SEC Armadillo in Glasgow; Photo by Guy Percival

The World Federation of Hemophilia (WFH) 2018 World Congress took place May 20-24 in Glasgow, Scotland.

Perioperative rVWF alone sufficient for some VWD patients

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rVWF (Vonvendi)
Photo from Shire
Recombinant von Willebrand factor (rVWF) alone can be sufficient as perioperative management for some patients with severe von Willebrand disease (VWD), according to researchers. In a phase 3 study, 10 of 15 patients were able to achieve hemostatic efficacy ratings of “good” or “excellent” when receiving only rVWF before,... [Read Article]
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Emicizumab controls bleeding regardless of inhibitors

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Vials of drug
Photo by Bill Branson
Emicizumab prophylaxis provides “clinically meaningful” control of bleeding whether or not patients have factor VIII inhibitors, according to researchers. In the phase 3 HAVEN 4 study, researchers evaluated emicizumab prophylaxis, given every 4 weeks, in hemophilia A patients with or without factor VIII inhibitors. At a median... [Read Article]
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Gene therapy reduces ABR, AIR in hemophilia B

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DNA helix
Image by Spencer Phillips
New research suggests the gene therapy SPK-9001 can reduce bleeding and the need for factor IX infusions in patients with hemophilia B. In an ongoing, phase 1/2 trial, SPK-9001 reduced the annualized bleeding rate (ABR) by 98% and the annualized infusion rate (AIR) by 99%. All 15 patients... [Read Article]
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N9-GP has better PK profile than rFIXFc, team says

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N9-GP (Rebinyn)
Photo from Novo Nordisk
Nonacog beta pegol (N9-GP) has a better pharmacokinetic (PK) profile than recombinant factor IX-Fc fusion protein (rFIXFc), according to researchers. In a phase 1 trial, adults with hemophilia B who received a single dose of N9-GP achieved greater total factor IX exposure than those treated with rFIXFc, and... [Read Article]
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Therapy can extend half-life of FVIII

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Antihemophilic factor
Preliminary data suggest an investigational therapy can extend the half-life of factor VIII (FVIII) in patients with severe hemophilia A. Researchers are testing the therapy, BIVV001, in a phase 1/2a trial and have reported results in 4 patients. BIVV001 extended the half-life of FVIII to 37 hours, with an average FVIII activity... [Read Article]
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Emicizumab reduces bleeding in hemophilia A

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Emicizumab (Hemlibra)
Photo from Business Wire
Final results from the HAVEN 3 study suggest emicizumab prophylaxis can reduce bleeding in hemophilia A patients without factor VIII inhibitors. Compared to patients who did not receive prophylaxis, those who received emicizumab prophylaxis had a 96% to 97% reduction in treated bleeds and a 94% to 95%... [Read Article]
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