On February 4, Bristol Myers Squibb China announced that its application for the listing of its red blood cell maturation agent luspatercept (English trade name: rebrozyl; self-made Chinese common name: rotxep for injection; self-made Chinese trade name: libloze) has been officially accepted by the drug evaluation center (CDE) of the State Drug Administration, and has been granted the priority qualification for evaluation, which is used to require regular transfusion of red blood cells（ RBC) in adult patients with beta thalassemia.
β - thalassemia is a kind of hereditary hematopathy characterized by anemia, which is caused by the decrease or deficiency of β - globin peptide chain synthesis. Severe β - thalassemia is a rare disease with chronic progressive hemolytic anemia. At present, patients rely on life-long blood transfusion, iron removal therapy or hematopoietic stem cell transplantation to maintain life.
As the first recombinant fusion protein drug in the world, luspatercept improves hemoglobin level by promoting the maturation of advanced red blood cells.
According to a global phase III clinical study named believe based on which luspatercept was included in the priority review, 21.4% of patients after treatment with luspatercept had a blood transfusion burden reduction of more than 33% compared with the baseline, which was significantly better than that in the placebo group (4.5%), and the iron overload was significantly reduced, which may improve the quality of life of patients.