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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PI3K inhibitors could treat HHT

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Lab mouse
Preclinical research suggests PI3K inhibitors could treat hereditary hemorrhagic telangiectasia (HHT). Experiments in mice and patient samples revealed that loss of ALK1 function induces vascular hyperplasia and increases activity of the PI3K pathway. Pharmacological inhibition of PI3K was able to eliminate vascular hyperplasia in mouse models. Francesc Viñals, PhD, of Institut Catala d’Oncologia... [Read Article]
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Fostamatinib produces responses in ITP

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Fostamatinib (Tavalisse™)
Photo courtesy of
Rigel Pharmaceuticals
Fostamatinib has produced “clinically meaningful” responses in adults with persistent or chronic immune thrombocytopenia (ITP), according to researchers. In a pair of phase 3 trials, 18% of patients who received fostamatinib had a stable response, which was defined as having a platelet count of at least 50,000/µL... [Read Article]
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