The U.S. Food and Drug Administration (FDA) has approved the one-time gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for eligible adults with severe hemophilia A. Roctavian now becomes the first gene therapy to win FDA approval for the treatment of hemophilia A in the U.S. “Today’s approval of Roctavian builds…
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…
The Hemophilia Federation of America (HFA) is now offering a “clinical trial finder” to help people with hemophilia and other bleeding disorders more easily connect with the studies they are eligible for. The platform enables visitors to search across all clinical trials, regardless of sponsor, for key…
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…
A researcher at the University at Buffalo has won a three-year grant worth $1.5 million to develop a way to tame a patient’s immune system and prevent it from rejecting factor VIII as a replacement therapy for hemophilia A. “My interest in hemophilia started when I read a mother’s…
People with hemophilia A who start on preventive treatment in the first years of life generally report better health-related quality of life and joint health, a study reports. “Our results indicate that delayed start of prophylaxis in an older cohort with severe [hemophilia A] can still achieve excellent bleeding…
Electrically stimulating the vagus nerve — the main set of nerve fibers that control involuntary bodily functions — significantly reduced blood loss and sped up clot formation after injury in a mouse model of hemophilia A, according to a recent study. Controlling bleeding by stimulating the vagus nerve was…
Incidences of bleeding from playing sports are rare among people with hemophilia, and more likely to occur in those with lower levels of clotting factors — proteins that play a key role in promoting blood clotting — a study in the Netherlands has found. More specifically, the study reveals…
Weekly treatment with the experimental therapy marstacimab (PF-06741086) was found to significantly reduce bleed rates for adult and adolescent hemophilia A and B patients without inhibitors relative to standard therapies. In so doing, it met the main goal of the pivotal Phase 3 BASIS clinical trial, designed to…
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