FDA approves Reblozyl (luspatercept-aamt) for treatment of adult patients with thalassemia

recently, The u.SFood andDrug(http://Authority (
) of the Food andMedicineshttp://
(
) has approved Reblozyl (luspatercept-aamt) for adult patients with beta-thalassemia who require regular red blood cell infusions,announced by Celgene and acceleronpharmaceutical http://(http://
)AboutReblozyl is the first FDA-approved drug to treat beta thalassemia-related anemia, and the first and only DA-approved red blood cell maturation agent, representing a new class of therapy that helps patients reduce the burden of red blood cell infusionby by regulating the later stages of red blood cell maturationReblozyl is not suitable as an alternative to red blood cell infusions in patients who need to correct anemia immediatelyReblozyl's treatment of beta thalassemia indications is based on data from the key PHASE III BELIEVE studyThis is a randomized, double-blind, placebo-controlled, multicenter study carried out in patients with blood transfusion-dependent beta thalassemiathe study reached the primary and all key secondary endpointsThe results showed that patients in the luspatercept treatment group had significantly less blood transfusion burden than the placebo groupReblozyl's active pharmaceutical ingredient is luspatercept, a first-of-the-first red blood cell maturant (EMA) that regulates the maturation of late red blood cellsReblozyl 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, which regulates the specific ligand of late RBC maturation conversion growth factor (TGF)-beta superfamily, reduces the activation of smad2/3 signaling pathways, improves the activation of invalid red blood cell production, and promotes the level of late red blood cellLuspatercept is developed globally by Shinji in partnership with Acceleron PharmaceuticalsCurrently, both parties are evaluating the potential of luspatercept to treat primary treatment of erythrocytosis (ESA), low-risk MDS patients (Phase III COMMANDS study) and non-transfusion beta-thalassemia (Phase II BEYOND study) and bone marrow fibrosis