A number of clinical studies have reported that bone marrow-derived cells contribute to the production of clotting factor VIII (FVIII), the molecule linked to hemophilia A. Because the current treatment for this bleeding disorder is associated with some disadvantages and risk, hematopoietic stem cell transplant (HSCT) has become an emerging therapy approach…

The National Institute for Health and Care Excellence (NICE) has issued a final guidance recommending the use of Altuvoct (efanesoctocog alfa) in England and Wales as an option for people with severe hemophilia A, ages 2 and older. The National Health Service, the U.K.’s public healthcare system, is…

Joint problems caused by chronic bleeding often trouble people with hemophilia, especially in the knees, ankles, and elbows. A paper by scientists in Spain and the U.S. reviewed existing literature on knee treatments for people with this disease, focusing on best current practices — including a preventive, if inconveniently lifelong, therapy — and future…

The World Federation of Hemophilia (WFH) announced that Takeda has joined its WFH Humanitarian Aid Program, agreeing to large, annual donations of treatments for people with inherited bleeding disorders like hemophilia in developing countries. Takeda,…

Treatment with the extended half-life (EHL) therapies Eloctate (efmoroctocog alfa) and Alprolix (eftrenonacog alfa) was found to safely and effectively control bleeding during major orthopedic surgeries in people with hemophilia A or B, according to real-world data from two Italian centers. The therapies helped patients maintain…

The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data on Roctavian, a potential gene therapy for hemophilia A, took its developer — BioMarin — and others in the hemophilia community by surprise. That decision, announced in a complete response letter of…

Scientists report that hemophilia is three times more prevalent in men worldwide than previously thought and still associated with a poorer life expectancy, especially in low-income countries. Findings of the study, “Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries,”…

Takeda Pharmaceuticals has decided to discontinue Hemofil M (human antihemophilic factor) and Recombinate (recombinant antihemophilic factor), both of which are approved as replacement therapies to prevent and control bleeding episodes in children and adults with hemophilia A. “This was not a decision we made lightly,” Anthea Cherednichenko,…

For adults and adolescents with hemophilia A without inhibitors, Altuviiio (efanesoctocog alfa) may work better than other standard or extended half-life factor replacement therapies at preventing bleeding episodes, while requiring less frequent injections, a study found. Researchers made an indirect comparison of data from XTEND-1 (NCT04161495) and published…

One-month treatment with Hemlibra (emicizumab) and an immunosuppressive medication were effective for patients with acquired hemophilia at rapidly controlling bleeding and reducing the use of other therapies, a single-center study shows. The researchers also noted that using less toxic immunosuppressive agents such as rituximab was effective at blocking…