FDA Committee to Hold Review for Reblozyl as a Treatment for Myelodysplastic Syndromes

According to a story from BioPortfolio, Acceleron Pharma and the Bristol-Myers Squibb Company have announced recently that the US Food and Drug Administration (FDA) will review the supplemental Biologics License Application (BLA) from Bristol-Myers Squibb for its drug luspatercept-aamt (marketed as Reblozyl). This review will be conducted at the December 18th, 2019 meeting of the agency’s Oncologic Drugs Advisory Committee. This application is for the use of Reblozyl as a treatment for myelodysplastic syndromes (MDS).

About Myelodysplastic Syndromes

Myelodysplastic syndromes are a type of blood cancer in which developing blood cells remain immature and fail to transform into usable blood cells. Risk factors for this disease include exposure to radiation, chemotherapy, benzene, xylene, and Agent Orange. Family history is also a risk, as are certain genetic disorders such as Down syndrome. In an individual case, it is rare for the direct cause to be identified. Myelodysplastic syndromes rarely present with symptoms initially, but it can eventually present with anemia, neutropenia, thrombocytopenia, cell abnormalities, chromosome abnormalities, enlarged spleen and/or liver, easy bleeding and bruising, and infections. The disease also has the potential to evolve into acute myeloid leukemia. Treatment may include bone marrow transplant, stem cell transplant, blood products, and certain chemotherapy agents. Outcomes in this disease ranges widely and can depend on a number of factors. To learn more about myelodysplastic syndromes, click here.

About Reblozyl

The treatment is more specifically intended for patients that require blood transfusions as a result of anemia with ring sideroblasts. The FDA is expected to complete its review of Reblozyl on April 4th, 2020. The drug was recently approved in the US for beta thalassemia patients suffering from anemia that requires transfusions. Anemia often appears in myelodysplastic syndromes because of a deficiency of mature red blood cells. Reblozyl is currently being tested in clinical trials for myelofibrosis, non-transfusion dependent beta thalassemia, and myelodysplastic syndromes.

Current management approaches for anemia associated with myelodysplastic syndromes are limited and include transfusions and medications that can encourage the production of erythropoietin. Unfortunately, transfusions are a major disruption to quality of life and also increase the risk of infections and iron overload.



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