New Base and Acceleron Pharmaceuticals submit to FDA application for license to biologics for experimental red blood cell maturant luspatercept

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pharmaceutical(http://giants Newgene and Acceleron Pharmaceuticals(http://announced that they have submitted an experimental red blood cell maturation agent luspatercept to treat blood transfusion-dependent beta thalassemia and bone marrow hyperplasia syndrome (MDS) to the U.SFood andDrug(http://Administration (http://)the BLA application for approval of luspatercept for: (1) treatment is associated with very low-risk to medium-critical MDS (bone marrow smears present in ring iron granulocytes (ringroblast, RS)) and requires red blood cell infusion in anane adult patients who require beta thalassemia and requireregular red blood cell infusionabout theof luspaterceptLuspatercept is developed globally by Shinji in partnership with Acceleron PharmaceuticalsAccording to the data from the two studies, Shinji and Acceleron also plan to submit regulatory documents to the EU in the first half of 2019Luspatercept is a first-of-the-first red blood cell maturation agent (EMA) that regulates the maturation of late red blood cells luspatercept is a soluble fusion protein, which is fused by the fc domain of human IgG1 and the extracellular domain of activator IIB receptor (ActRIIB), as a ligand trap, and is a specific ligand that regulates late RBC maturation of late-stage RBC by binding, reducing the activation of Smad2/3 signaling pathways, and improving the level of red blood cell production 　　At present, New Base and Acceleron are also evaluating the potential of luspatercept to treat non-transfusion beta thalassemia and bone marrow fibrosis The industry is also very optimistic about the business prospects of luspatercept Late last year, Valuate Pharma released a report, Vantage 2019 Preview, which took stock of the world's 20 most valuable research and development projects, placing luspatercept in 18th place with a net present value (NPV) of $3.1 billion 　The study the BLA submission, based on data from two Phase III clinical studies, of which: THE BELIEVE study was conducted in patients with blood transfusion-dependent beta thalassemia, and the MEDALIST study was conducted in patients with very low to moderate MDS Both studies reached the primary and all key secondary endpoints results showed a significant lynautopsy burden in patients in the luspatercept treatment group compared to the placebo group Data from both studies were presented at the 2018 annual meeting of the American Society of Hematology (ASH)