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…
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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…
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 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…
Labor and delivery in women with hemophilia Planning for childbirth depends on the needs of the mother and the child affected by hemophilia. Levels of clotting factors should be measured in the last trimester of pregnancy, since it is difficult to measure during labor. If levels are low, treatment may…
The Phase 1/2 trial looking at the Factor VIII protein SHP656 as a long-acting therapeutic to treat hemophilia A did not meet a pre-defined once-weekly dosing criterion, according to Xenetic Biosciences and Shire, which are jointly developing the drug. The study demonstrated SHP656’s efficacy and pharmacokinetic data (the drug’s behavior once…
Sangamo Therapeutics and Pfizer have entered a global collaboration and license agreement to develop and market gene therapy programs for hemophilia A, including SB-525. SB-525 was cleared by the U.S. Food and Drug Administration (FDA) in January as an investigational new drug (IND) for the…
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Explaining Hemophilia A and B
https://www.youtube.com/watch?v=S_YMGmHqIW4 This video from Claire Blatt shares a lecture about the effects of two blood-clotting disorders: hemophilia A and hemophilia B. MORE: A study shows that NovoSeven counters bleeding in children with hemophilia. Though the lecture is aimed at nurses, it’s helpful for anyone looking to learn about these…