The European Commission has approved BMS’ blood disease therapy Reblozyl (luspatercept) for the treatment of transfusion-dependent anaemia associated with myelodysplastic syndromes (MDS) or beta thalassaemia.
The decision makes Reblozyl the first and only erythroid maturation agent approved in the EU, representing a new class of therapy for eligible patients, who lack new options to address their anaemia.
It is hoped that the drug could address the important need among patients suffering from disease-associated anaemia by helping decrease or eliminate their dependence on red blood cell transfusions.
In the EU, around 25 million blood transfusions occur every year, many of which are needed by people with anaemia due to haematologic diseases like MDS and beta thalassaemia.
Reblozyl's clearance was based on findings of the Phase III MEDALIST study, which assessed the safety and efficacy of the drug compared to plus best supportive care (BSC) in adults with IPSS-R-defined very low-, low- or intermediate-risk non-del(5q) MDS.
The trial showed a statistically significant improvement in red blood cell (RBC) transfusion burden with Reblozyl, the study’s primary endpoint, with 37.9% of patients treated with Reblozyl achieving independence from RBC transfusions for at least eight weeks during the first 24 weeks of the trial compared to 13.2% of patients on placebo.
The trial also met the secondary endpoint of transfusion independence for at least 12 weeks within the first 24 and 48 weeks of the study, which was achieved in a significantly greater proportion of patients receiving Reblozyl versus placebo.
The most common (>10%) all-grade adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity reactions, hypertension, headache, upper respiratory tract infection, bronchitis and urinary tract infection.
"Dependence on blood transfusions caused by anemia in hematologic malignancies like MDS can often mean frequent and lengthy hospital visits, which can pose additional health risks and affect patients’ quality of life,” said Uwe Platzbecker, lead investigator of the MEDALIST study, head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital.
The approval of Reblozyl “provides healthcare professionals with a new therapy that has been shown to significantly reduce the number of red blood cell transfusions needed by MDS patients and, in some cases, helped them to achieve transfusion independence.”