Bristol Myers Squibb and Acceleron Pharma have secured approval from the European Commission (EC) for their jointly developed erythroid maturation agent Reblozyl (luspatercept) for the treatment of transfusion-dependent anemia in adult patients.
The approval of Reblozyl is for transfusion-dependent anemia caused by very low-, low- and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response or are not eligible for erythropoietin-based therapy. Reblozyl has also been approved in the European Union for adult patients having transfusion-dependent anemia associated with beta thalassemia.
Reblozyl is now the first and only erythroid maturation agent approved in the European Union. Its approval is based on the findings from the phase 3 MEDALIST and BELIEVE clinical trials, which assessed the ability of Reblozyl to effectively address anemia associated with myelodysplastic syndromes and beta thalassemia, respectively.
In the MEDALIST clinical trial, Reblozyl delivered a statistically significant improvement in RBC transfusion burden, which is the study’s primary endpoint, with 37.9% of patients subjected to it achieving independence from RBC transfusions for at least eight weeks during the first 24 weeks of the trial in comparison to 13.2% of patients on placebo.
Diane McDowell – vice president of Bristol Myers Squibb Hematology Global Medical Affairs said: “Across the EU, 25 million blood transfusions occur every year, some of which are needed by patients with anemia due to hematologic diseases like MDS and beta thalassemia.
“Reblozyl has the potential to address the ineffective erythropoiesis associated with MDS and beta thalassemia, decrease patients’ dependence on red blood cell transfusions and impact the underlying consequences of the high burden of anemia for these patients. Alongside our partners at Acceleron, we recognize the continuing need in disease-related anemias and are committed to working collaboratively with European health authorities to make Reblozyl available to these patients as quickly as possible.”
The erythroid maturation agent has approval in the US for the treatment of anemia in adults with beta thalassemia who need regular red blood cell transfusions. Reblozyl also has the FDA approval for the treatment of anemia failing an erythropoiesis stimulating agent and needing two or more red blood cell units over eight weeks in adults with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).