A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of understanding how treatment decisions affect patients. The investigator-led MOTIVATE study (NCT04023019; EudraCT No. 2019-003427-38) is recruiting…

A person in Germany with severe hemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), the first time the gene therapy has been used commercially in Europe, according to BioMarin Pharmaceutical, its developer. “Today represents an important milestone for the hemophilia community and for patients and…

Patients with moderate to severe hemophilia A who switched to Kovaltry (octocog alfa) showed no worsening of their annual bleeding rate, according to real-world data from a Phase 4 study. Adhering to the therapy remained high and patients reported high treatment satisfaction. Overall, the study “confirms and extends clinical trial…

The U.S. Food and Drug Administration (FDA) has given orphan drug and rare pediatric disease designations to Sernova’s experimental and cell-based hemophilia A treatment program using the Cell Pouch System, the company’s novel medical device. Orphan drug status is awarded to therapies aiming to treat…

The U.S. Food and Drug Administration (FDA) has approved Hemlibra (emicizumab-kxwh) for routine prophylaxis to prevent or lessen bleeding episodes in children and adults with hemophilia A with factor VIII inhibitors, Genentech announced. Hemlibra is first newly approved treatment for this patient group in almost 20 years, the…

Hemophilia A patients who receive replacement therapies of factor VIII often develop inhibitors against factor concentrates which hinders the effectiveness of the therapy, increases disability, and reduces quality of life. Although immune tolerance induction (ITI) can reduce inhibitors against factor VIII concentrates, the study “Long-term course of…

Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…

A new, modified version of recombinant factor VIII (rFVIII), called rVIII-SingleChain, showed great efficacy and safety in treating bleeding events in children with severe hemophilia A. The study, “Safety, efficacy and pharmacokinetics of rVIII-SingleChain in children with severe hemophilia A: results of a multicenter clinical trial,” was published in…

Children and adults with hemophilia A in the United States may soon be treated with N8-GP (turoctocog alfa pegol), an engineered formulation of clotting factor VIII developed by Novo Nordisk. The therapy will be available under the brand name Esperoct. The U.S. Food and Drug Administration (FDA) approved N8-GP, or…

Swedish Orphan Biovitrum AB recently announced that Eloctate (efmoroctocog alfa; brand name Elocta in Europe), a treatment for hemophilia A, has been approved for reimbursement in the United Kingdom, Italy, and France. These countries join others in the European Union — Germany, Sweden, Denmark, Norway, Switzerland, the Netherlands,…