U.S. FDA Approves Celgene's Rare Blood Disorder Drug

By Reuters Staff

November 12, 2019

(Reuters) - 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 (luspatercept–aamt), 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.

The approval of Reblozyl was based on the results of a clinical trial of 336 patients with beta thalassemia who required RBC transfusions, of which 112 received a placebo.

Twenty-one percent of the patients who received Reblozyl achieved at least a 33% reduction in transfusions compared to 4.5% of the patients who received a placebo. The transfusion reduction meant that the patient needed fewer transfusions over 12 consecutive weeks while taking Reblozyl.

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. http://bit.ly/2JZP7Mj