Europe’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion supporting the approval of Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in hemophilia A patients with factor VIII inhibitors. Hemlibra is a humanized monoclonal antibody that binds to activated factor IX and factor X to…

Bayer’s investigational gene therapy BAY 2599023 safely and effectively increased the levels of clotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated in a Phase 1/2 clinical trial, preliminary data show. The ongoing trial (NCT03588299; 2017-000806-39) is enrolling…

The U.S. Food and Drug Administration (FDA) has granted priority review for the approval of efanesoctocog alfa, with a decision on the potentially longer-lasting hemophilia A treatment due early next year. The regulatory agency accepted an application from developers Sobi and Sanofi requesting approval of the…

Using a combination of genetic and functional approaches, scientists have identified new mutations in parts of the F8 gene that do not code for factor VIII (FVIII) in a small group of French and Canadian patients with hemophilia A. These results were described in the study, “The…

A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in lowering the frequency of bleeds and the need for infusions in men with severe hemophilia A. These preliminary findings from an ongoing Phase 1/2 trial (NCT03734588) were presented by Spencer Sullivan, MD,…

Prophylactic (preventive) treatment with Adynovate safely and effectively lowers the number of bleedings, including those in target joints, in children and adults with severe hemophilia A, according to data from two Phase 3 clinical trials. Notably, personalized Adynovate treatment targeting higher minimum blood levels of factor VIII (FVIII) —…

Cell therapy with endothelial progenitor cells and stem cells genetically engineered to produce a functional clotting factor VIII (FVIII) may provide a stable and long-term treatment for hemophilia A, a mouse study has found. The study shows that the transplant of cells…

A first patient has been dosed in XTEND-Kids, a Phase 3 clinical trial evaluating the safety and efficacy of the investigational replacement therapy efanesoctocog alfa in boys with severe hemophilia A. Hemophilia A is caused by the lack of a functional blood clotting protein called factor VIII…

Almost three years of treatment with Jivi (damoctocog alfa pegol) reduced the annual bleeding rate among a large, multinational group of previously treated hemophilia A patients, a real-world study reports. The number of patients without joint bleeds increased during the study and…

Treatment with Esperoct led to stabilization and/or improvement of health-related quality of life (HRQoL) in patients with severe hemophilia A from all age groups, a study suggests. The study, “Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant…