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    EMA Consults on Revised Factor VIII Product Guidelines

    The European Medicines Agency (EMA) on Monday issued two draft revised guidelines on plasma-derived and recombinant coagulation factor VIII products that remove requirements for conducting clinical studies in previously untreated patients (PUPs). Background Factor VIII products are used to treat or prevent bleeding in patients with hemophilia A (congenital factor VIII deficiency). The development of inhibitor antibodies is one of the greatest potential complications inv...
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    EMA Finds No Difference in Inhibitor Risk Between Factor VIII Classes

    The European Medicines Agency (EMA) on Friday said it could find "no clear and consistent" evidence of a difference in the risk of inhibitor development between recombinant and plasma-derived factor VIII medicines. The announcement reconfirms EMA's conclusion from two earlier reviews of factor VIII medicines in 2013 and 2016 . The development of inhibitor antibodies is one of the greatest potential complications involved with treating patients with hemophilia A. When...
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    CBER Director Offers a Peek Into the Complexities of Where the Center is Headed

    In addition to protecting the US blood supply and ensuring vaccines are safe and effective, the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) is also now regulating complex gene and cell therapies, as well as a new category of treatments: regenerative medicines. Peter Marks, director of CBER, offered attendees of DIA’s annual conference on Tuesday a few examples of the changing landscape for the center, noting that with...
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    European Regulatory Roundup: EMA to Revise Trial Guidance on Hemophilia, Crohn’s, Ulcerative Colitis, Diabetes and Bipolar Disorder (4 August 2016)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. Boom in Hemophilia R&D Prompts EMA to act to Prevent Enrollment Bottleneck The European Medicines Agency (EMA) is planning to revise its guideline on development of drugs to treat hemophilia to cut the risk of patient enrollment bottlenecks slowing progress. EMA began the process after becoming concerned that the minimum data requirements of its current guideline are i...
  • EMA Calls for Further Research on Personalized Approaches to Treating Hemophilia

    The European Medicines Agency (EMA) has released a new reflection paper on the risks of adverse immune system reactions in hemophilia patients treated with Factor VIII products and how these factors might be mitigated in the future. EMA explains in its 31 July reflection paper that up to 30% of patients with severe hemophilia A experience a buildup of neutralizing antibodies against the treatment, which can cause bleeding. Current therapies tend to focus on treating th...