The European Commission (EC) has granted full Marketing Authorization for Bristol Myers Squibb’s Reblozyl (luspatercept), a first-in-class therapeutic option, for treatment in adult patients of anemia associated with non-transfusion-dependent (NTD) beta thalassemia.
Reblozyl is currently approved in the European Union (EU), United States and Canada to address anemia associated with transfusion-dependent beta thalassemia and transfusion-dependent lower-risk myelodysplastic syndromes. The centralized Marketing Authorization approves use of Reblozyl in all EU member states, as well as Norway, Iceland and Liechtenstein.
“Beta thalassemia is an inherited blood disorder that puts patients at significant risk for long-term clinical complications due to anemia, leaving a substantial need for treatment options, regardless of a patient’s dependence on blood transfusions. This announcement is welcome news for patients with non-transfusion-dependent beta thalassemia associated anemia across the EU who are seeking newer treatment options to reduce these burdens,” said Noah Berkowitz, M.D., Ph.D., senior vice president, Hematology Development, Bristol Myers Squibb.
“Today’s approval represents the third indication for Reblozyl in Europe, and we look forward to continuing to evaluate this first-in-class therapeutic option across multiple diseases impacted by the burden of anemia in a broad clinical development program.”
The EC approval of Reblozyl was based on results from the Phase 2 BEYOND study, evaluating the efficacy and safety of Reblozyl versus placebo in 145 adults with NTD beta thalassemia. Patients were eligible to receive best supportive care, including red blood cell transfusions and iron-chelating agents.
Reblozyl is being developed and commercialized through a global collaboration with Merck following Merck’s acquisition of Acceleron Pharma, Inc. in November 2021.