Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively reduces bleeding rates in children younger than 12 with hemophilia type A, regardless of development of inhibitors (antibodies) against synthetic factor VIII (FVIII), data from two Phase 3 studies show. Hemlibra is a type of replacement therapy that mimics…

Treatment with Esperoct (turoctocog alfa pegol) effectively prevents bleeding during and after major surgery in patients with severe hemophilia A, final data from a Phase 3 trial show. The study, “…

Brazilian hemophilia A patients who are 19 or older had significantly higher median care costs per patient per year than younger patients, a study reported. The study also revealed an increase in median costs for patients with more severe disease — that is, those who have less than 1%…

Researchers reviewed the current hemophilia research that focuses on the development of improved longer-acting factor replacement therapies, particularly PEGylated products. The new clinical research advances lead the scientific community to look at such developments with optimism, but also some caution. The study, “Potential role of a new PEGylated recombinant…

A collaborative research study offers new insights into how a protein called furin influences blood clotting and found it functions differently in hemophilia A and B, findings that are promising for improving gene therapy for hemophilia A. The study, “Circumventing furin enhances factor VIII biological activity and ameliorates bleeding…

In hemophilia A, the most significant therapeutic complication is the development of antibodies against factor VIII (FVIII) that inhibit its coagulant activity and make the therapy ineffective. The plasma-derived porcine FVIII product represents a therapy with low levels of autoantibodies and successful management of bleeding when compared to human plasma-derived products.

Chugai Pharmaceutical filed a New Drug Application for the use of emicizumab as a potential treatment to prevent or decrease the occurrence of bleeding episodes in patients with hemophilia A with factor VIII inhibitors. The application is based on…

The levels of sclerostin, a protein that regulates bone formation, may be linked to disease severity and reflect bone alterations in children with hemophilia A, a study suggests. The study, “Serum Sclerostin Level and Bone Mineral Density in Pediatric Hemophilic Arthropathy,” was published in The Indian…

Hemlibra (emicizumab) was able to prevent excessive bleeding in patients with severe hemophilia A without inhibitors, according to results from a Phase 3 trial. Trial findings were published in the study, “Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors,” in The New England Journal of…

The U.S. Food and Drug Administration (FDA) is extending its review of BioMarin Pharmaceutical’s request to approve the gene therapy Roctavian (valoctocogene roxaparvovec) for adults with severe hemophilia A. A decision had been expected by the end of this month, but that date has now been…