Shire is developing a potential new strategy to improve the effectiveness of genetic delivery of clotting factor VIII (FVIII) for the treatment of patients with hemophilia A, and clotting factor IX (FIX) for patients with hemophilia B. The most recent findings on these new therapeutic strategies and the latest…
News
Spark Therapeutics will present novel results regarding the ongoing Phase 1/2 clinical trial (NCT02484092) investigating SPK-9001 in hemophilia B patients at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, to be held July 8-13 in Berlin. Two presentations focus on data from the trial: “SPK-9001:…
A new study suggests that whole body vibration training increases muscle strength, bone mineral density, and functional capacity in children with hemophilia. The study, “Effect of whole body vibration training on quadriceps strength, bone mineral density, and functional capacity in children with hemophilia: a randomized clinical trial,” was published…
An international team has joined efforts to establish guidelines for effectiveness and outcome measurements regarding gene therapies in hemophilia. The CoreHEM project will be led by researchers from McMaster University in Ontario, Canada, in collaboration with the National Hemophilia Foundation (NHF) in the U.S. and the Green Park…
News
FDA Accepts Bioverativ’s Investigational New Drug Application for BIVV001 to Treat Hemophilia A
The U.S. Food and Drug Administration (FDA) has accepted Bioverativ’s investigational new drug application (IND) for BIVV001, an investigational Factor VIII therapy designed to extend protection from bleeds in hemophilia A patients. The FDA will now review the IND application for safety. The Waltham, Massachusetts-based company plans to initiate a Phase…
The Hemophilia Federation of America (HFA) has been awarded a $250,000 research grant through the Eugene Washington PCORI Engagement Awards Program to support the HFA’s PRIDE Project through training and education of bleeding disorder community stakeholders, including the hemophilia community. The Awards Program is an initiative of the Patient-Centered…
The European Medicines Agency (EMA) has granted orphan medicinal product status to SB-525, a clinical stage gene therapy candidate under development for the treatment of hemophilia A, according to Sangamo Therapeutics and Pfizer. The announcement coincides with the opening of patient enrollment for a Phase 1/2…
Forty percent of men with hemophilia who responded to a survey said the condition can weigh on their sex life, although University of California researchers cautioned that the 20-person sample was very small. Some of the respondents said they can experience bleeding from sex. In addition, the joint stiffness and joint…
Decades ago most hemophilia patients died young. Advances in treatment have led to them living about as long as the general population, which means that for the first time, doctors are facing the challenge of caring for aging hemophiliacs. A key problem is that in the days before treatment breakthroughs,…
Novo Nordisk’s recombinant coagulation factor IX nonacog beta pegol (N9-GP), under the U.S. trade name Rebinyn, has been approved by the U.S. Food and Drug Administration (FDA) to treat adults and children with hemophilia B. The company expects to launch this new treatment in the United States early in 2018. The…
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