PRINCETON -
Reblozyl is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.1
'In clinical trials, Reblozyl has shown to have significant benefit for the treatment of anemia in patients with myelodysplastic syndromes who have ring sideroblasts,' said
'Today's approval of Reblozyl is an important milestone for a majority of patients with myelodysplastic syndromes who have limited treatment options to address anemia associated with their disease. It also demonstrates our continued commitment to develop innovative products that improve the lives of patients living with serious diseases,' said
The FDA approval in MDS is based on results from the pivotal Phase 3 MEDALIST trial and marks the second indication for Reblozyl, which received its first approval in
'We're excited that Reblozyl has been approved to help even more patients in need of treatment options,' said
About MEDALIST
The approval of Reblozyl was based on the findings of MEDALIST, a Phase 3, randomized, double blind, placebo-controlled, multi-center study evaluating the efficacy and safety of Reblozyl in patients with IPSS-R-defined very low-, low- and intermediate-risk non-del(5q) myelodysplastic syndromes (MDS) with ring sideroblasts. All patients were red blood cell (RBC) transfusion-dependent and were either refractory or intolerant to prior erythropoiesis-stimulating agent (ESA) therapy or were
In the trial, a significantly greater proportion of patients receiving Reblozyl achieved independence from RBC transfusions for at least eight weeks during the first 24 weeks of the trial compared with those receiving placebo, meeting the study's primary endpoint.2 Additionally, a significantly greater proportion of patients receiving Reblozyl vs. placebo achieved at least 12 weeks of independence from transfusions within the first 24 and 48 weeks of the study.2
The majority of treatment-emergent adverse events (TEAEs) in the trial were Grade 1-2. Grade 3 or 4 treatment-emergent adverse events were reported in 42.5% of patients who received Reblozyl and 44.7% of patients who received placebo. The most common (>10%) all-grade adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, dyspnea, nausea, hypersensitivity reactions, headache, and upper respiratory tract infection.2
About MDS
Myelodysplastic syndromes (MDS) are a group of closely related blood cancers characterized by ineffective production of healthy red blood cells, white blood cells and platelets, which can lead to anemia and frequent or severe infections.2,3 People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation.4 Frequent transfusions are associated with an increased risk of iron overload, transfusion reactions and infections.5
About Reblozyl
Reblozyl, the first and only erythroid maturation agent, promotes late-stage red blood cell maturation in animal models.1
Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.
About
Celgene and
About Acceleron
Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner,
Bristol Myers Squibb Cautionary Statement Regarding Forward-Looking Statements
This press release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on historical performance and current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, whether Reblozyl for the additional indication described in this release will be commercially successful and that continued approval of Reblozyl for such additional indication described in this release may be contingent upon verification and description of clinical benefit in confirmatory trials. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect
Acceleron Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements about Acceleron's strategy, future plans and prospects, including statements regarding the development and commercialization of Acceleron's compounds, the timeline for clinical development and regulatory approval of Acceleron's, the expected timing for reporting of data from ongoing clinical trials, and the potential of Reblozyl (luspatercept-aamt) as a therapeutic drug. The words 'anticipate,' 'believe,' 'could,' 'estimate,' 'expect,' 'goal,' 'intend,' 'may,' 'plan,' 'potential,' 'project,' 'should,' 'target,' 'will,' 'would,' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Actual results could differ materially from those included in the forward-looking statements due to various factors, risks and uncertainties, including, but not limited to, that the results of any clinical trials may not be predictive of the results or success of other clinical trials, that regulatory approval of Acceleron's compounds in one indication or country may not be predictive of approval in another indication or country, that the development of Acceleron's compounds will take longer and/or cost more than planned or accelerate faster than currently expected, that Acceleron or its collaboration partner,
The forward-looking statements contained in this press release are based on management's current views, plans, estimates, assumptions, and projections with respect to future events, and Acceleron does not undertake and specifically disclaims any obligation to update any forward-looking statements.
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