Celgene, Acceleron bag Reblozyl FDA approval for anemia in beta thalassemia

Reblozyl FDA approval : Celgene and Acceleron Pharma have bagged approval from the US Food and Drug Administration (FDA) for Reblozyl (luspatercept-aamt) for the treatment of anemia associated with beta thalassemia in adults who need regular red blood cell (RBC) transfusions.

Reblozyl is now the first and only erythroid maturation agent to be approved by the FDA. The latest anemia drug represents a new class of therapy which helps in controlling late-stage red blood cell maturation to help patients cut down their RBC transfusion burden.

The erythroid maturation agent, which has been jointly developed by Celgene and Acceleron Pharma, has not been approved for use as an alternative for RBC transfusions in patients who need immediate correction of anemia.

Considered to be a rare, inherited blood disorder, Beta thalassemia, which is also called Cooley’s anemia, is caused by a genetic defect in hemoglobin. This genetic blood disorder is associated with ineffective erythropoiesis, which leads to the production of fewer and less healthy RBCs, which in turn mostly leads to severe anemia.

Celgene, Acceleron secure Reblozyl FDA approval for anemia in beta thalassemia. Image courtesy of Celgene Corporation/Acceleron Pharma Inc.

Anemia associated with beta thalassemia has limited treatment options, of which most of them are RBC transfusions. Treatment through RBC transfusions could potentially result in iron overload, thereby causing organ damage and other serious complications.

Richard Pazdur – director of the FDA Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA Center for Drug Evaluation and Research, commenting on Reblozyl FDA approval, said: “When patients receive multiple blood transfusions, there is a risk for iron overload, which can affect many organs.

“Today’s approval provides patients with a therapy that, for the first time, will help decrease the number of blood transfusions. This approval is an example of our continued progress for rare diseases and providing important new drugs to patients earlier.”

Reblozyl FDA approval driven by BELIEVE clinical trial results

Reblozyl FDA approval has been driven by the results of the phase 3 BELIEVE clinical trial held in 336 adult patients for assessing the safety and efficacy of the luspatercept for the treatment of anemia associated with beta thalassemia. The participants were those who need regular RBC transfusions because of their genetic blood disorder.

The BELIEVE clinical trial compared Reblozyl plus best supportive care (BSC) against placebo. The late-stage trial achieved a clinically meaningful and statistically significant improvement in the primary endpoint, while also meeting key secondary endpoints like transfusion burden reduction of at least 33%.

Commenting on Reblozyl FDA approval, Nadim Ahmed – President of Celgene Global Hematology and Oncology, said: “There are very limited options for patients living with anemia due to beta thalassemia who are dependent on long term red blood cell transfusions. We are pleased to make Reblozyl available as a new therapy for these patients to help address their anemia, a significant clinical complication of beta thalassemia.”

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