According to a story from BioSpace, Acceleron Pharma Inc. and Bristol Myers Squibb have announced recently that the US Food and Drug Administration (FDA) has recently approved a new drug called luspatercept-aamt (marketed as Reblozyl®). It will be used to treat anemia in patients with low to moderate risk myelodysplastic syndromes (MDS) that have failed to respond to erythropoiesis stimulating agents and require at least red blood cell units over a period of eight weeks.
About Myelodysplastic Syndromes (MDS)
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
Reblozyl is categorized as an erythroid maturation agent and it is the first medication of this kind to gain approval for use. The drug is designed for patients whose disease presents with ring sideroblasts. These are a certain type of cell that is produced by the bone marrow instead of healthy red blood cells. The ultimate goal of treatment with Reblozyl is to restore the patient’s ability to produce red blood cells and no longer have to rely on transfusions of blood, which in the long term poses several risks for patients, such as infections, site reactions, and iron overload.
This drug was first approved in November 2019 as a treatment for anemia associated with beta thalassemia, a rare blood disorder. The approve of Reblozyl for anemia associated with myelodysplastic syndromes follows the results from a phase 3 trial in which the therapy was able to allow a significantly greater number of patients to be transfusion-free for a period of eight weeks during the first 24 weeks of the study. The drug also had a comparable safety profile when compared to placebo.
This approval will give patients with low to moderate risk myelodysplastic syndromes are chance to resolve anemia and experience a greater quality of life.
