The recommendation will now be reviewed by the
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,
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
Reblozyl is being developed and commercialized through a global collaboration with Merck following Merck's acquisition of
Centralized Marketing Authorization does not include approval in
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
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