Variant not associated with CLL, AIHA, or ITP in certain patients

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DNA helix
Image by Spencer Phillips
New research suggests there is no association between the PTPN22 R620W polymorphism and chronic lymphocytic leukemia (CLL) or autoimmune hematologic disorders in patients from the Republic of Macedonia. Past studies have shown an association between the PTPN22 R620W variant and both CLL and autoimmune diseases in patients from... [Read Article]
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Emicizumab now also approved for hemophilia A without inhibitors

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Emicizumab (Hemlibra)
Photo from Business Wire
The U.S. Food and Drug Administration (FDA) approved emicizumab-kxwh (Hemlibra) for prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients, including newborns, with hemophilia A with or without factor VIII (FVIII) inhibitors. It was first approved in 2017 for hemophilia A patients with FVIII... [Read Article]
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CHMP recommends factor VIII therapy for hemophilia A

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U.S. version of damoctocog alfa pegol (Jivi®)
Photo from Bayer
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval for damoctocog alfa pegol, a recombinant human factor VIII therapy. Bayer is seeking European marketing authorization for damoctocog alfa pegol (formerly BAY94-9027), for the treatment and prophylaxis of... [Read Article]
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Caplacizumab approved to treat aTTP

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Vials and a syringe
The European Commission has granted marketing authorization for caplacizumab (Cablivi™), a humanized bivalent nanobody that inhibits the interaction between von Willebrand factor and platelets. Caplacizumab is now approved to treat adults with acquired thrombotic thrombocytopenic purpura (aTTP) in all member countries of the European Union as well as Norway, Iceland, and... [Read Article]
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Factor VIII product approved for hemophilia A

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Antihemophilic factor
(recombinant)
PEGylated-aucl (Jivi®)
Photo from Bayer
The US Food and Drug Administration (FDA) has approved Jivi® (antihemophilic factor [recombinant] PEGylated-aucl) for the treatment of hemophilia A. Jivi (formerly BAY94-9027) is a DNA-derived, factor VIII concentrate approved for use in previously treated adults and adolescents (age 12 and older) with hemophilia A. The product... [Read Article]
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A new standard of care in hemophilia A?

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Emicizumab (Hemlibra)
Photo from Business Wire
Results of a phase 3 trial showed that prophylaxis with emicizumab significantly reduced bleeds, compared to no prophylaxis, in patients with hemophilia A without inhibitors. Emicizumab also reduced bleeds when compared to prior factor VIII prophylaxis. The most common adverse events (AEs) in this trial were injection site reactions,... [Read Article]
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FDA grants priority review to drug for PNH

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Red blood cells
The US Food and Drug Administration (FDA) has accepted for priority review the biologics license application (BLA) for ALXN1210, a long-acting C5 complement inhibitor. With this BLA, Alexion Pharmaceuticals, Inc., is seeking approval for ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). The FDA grants priority review to applications... [Read Article]
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Marzeptacog alfa may prevent bleeds in hemophilia A/B with inhibitors

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Syringe in vial
Photo courtesy of
Catalyst Biosciences
The activated factor VIIa variant marzeptacog alfa has demonstrated efficacy as prophylaxis for patients with hemophilia A or B who also have inhibitors, according to researchers. Three patients have completed dosing with marzeptacog alfa in a phase 2/3 study. None of these patients experienced bleeding during treatment,... [Read Article]
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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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