Anemic new drug! Reblozyl, the first red blood cell maturation agent of BMS, has been awarded the third orphan drug qualification by the US FDA to treat bone marrow fibrosis!

January 18, 2020 / BIOON / -- reblazyl (lupatercept), a red blood cell ripening agent developed by BMS in cooperation with accelerator pharmaceutical company, has recently been awarded the orphan drug qualification (odd) by the US Food and Drug Administration (FDA) to treat myelofibrosis It is worth mentioning that this is also the third odd obtained by FDA Previously, the FDA has awarded lupatercept for the treatment of β - thalassemia and myelodysplastic syndrome (MDS) of odd Orphan drug (Orphan Drug) is a kind of medicine used for prevention, treatment and diagnosis of rare diseases Rare diseases are a general term for diseases with very low incidence rate, also known as orphan diseases In the United States, rare diseases refer to the types of diseases with a population of less than 200000 Incentives for drug research and development of rare diseases include various incentives for clinical development, such as tax credits related to clinical trial costs, FDA user fee relief, FDA assistance in clinical trial design, and a 7-year market monopoly period for the approved indications after the drug is launched Reblozyl is a kind of red blood cell maturation agent, which was approved by FDA in November 2019 It is used to treat anemia in adult patients with β thalassemia who need to infuse red blood cells regularly It is worth mentioning that reblazyl is the first drug approved by FDA to treat β thalassemia related anemia, and also the first red blood cell maturation agent approved by Da, which represents a new type of therapy, helping patients reduce the burden of red blood cell infusion by regulating the late stage of red blood cell maturation It should be noted that reblozyl is not suitable as a substitute for red blood cell transfusion in patients who need immediate correction of anemia At present, the biological product license application (BLA) of reblazyl for the treatment of MDS anemia is under the review of FDA, which applies for the approval of reblazyl for bone marrow smear with ring Sideroblast, RS +, RBC and MDS patients with very low to medium risk are required to treat anemia The target date of PDUFA is April 4, 2020 In Europe, the application for marketing authorization (MAA) of reblozyl for the treatment of β - thalassemia and MDS anemia in adults is being reviewed by the European Drug Administration (EMA) The active component of reblozyl is luspatercept, which is the first in class erythrocyte maturation agent (EMA), which can regulate the maturation of advanced erythrocytes The drug is a soluble fusion protein, which is formed by the fusion of Fc domain of human IgG1 and extracellular domain of activin IIB receptor (actriib) As a ligand trap, it can regulate the specific ligand of transforming growth factor - β superfamily of late RBC maturation through targeted binding, reduce the activation of Smad2 / 3 signal pathway, improve the generation of invalid erythrocytes, and promote the late erythropoiesis Cell maturation, improve hemoglobin level Mechanism of luspatercept: luspatercept is developed globally by Xinji (which has been acquired by BMS) and accelerator pharmaceutical company At present, both sides are also evaluating the potential of luspatercept in the treatment of erythropoietic stimulants (ESA) in the initial treatment, low-risk MDS patients (phase III commands study), non transfusion β thalassemia (phase II beyond study) and myelofibrosis The industry is also very optimistic about the business prospects of luspatercept At the end of last year, evaluatepharma released the vantage 2019 preview report, which included 20 of the most valuable R & D projects in the world Luspatercept ranked 18th with a net present value of US $3.1 billion Earlier, Jefferies analysts, a famous investment bank on Wall Street, pointed out that if the anemia of myelodysplastic syndrome (MDS) was also approved by the FDA, the annual sales peak of reblazyl would reach $2 billion Reference source: FDA orphan drug public database, Bristol Myers Squibb