Blood levels of two proteins — C-reactive protein (CRP) and vascular endothelial growth factor (VEGF) — involved in inflammation and blood vessel formation are significantly associated with, and may help predict, acute bleeding in the joints of people with severe hemophilia A, a study has found. The study,…

A new combination of gene and cell-based therapy durably delivered therapeutic levels of factor VIII (FVIII) — the blood clotting protein missing or defective in people with hemophilia A — and eased bleeding in a mouse model of the disease, a study from the HemAcure Consortium shows.

The U.S. Food and Drug administration (FDA) has approved Genentech’s Hemlibra (emicizumab-kxwh) for routine prophylactic (preventive) treatment of patients with hemophilia A without factor VIII inhibitors. Hemlibra is now the sole available prophylactic treatment for patients with hemophilia A with and without factor VIII inhibitors that can…

Esperoct (turoctocog alfa pegol) is now available in the U.S. to treat and control bleeding in adults and children with hemophilia A, the therapy’s manufacturer, Novo Nordisk, announced. The treatment’s use was approved by the U.S. Food and Drug Administration (FDA) in February 2019, but its availability…

The European Commission has approved Hemlibra (emicizumab) as a routine preventive treatment for people with moderate hemophilia A without inhibitors. “We welcome the European Commission’s decision to approve Hemlibra also for people with moderate hemophilia A in the EU,” said Levi Garraway, MD, PhD, Roche’s chief medical officer…

Hemlibra (emicizumab-kxwh) was recently granted priority review status by the U.S. Food and Drug Administration following the agency’s acceptance of Genentech’s supplemental biologics license application (sBLA) for the treatment for people with hemophilia A without factor VIII inhibitors. Priority review status is granted to therapies that could…

Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up to two years in people with hemophilia A, a long-term extension study shows. Findings were reported in the study “BAY 81-8973 demonstrated efficacy, safety and joint status improvement…

The European Commission has approved Hemlibra (emicizumab) for routine preventive treatment of bleeding episodes in people who have hemophilia A with factor VIII inhibitors. The antibody therapy is designed to restore the factor VIII, which is deficient in hemophilia A. Hemlibra is being co-developed by Japan’s Chugai Pharmaceuticals, Switzerland’s…

Hemlibra (emicizumab) is safe and effective to treat children younger than 12 who have hemophilia type A and are negative for antibodies against synthetic factor VIII, results from a Japanese clinical study show. The trial findings were reported in the study, “A multicentre, open‐label study of emicizumab given…

People with severe hemophilia A who have two or more self-described problem joints — a person-centric characterization of joints based on underlying joint damage, pain, and mobility impairment, regardless of bleeding — have worse clinical outcomes, including more chronic pain, a new study from researchers in Europe has found.