in annual treatment costs among hemophilia B patients in Canada, a study based on real-world data reported. Its findings add to an earlier and real-world analysis of patients who…

My bleeding disorders, hemophilia B and von Willebrand disease, have significantly affected my family. As I’ve aged, my appreciation for my family has grown tremendously. I’ve come to realize that hemophilia‘s impact isn’t just limited to me; it’s also affected my family in profound ways. True, patients…

Concizumab has been approved as a preventive treatment in Canada for hemophilia B patients ages 12 and older who are positive for factor IX inhibitors and need a routine prophylactic to prevent bleeds or reduce their frequency. This is the first approval for the daily under-the-skin injectable therapy, which…

Higher replacement factor doses and the use of preventive treatments are just two ways prescription practices for hemophilia have changed over time, according to a survey of specialists over 22 years. The surveys also show the impact of Hemlibra’s (emicizumab-KXWH) approval and showcase the expectations for gene therapy,…

Significant disparities in access to hemophilia treatment exist around the world. In general, people with hemophilia can live a relatively normal life if their condition is properly managed and they have access to appropriate medical care. However, in developing countries, access to adequate treatment and medical care can…

Despite preventive treatment, people with hemophilia A or B report lower quality of life, mainly due to poor joint health, a study in Sweden shows. No significant difference was found between these two patient groups, who report high frequency of pain, mobility problems, and mental health issues like anxiety…

For children with severe hemophilia A who develop inhibitors — neutralizing antibodies that reduce the effectiveness of standard therapies — starting treatment to eliminate inhibitors earlier is associated with a higher chance of success, a recent study reports. The study, “Low-dose immune tolerance induction therapy in…

Hemgenix (etranacogene dezaparvovec), the first gene therapy approved for adults with hemophilia B, has been shown to work better than standard preventive replacement therapy to lower the frequency of bleeds — both spontaneous and joint bleeds — in this patient population. The therapy also was superior in…

Most children with hemophilia A given Altuviiio (efanesoctocog alfa) once a week were bleed-free throughout about a year of treatment. That’s according to top-line data from the Phase 3 XTEND-Kids trial (NCT04759131) announced by Sobi and Sanofi, the therapy’s co-developers, less than a month after…

Note: This story was updated Feb. 24, 2023, to correct the secondary headline since there are several other recombinant factor VIII therapies available.  Altuviiio is a first-in-class therapy, but not the first. Efanesoctocog alfa, Sobi and Sanofi’s first-in-class, long-lasting recombinant or man-made factor…