A hemophilia patient has died in a Phase 3 trial evaluating Roche’s  experimental treatment emicizumab (ACE910), raising further concern about its safety. Preliminary indications are that the patient died of a rectal hemorrhage and not emicizumab therapy, however, the company said. The death follows reports of serious blood-clotting events in…

An analysis was highly critical of the Orphan Drug Act, used by the U.S. Food and Drug Administration since 1983 to stimulate research into rare diseases. The study found that the act is not “sufficiently” effective in meeting the needs of patients with less common diseases, like hemophilia, while pharmaceutical companies are finding it profitable,…

Sangamo Therapeutics recently presented what it characterized as key improvements to its technology platform for engineering highly specific zinc finger nucleases (ZFNs), which are used for targeted editing of the human genome. This technique has broad applicability, namely for the treatment of hemophilia B and other disorders. The…

The U.S. Food and Drug Administration (FDA) has approved Shire’s Adynovate for the treatment of pediatric patients under 12 years of age with hemophilia A. Adynovate also was approved for use by both adults and children undergoing surgical procedures, to control bleeding episodes. Adynovate is a recombinant Factor…

Biogen recently hired two more executive management team members for Bioverativ, a Biogen spin-off company that is expected to be up and running in early 2017. Bioverativ will focus on the discovery, research, development, and commercialization of new treatments for hemophilia and other blood disorders. Rogerio Vivaldi, MD, is Bioverativ’s new executive…

NuFACTOR is accepting applications for the 2017 Annual Eric Dostie Memorial College Scholarship. NuFACTOR is a specialty pharmacy of FFF Enterprises, a distributor of plasma products, vaccines and biosimilars, among others. NuFACTOR specializes in the treatment of chronic conditions in several areas of medicine, including blood disorders and…

Genentech will present more than 60 abstracts at the 58th American Society of Hematology Annual Meeting (ASH 2016). Nine of Genentech’s approved or investigational medicines will be featured, including Gazyva (obinutuzumab), Rituxan (rituximab), Venclexta (venetoclax) and emicizumab, the company’s investigational drug in hemophilia. More specifically, Genentech will present…

Scientists have improved protein drugs using an approach based on “ancestral sequence reconstruction,” or ASR. The newly established strategy promises to enhance the ability to engineer more efficient and durable proteins, as in the case of human factor VIII for hemophilia. The study “Enhancing the pharmaceutical properties of protein drugs by…

Researchers reviewed the use of high-purity concentrates containing two plasma molecules that are lacking in patients with Von Willebrand disease (VWD), a hemorrhagic disorder. These products may be a good strategy for managing pediatric patients with VWD and hemophilia A (HA) who do not respond to other treatments. The study, “Human Von…

Researchers analyzed the patterns of bruising in children younger than six years old to understand the extent to which their inherited bleeding disorders, such as hemophilia, affected the number, size, and location of these bruises — and found that bruising in the youngest of these children may be an early…