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 anemia in lower-risk (e.g., erythropoiesis-stimulating agents) and higher-risk (e.g., hypomethylating agents) patients, there is a significant gap in treatment for patients who are refractory to the first-line standards of care. The MDS pipeline is robust, and several projected market entrants are poised to penetrate subpopulations of the lower-risk and higher-risk MDS patients. Although the development of novel agents for higher-risk MDS patients has been particularly challenging, 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.
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.