US FDA approves rare blood disorder drug

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The U.S. Food and Drug Administration said on Friday it had approved a treatment for a rare blood disorder from Celgene Corp, which is in the process of being bought by Bristol-Myers Squibb Co.

Reblozyl, which has been jointly developed by Celgene and Acceleron Pharma Inc, is meant for treatment of anemia in patients with beta thalassemia, who require regular red blood cell transfusions.

Beta thalassemia is an inherited disorder that reduces the production of hemoglobin in blood, which can lead to lack of oxygen in many parts of the body and anemia, the FDA said.

The approval is a shot in the arm for Celgene as its flagship multiple myeloma drug, Revlimid awaits loss of exclusivity starting 2022.

Treatment for people with beta thalassemia often consists of lifelong regimens of chronic blood transfusions for survival and treatment for iron overload due to the transfusions.

“Today’s approval provides patients with a therapy that, for the first time, will help decrease the number of blood transfusions”, FDA’s Richard Pazdur said in a statement. Celgene said the drug is expected to be available a week following the approval.