Bristol Myers Squibb Receives European Commission Approval of Reblozyl® (luspatercept) for Anemia in Adult Patients with Non-Transfusion-Dependent Beta Thalassemia

2023-03-06

研发

上市批准临床结果并购

Third authorized indication in Europe for Reblozyl, a first-in-class treatment for patients with diseases impacted by anemia

Bristol Myers Squibb (NYSE: BMY) today announced that the European Commission (EC) has granted full Marketing Authorization for 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. *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-aamt (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) 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 In a key secondary endpoint of the study, 47 of 96 patients (49.0%) treated with Reblozyl achieved mean Hb increase of ≥1.5 g/dL compared to baseline from Week 37-48 in the absence of transfusions versus 0 patients (0%) in the placebo arm (PReblozyl arm, 89.6% of patients remained transfusion free at weeks 1-24 versus 67.3% of patients in the placebo arm (P=0.0013). Improvements in patient-reported QoL outcomes (tiredness and weakness) were also observed to correlate with Hb increases. Serious adverse reactions occurred in 11.5% of patients (n=11) who received Reblozyl. The most common adverse reactions occurring in ≥10% of patients treated with Reblozyl were bone pain (36%), headache (30%), arthralgia (29%), back pain (28%), prehypertension (23%), hypertension (20%), cough (18%), diarrhea (17%), influenza-like illness (17%), asthenia (13%), influenza (13%), insomnia (11%) and nausea (10%).

About Beta Thalassemia Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. It is one of the most common autosomal recessive disorders, and the total annual incidence of symptomatic individuals is estimated at 1 in 100,000 people globally.1 The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy red blood cells (RBCs), often leading to severe anemia—a condition that can be debilitating and can lead to other complications for patients—as well as other serious health issues.2 Treatment options for anemia associated with beta thalassemia are limited, consisting mainly of frequent RBC transfusions that have the potential to contribute to iron overload, which can cause serious complications such as organ damage.1 Non-transfusion-dependent beta thalassemia is a term used to describe patients who do not require lifelong regular transfusions for survival, although they may experience a range of clinical complications and require occasional or even frequent transfusions, usually for defined periods of time.3

About Reblozyl® Reblozyl, a first-in-class therapeutic option, promotes late-stage red blood cell (RBC) maturation in animal models.1 Reblozyl is being developed and commercialized through a global collaboration with Merck following Merck’s acquisition of Acceleron Pharma, Inc. in November 2021. Reblozyl is currently approved in the U.S. for the treatment of:

Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.

About Bristol Myers Squibb Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

References:

1.Galanello R, Origa R. Beta thalassemia. Orphanet Journal of Rare Diseases. 2010;5(11). Available at: https://ojrd-biomedcentral-com.libproxy1.nus.edu.sg/articles/10.1186/1750-1172-5-11. Accessed February 2022.

2.Rivella, S. (2013). Ineffective erythropoiesis and thalassemias. Available at: https://www-ncbi-nlm-nih-gov.libproxy1.nus.edu.sg/pmc/articles/PMC3703923/pdf/nihms-490109.pdf. Accessed February 2022.

3.Musallam, K. M., Rivella, S., Vichinsky, E., & Rachmilewitz, E. A. (2013). Non-transfusion-dependent thalassemias. Haematologica, 98(6), 833–844. https://doi-org.libproxy1.nus.edu.sg/10.3324/haematol.2012.066845. Accessed February 2022.

SOURCE: Bristol Myers Squibb

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