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…
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A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) continues to prevent bleeds and the need for preventive treatment, or prophylaxis, over at least two years in men with severe hemophilia A, according to updated data from the Phase 3 GENEr8-1 trial. Notably, the activity of…
The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved efanesoctocog alfa — to be sold under the brand name Altuvoct — as a treatment for moderate or severe hemophilia A in adults and children, ages 2 and older. Approved in the European Union in June…
My Facebook friends fall into a few categories: biological family, colleagues and friends from my music and teaching days, high school and college friends and teachers, and my “bleedin’ folk” family. Raising two sons with severe hemophilia is a blessing because of the people in my life (I work…
Long-term treatment with Jivi safely and effectively reduced the number of bleeding episodes in adolescents and adults with severe hemophilia A, according to up to seven years of data from the Phase 2/3 PROTECT VIII trial and its extension study. The new data confirmed the previous findings from…
Canadian Blood Services (CBS) has awarded a three-year tender — an exclusive bulk purchase agreement aiming to minimize medication cost — to Takeda Canada for the purchase of Adynovate, an injectable replacement therapy for the prevention and treatment of bleeding episodes in people with hemophilia A. This…
CAR T-cell therapy, a type of treatment that weaponizes the body’s immune system and was originally developed to treat cancer, for the first time successfully treated a patient with acquired hemophilia A (AHA), a study reported. The study, “Anti-CD19 CAR-T cell therapy for acquired hemophilia A,”…
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 single dose of BIVV001 is safe and effectively increases factor VIII (FVIII) activity over one month in men with severe hemophilia A, lasting longer in the body than the replacement therapy Advate, results from the EXTEN-A clinical trial suggest. Gains in half-life seen with BIVV001 “could signal…
A single dose of the experimental gene therapy AMT-180 promotes clinically meaningful blood-clotting activity, independent of factor VIII levels, in mouse and primate models of hemophilia A, a study shows. These preclinical findings were discussed in a presentation, “Towards AAV5-Mediated Gene Therapy for Hemophilia A with a Factor IX…