Health Canada approves emicizumab

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Emicizumab (Hemlibra)
Photo from Business Wire
Health Canada has approved emicizumab (Hemlibra®) for use as routine prophylaxis to prevent or reduce bleeding episodes in hemophilia A patients with factor VIII inhibitors. Emicizumab is a bispecific factor IXa- and factor X-directed antibody. It bridges activated factor IX and factor X to restore the natural function of... [Read Article]
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FA pathway key to DNA repair after CRISPR cutting

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DNA repair
Image by Tom Ellenberger
New research suggests the Fanconi anemia (FA) pathway plays a key role in repairing double-strand breaks (DSBs) created by CRISPR-Cas9 genome editing. Researchers said they found that Cas9-induced single-strand template repair requires the FA pathway, and the protein FANCD2 localizes to Cas9-induced DSBs. The team said this research provides... [Read Article]
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FDA releases guidance docs on gene therapy

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DNA helix
Image by Spencer Phillips
The US Food and Drug Administration (FDA) has released several draft guidance documents on gene therapy. Three are disease-specific guidances—for hemophilia, rare diseases, and retinal disorders—and 3 are guidances on manufacturing gene therapies. These 6 documents are intended to serve as the building blocks of a modern, comprehensive framework... [Read Article]
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CHMP recommends rVWF for VWD

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US version of rVWF (Vonvendi)
Photo from Shire
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for vonicog alfa (VEYVONDI), a recombinant von Willebrand factor (rVWF) product. The CHMP is recommending vonicog alfa for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age... [Read Article]
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CHMP backs approval of caplacizumab

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Vials of drug
Photo by Bill Branson
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of caplacizumab (Cablivi) for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP). Caplacizumab is a humanized bivalent nanobody that inhibits the interaction between von Willebrand factor and platelets. The CHMP’s... [Read Article]
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Survey reveals patient perceptions of ITP

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Poster session at the 23rd
Congress of the European
Hematology Association (EHA)
Photo from EHA
A new survey has revealed patients’ perceptions of immune thrombocytopenia (ITP) and how the condition impacts their quality of life (QOL). Patients reported delays in diagnosis, lack of support, severe fatigue, and impacts on both emotional well-being and their ability... [Read Article]
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Drug is convenient alternative for PNH, doc says

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Session at the 23rd
Congress of the European
Hematology Association (EHA)
Photo from EHA
Results of a phase 3 study suggest the long-acting C5 complement inhibitor ravulizumab produces similar results as eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH). Treatment with ravulizumab every 8 weeks proved noninferior to treatment with eculizumab every 2 weeks... [Read Article]
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Emicizumab granted priority review for hemophilia A without inhibitors

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Bleeding human finger
By Crystal (Crystl) from Bloomington, USA - Flickr
The US Food and Drug Administration (FDA) has granted priority review for emicizumab (Hemlibra®) for adults and children with hemophilia A without factor VIII inhibitors. Earlier this year, the agency awarded emicizumab breakthrough therapy designation for the same population. Emicizumab is a bispecific factor... [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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Single injection could treat hemophilia B long-term

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Induced pluripotent stem cells
Image by James Thomson
Cell therapy could produce lasting effects in hemophilia B, according to a group of researchers. They genetically modified induced pluripotent cells (iPSCs) derived from patients with hemophilia B and converted those iPSCs into hepatocyte-like cells (HLCs). When transplanted into mouse models of hemophilia B, the... [Read Article]
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