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Celgene-Acceleron's blood disorder drug gets US FDA priority review


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Celgene-Acceleron's blood disorder drug gets US FDA priority review

The U.S. Food and Drug Administration granted priority review to Celgene Corp. and Acceleron Pharma Inc.'s experimental drug luspatercept, which can help treat certain types of blood disorders.

The companies said the drug was granted priority review to treat patients with very low to intermediate-risk myelodysplastic syndromes, or MDS,-associated anemia who have ring sideroblasts, or immature red blood cells, who require red blood cell transfusions.

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MDS, which is considered a type of cancer, is a group of disorders in which blood cells in the bone marrow fail to mature, making patients prone to infections, anemia and spontaneous bleeding, among other things.

Luspatercept works by helping regulate late-stage red blood cell maturation.

The FDA is expected to make a decision by April 4, 2020, for this indication.

Luspatercept was also granted priority review to treat adult patients with beta-thalassemia-associated anemia who require red blood cell transfusions. Beta thalassemia is a blood disorder that reduces the production of hemoglobin — the iron-containing protein in red blood cells that carries oxygen to the cells in the body.

The FDA is likely to decide by Dec. 4 whether to approve luspatercept for that indication.

The companies said the biologics license application was backed by positive data from studies that showed that luspatercept helped treat anemia associated with MDS and beta-thalassemia.

In addition, Celgene said its marketing authorization application in the EU for luspatercept had been validated and the review was underway.

Summit, N.J.-based Celgene and Cambridge, Mass.-based Acceleron are jointly developing luspatercept as part of a global collaboration. The drug has not been approved anywhere.