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 recent approval of Bristol Myers Squibb (BMS) / Acceleron’s Reblozyl (luspatercept) for lower-risk adult MDS patients and Astex Pharmaceuticals’ Inqovi (decitabine and cedazuridine) for higher-risk adult MDS patients and refractory patients 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 and higher-risk MDS patients. Notably, emerging therapies are expected to fulfill the existing unmet need for a second-line therapy for both lower-risk and higher-risk MDS. Interviewed experts express enthusiasm for the clinical potential of several agents that are approved or marketed for AML, particularly those that target specific genetic populations, as treatments for MDS.
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.