Myelodysplastic syndromes (MDS) are a heterogeneous collection of hematopoietic stem cell disorders that result in cytopenias and the risk of progression to acute myeloid leukemia (AML). Although there are entrenched options to manage the anemia in lower-risk (e.g., erythropoiesis-stimulating agents) and higher-risk (e.g., hypomethylating agents) patients, there is a significant gap in the treatment of refractory disease. The FDA’s approval of Bristol Myers Squibb / Acceleron’s Reblozyl (luspatercept) for lower-risk adult MDS (2020) and Astex Pharmaceuticals’ Inqovi (decitabine and cedazuridine) for higher-risk adult MDS and refractory disease (2020) will provide additional effective treatments. The MDS pipeline is robust and promising, and several projected market entrants are poised to penetrate subpopulations of the lower-risk, intermediate-risk, and higher-risk MDS patients. Notably, emerging therapies are expected to fulfill the existing unmet need for second-line therapies for lower-risk, intermediate-risk, and higher-risk MDS.
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Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.
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Niche & Rare Disease Landscape & Forecast introduces a new Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.