The US Food and Drug Administration (FDA) has approved Reblozyl (luspatercept-aamt) for the treatment of anaemia in beta thalassemia in adults needing regular red blood cell (RBC) transfusions.
Reblozyl is an erythroid maturation agent, which was co-developed by Celgene and Acceleron Pharma.
According to Celgene, the FDA-approved drug is a new class of therapy that has been designed to regulate late-stage maturation of red blood cells for reducing the RBC transfusion burden for patients.
Reblozyl cannot be used as a substitute for RBC transfusions in patients requiring immediate correction of anemia.
Beta thalassemia is an inherited blood disease that reduces haemoglobin production.
As per the FDA, supportive treatment for people having beta thalassemia usually includes lifelong regimens of chronic blood transfusions for survival and treatment for iron overload caused by the RBC transfusions. Patients with beta thalassemia are also have an increased risk of developing abnormal blood clots, said the regulator.
FDA Oncology Center of Excellence director and in the FDA Center for Drug Evaluation and Research Office of Oncologic Diseases acting director Richard Pazdur 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.”
The FDA approval for Reblozyl was based on the data from the phase 3 BELIEVE trial. The late-stage study evaluated the safety and efficacy of the drug for the treatment of anaemia in beta thalassemia in 336 adults, who require regular RBC transfusions.
The trial compared the erythroid maturation agent alongside the best supportive care (BSC) against placebo.
The BELIEVE trial registered a clinically meaningful and statistically significant improvement in the primary endpoint. The trial saw 21% of the patients subjected to Reblozyl showing at least 33% reduction in transfusions compared to 4.5% in the placebo arm.
Additionally, the phase 3 trial met key secondary endpoints that include transfusion burden reduction of at least 33%.
Celgene global hematology and oncology president Nadim Ahmed 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.”