The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Hympavzi (marstacimab) for the prevention of bleeding episodes in people with both hemophilia A and hemophilia B, ages 12 and older. The therapy, already approved in the U.S. and the European Union, is designed to…
Search results for:
My sons have severe hemophilia A and are 10 years apart. Such a considerable age difference once posed challenges, especially when they were younger. Back then, my oldest son, Julian, felt he and his brother, Caeleb, didn’t have common interests. I reminded Julian that siblings close in age…
News
Valoctocogene Roxaparvovec Study Doses 1st Hemophilia Patient with Pre-existing AAV5 Antibodies
A Phase 1/2 trial (NCT03520712) testing BioMarin Pharmaceutical’s investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with pre-existing AAV5 antibodies has dosed its first participant. “Administration of valoctocogene roxaparvovec to this first patient seropositive for the AAV5 capsid is an important next step in our plan to…
Once-daily preventive treatment with Novo Nordisk’s experimental therapy concizumab significantly reduced bleeding rates in people with both hemophilia A and B with inhibitors, according to new data from the Phase 3 explorer7 trial. In fact, bleeding rates in patients receiving concizumab in trial decreased by more than 85%. Based…
Genetic variations in certain immune-related genes, namely the HLA and IL-10 genes, are linked with an increased risk for developing inhibitors against factor VIII replacement therapies in patients with severe hemophilia A. The study that determined that finding, “Risk stratification integrating genetic data for factor VIII inhibitor development…
In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin therapy for hemophilia A and B patients with inhibitors, which was being evaluated in an international Phase 3 clinical trial. “Based on several factors including a recently updated feasibility assessment,…
Prophylactic replacement therapy to prevent bleeds was more cost-effective than on-demand treatment of active bleeds for children and adolescents in China with moderate or severe hemophilia A without inhibitors, a study reported. The superior cost-effectiveness of prophylaxis over on-demand treatment was mainly driven by the on-demand costs of treating…
Adynovate (antihemophilic factor, rurioctocog alfa pegol), sold as Adynovi in Europe, is a recombinant (lab-made) clotting factor VIII (FVIII) concentrate given as an intravenous (into-the-vein) injection to prevent and control bleeding in children and adults with hemophilia A.
Grand Life Sciences Group has entered into an exclusive collaboration agreement with Belief Biomed (BBM) to commercialize BBM-H803, BBM’s one-time investigational gene therapy for hemophilia A, in Greater China if it is eventually approved in the region. The agreement covers Mainland China, as well as Hong Kong,…
Less-frequent and easier administration of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — is likely to boost treatment adherence among patients with severe disease, a South Korean study has found. The study, “Patients’ and parents’…