The BELIEVE trial has reached both primary and secondary end points and REBLOZYL has demonstrated reduced RBC transfusion burden in patients, compared to placebo
Image: FDA centre for drug evaluation and research. Photo: Courtesy of The U.S. Food and Drug Administration/Wikipedia.
Celgene and Acceleron have reported the US Food and Drug Administration (FDA) approval of REBLOZYL (luspatercept-aamt) for the treatment of anaemia in adult beta thalassemia patients who require regular red blood cell (RBC) transfusions.
Jointly developed by Celgene and Acceleron under a global collaboration, REBLOZYL is an erythroid maturation agent designed to promote the late-stage red blood cell maturation in animal models.
Acceleron president and chief executive officer Habib Dable said: “We’re thrilled that Acceleron’s first approved medicine is one with the potential to help patients with beta thalassemia, who have been in need of new treatments for this lifelong disease.
“We are enormously grateful to the patients, families and caregivers who participated in and supported our research. Their contributions have been essential in helping to ensure that REBLOZYL would emerge successfully from our longstanding collaboration with Celgene.”
REBLOZYL received Priority Review designation from the FDA
Beta thalassemia is a rare blood disorder caused by a genetic defect in haemoglobin, and is related with ineffective erythropoiesis, resulting in reduced and less healthy RBCs, which leads to severe anaemia.
Anaemia associated with beta thalassemia has very limited treatment options, which primarily comprise RBC transfusions. The treatment options may contribute to iron overload that causes serious complications including organ damage.
Celgene said that REBLOZYL marks the first and only erythroid maturation agent to secure FDA approval, and represents a new class of therapy that regulates red blood cell maturation to reduce the RBC transfusion burden for patients.
In addition, the regulatory approval of REBLOZYL for treating beta thalassemia is based on results from the Phase 3, randomized, double-blind, placebo-controlled, multicentre clinical trial BELIEVE, designed to evaluate the safety and efficacy of the drug in treating anaemia with beta thalassemia.
The BELIEVE trial has reached both primary and secondary end points and REBLOZYL has demonstrated reduced RBC transfusion burden in patients, compared to placebo.
Celgene said that REBLOZYL is not indicated to be used as a substitute for RBC transfusions for patients who need immediate correction of anaemia.
Celgene global haematology and oncology president Nadim Ahmed said: “Today’s approval is an important milestone and underscores our continued commitment to patients with hematology disorders.
“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.”