News

Shire has asked the U.S. Food and Drug Administration to grant Investigational New Drug status to its hemophilia A treatment SHP654. The gene therapy helps generate a clotting protein known as factor VIII that is missing or defective in hemophilia A. Shire said an FDA approval of its request will…

Organizations championing patients with hemophilia and other bleeding disorders have formed a united front to oppose the Better Care Reconciliation Act, the Obamacare replacement legislation that the groups contend will damage the hemophilia community. The National Hemophilia Foundation, Hemophilia Federation of America, Coalition for Hemophilia B and…

European regulators have granted orphan drug status to CB 2679d, a hemophilia B therapy that Catalyst Biosciences developed to prevent bleeding episodes. The therapy, known as ISU304 in South Korea, prevents bleeding by increasing hemophilia B patients’ level of a blood protein called Factor IX, which is deficient in the disease.

Results from two Phase 3 trials support the use of emicizumab for the preventive treatment of hemophilia A in children, adolescents, and adults, Genentech announced. These results will be presented at the 26th International Society on Thrombosis and Haemostasis (ISTH) Meeting July 8-13 in Berlin, Germany. Emicizumab is an…

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…

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…

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…

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…