Approval by the European Commission would expand the indications of Reblozyl for treatment of anemia in adults with beta thalassemia in Europe

In the pivotal BEYOND study, Reblozyl significantly increased hemoglobin levels, which were sustained over longer time compared to placebo

PRINCETON N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has recommended approval of Reblozyl® (luspatercept) as a treatment for adult patients with anemia associated with non‑transfusion-dependent (NTD) beta thalassemia. The recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for use in the European Union (EU). Upon approval, this would represent the third authorized indication for Reblozyl in the EU.

The CHMP adopted a positive opinion based on results from the pivotal 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, iron-chelating agents, use of antibiotic, antiviral, and antifungal therapy, and/or nutritional support, as needed.

Reblozyl is an erythroid maturation agent approved in the EU, the United States, China and Canada to address anemia-associated, transfusion-dependent beta thalassemia and transfusion-dependent lower-risk myelodysplastic syndromes, representing an important class of therapy for eligible patients.

“Beta thalassemia is an inherited blood disorder that puts patients at significant risk for long-term clinical complications that can impair their quality of life, regardless of whether they require regular blood transfusions,” said Noah Berkowitz, ​M.D., Ph.D., senior vice president, Hematology Development, Bristol Myers Squibb. “Results from the BEYOND study showed Reblozyl improved anemia associated with non-transfusion-dependent beta thalassemia by sustaining hemoglobin increases in 77% of patients regardless of their baseline hemoglobin status. The potential approval of Reblozyl for patients with non-transfusion-dependent beta thalassemia represents an important development in the EU where several countries present with a high prevalence and where more people are impacted by the disease.”

The EC is expected to deliver its final decision within 67 days of receipt of the CHMP opinion. The decision will be applicable to all EU member states, Iceland, Norway and Liechtenstein.*

Reblozyl is being developed and commercialized through a global collaboration with Merck following Merck’s acquisition of Acceleron Pharma, Inc. in November 2021.

*Centralized Marketing Authorization does not include approval in Great Britain (England, Scotland and Wales).

About BEYOND

BEYOND (NCT03342404) is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion-dependent beta thalassemia. The study is divided into the Screening Period, Double-blind Treatment Period (DBTP), Open-label Phase (OLP), and Post-Treatment Follow-up Period (PTFP) and randomized 145 subjects at a 2:1 ratio of Reblozyl versus placebo. All patients were eligible to receive best supportive care, which included red blood cell transfusions; iron-chelating agents; use of antibiotic, antiviral, and antifungal therapy; and/or nutritional support, as needed. The primary endpoint of the study is the proportion of subjects who have an increase from baseline ≥1.0 g/dL in mean of hemoglobin values over a continuous 12-week interval from Week 13 to Week 24 of treatment in the absence of transfusions. Key secondary endpoints include mean change in non-transfusion-dependent beta thalassemia-patient reported outcome (NTDT-PRO) Tiredness and Weakness (TW) domain score and baseline hemoglobin (Hb).

Results demonstrated 74 of 96 (77.1%) patients in the Reblozyl treatment arm achieved the study’s primary endpoint, ≥1.0 g/dL mean Hb increase from baseline, versus 0 of 49 (0%) patients in the placebo arm (P