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FDA Approves Celegene's Reblozyl To Treat An Inherited Blood Disorder

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The U.S. Food and Drug Administration approved the first therapy for patients with a rare inherited blood disorder called beta thalassemia. Celegene's Reblozyl (luspatercept-aamt) is also the first and only FDA-approved erythroid maturation agent, a new class of therapy for such patients.

The regulator approved Reblozyl for treating anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. Reblozyl is expected to be available one week after the approval.

Beta thalassemia, also called Cooley's anemia, is an inherited blood disorder that reduces the hemoglobin production, an iron-containing protein in red blood cells that carries oxygen to cells in the body.

The low levels of hemoglobin leads to lack of oxygen in many parts of the body and anemia, which can cause pale skin, weakness, fatigue and more serious complications.

The disorder is often treated with lifelong regimens of chronic blood transfusions and treatment for iron overload due to the transfusions. This can cause serious complications such as organ damage Patients with this disorder are also at an increased risk of developing abnormal blood clots.

Reblozyl had received a Priority Review designation from the FDA.

The clinical trial was conducted on 336 patients with beta thalassemia who required RBC transfusions, of which 112 received a placebo. Twenty-one percent of the patients who received Reblozyl over 12 consecutive weeks achieved at least a 33% reduction in transfusions compared to 4.5% of the patients who received a placebo.

Celgene and Acceleron Pharma jointly developed Reblozyl. After receiving Fast Track designation, Reblozyl also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

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