Hematology

The U.S. Food and Drug Administration has approved mepolizumab (Nucala; GlaxoSmithKline) for the treatment of patients with hypereosinophilic syndrome (HES) for ≥ 6 months without an identifiable non-hematologic secondary cause. This is the first targeted biologic treatment for patients with this eosinophil-driven disease in the United States.

Approval was based on data from a pivotal phase 3 study which showed 50% fewer patients experienced a HES flare when mepolizumab was added to standard of care treatment, compared to placebo, over the 32-…

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Take a look at some of our conference coverage, visit our roundup section.

Conference Roundup

  1. Jun 25, 2020

First patients with sickle cell disease treated with experimental gene-editing cell therapy remains VOC-free after 9 months

The first patients with severe sickle cell disease (SCD) treated with an experimental gene-editing cell therapy is free of vaso-occlusive crises (VOCs) 9 months after treatment, according to data from Phase 1/2 clinical trial presented at the European Hematology Association (…

Journal Scan

  1. Sep 10, 2020

BIVV001 injection effective in severe hemophilia A

In the phase 1-2a open-label trial, 16 men with severe hemophilia A were treated with a single intravenous injection of recombinant factor VIII with a dose of 25 IU per kilogram of body weight or 65 IU per kilogram. A washout period of at least 3 days followed the…