FDA OK039s First Drug for Cooley039s Anemia



WASHINGTON -- The FDA approved luspatercept (Reblozyl) on Friday to reduce the frequency of blood transfusions for adult patients with anemia related to beta thalassemia -- so-called Cooley's anemia.

"When patients receive multiple blood transfusions, there is a risk for iron overload, which can affect many organs," Richard Pazdur, MD, director of the FDA's Oncology Center of Excellence and acting director of the newly named Office of Oncologic Diseases, said in a statement. "Today's approval provides patients with a therapy that, for the first time, will help decrease the number of blood transfusions."

According to Acceleron and Celgene, the drug's manufacturers, luspatercept is a first-in-class erythroid maturation agent designed to repair a defect in late-stage red blood cell (RBC) maturation. Beta thalassemia is associated with ineffective erythropoiesis, which results in less RBC production, and often leads to severe anemia in patients. Often, lifelong RBC transfusions are required to survive, and subsequently patients need treatment for iron overload and have increased risk for abnormal blood clots.

Data supporting the approval came from the phase III BELIEVE trial, which randomized 336 adult beta thalassemia patients with anemia that required six to 20 RBC units every 24 weeks to either luspatercept or placebo.

The trial met its primary endpoint, significantly decreasing transfusion need in the investigational arm during weeks 13 to 24. In the luspatercept group, 21.4% of patients achieved at least a 33% reduction in their transfusion burden from baseline, as compared with 4.5% of patients in the placebo group (P<0.0001).

Luspatercept also reduced transfusion need in weeks 37 to 48, a key secondary endpoint. Here, 19.6% of patients in the investigational arm had at least a 33% reduction in their transfusion need versus 3.6% in the placebo arm (P<0.0001).

Serious adverse events (AEs) occurred in 3.6% of patients on the study drug and 5.4% had to discontinue treatment for toxicity. AEs that were more common with the luspatercept than placebo groups included headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea, and dizziness.

FDA warned against use in pregnant women due to the risk of fetal harm and also advised healthcare professionals to monitor blood pressure -- about 11% of patients taking the drug on trials experienced this adverse reaction -- and to use antihypertensives if necessary. Patients should also be monitored for blood clots -- 3.6% of patients on luspatercept in the BELIEVE trial experienced thromboembolic events.

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