{"id":44692,"date":"2022-06-03T23:01:44","date_gmt":"2022-06-03T21:01:44","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/bristol-myers-squibb-withdraws-supplemental-biologics-license-application-sbla-for-reblozyl-luspatercept-aamt-for-non-transfusion-dependent-ntd-beta-thalassemia\/"},"modified":"2022-06-03T23:01:44","modified_gmt":"2022-06-03T21:01:44","slug":"bristol-myers-squibb-withdraws-supplemental-biologics-license-application-sbla-for-reblozyl-luspatercept-aamt-for-non-transfusion-dependent-ntd-beta-thalassemia","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/bristol-myers-squibb-withdraws-supplemental-biologics-license-application-sbla-for-reblozyl-luspatercept-aamt-for-non-transfusion-dependent-ntd-beta-thalassemia\/","title":{"rendered":"Bristol Myers Squibb Withdraws Supplemental Biologics License Application (sBLA) for Reblozyl\u00ae (luspatercept-aamt) for Non-transfusion Dependent (NTD) Beta Thalassemia"},"content":{"rendered":"
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PRINCETON, N.J.–(BUSINESS WIRE)–Bristol Myers Squibb<\/a> (NYSE: BMY) today announced that the company has withdrawn a supplemental biologics license application (sBLA) for Reblozyl<\/i>\u00ae<\/sup> (luspatercept-aamt) for the treatment of anemia in adults with non-transfusion dependent (NTD) beta thalassemia. The Company could not appropriately address the U.S. Food and Drug Administration\u2019s questions about the benefit-risk profile of Reblozyl<\/i> in this patient population based on the current dataset from the Phase 2 BEYOND trial.\n<\/p>\n

<\/a><\/p>\n

\n\u201cWhile we will not pursue this indication in the U.S., we\u2019re continuing to evaluate Reblozyl <\/i>in a broad clinical development program to bring this important therapeutic option to more patients living with the burden of anemia,\u201d said Noah Berkowitz, M.D., Ph.D., senior vice president, Hematology Development, Bristol Myers Squibb.\n<\/p>\n

\nReblozyl<\/i>, a first-in-class therapeutic option, is currently approved in the United States, European Union and Canada to address transfusion-dependent anemia-associated beta thalassemia and lower-risk myelodysplastic syndromes.\n<\/p>\n

\nAbout BEYOND<\/span><\/b>\n<\/p>\n

\nBEYOND (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 <\/i>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 \u22651.0 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).\n<\/p>\n

\nAbout Beta Thalassemia<\/span><\/b>\n<\/p>\n

\nBeta 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<\/sup> While beta thalassemia remains a rare disease, its prevalence has increased in the United States by approximately 7.5% over the last 50 years.2<\/sup> 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\u2014a condition that can be debilitating and can lead to other complications for patients\u2014as well as other serious health issues.3<\/sup> 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<\/sup> 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.4<\/sup>\n<\/p>\n

\nAbout Reblozyl\u00ae<\/sup><\/i><\/span><\/b>\n<\/p>\n

\nReblozyl, <\/i>a first-in-class therapeutic option, promotes late-stage red blood cell maturation in animal models.1<\/sup> Reblozyl<\/i> is being developed and commercialized through a global collaboration with Merck following Merck\u2019s acquisition of Acceleron Pharma, Inc. in November 2021. Reblozyl<\/i> is currently approved in the U.S. for the treatment of:\n<\/p>\n