Myelodysplastic Syndromes | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2019

Myelodysplastic syndromes (MDS) are a heterogeneous collection of hematopoietic stemcell disorders that result in cytopenias and the risk of progression to acute myeloid leukemia (AML). Although entrenched options are available to manage anemia in lower-risk (e.g., erythropoiesis-stimulating agents) and higher-risk (e.g., hypomethylating agents) patients, a significant gap in treatment exists 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.


  • What role(s) will Celgene/Acceleron’s luspatercept (ACE-536) have in the management of MDS patients?
  • What is the commercial outlook for Celgene’s oral azacitidine (CC-486)?
  • Which emerging therapies are most likely to have an impact on the MDS therapy market in 2028 (e.g., AbbVie/Genentech’s Venclexta, Celgene/Agios Pharmaceuticals’ Idhifa, Jazz Pharmaceuticals’ Vyxeos)? Which early-phase agents intrigue interviewed experts?
  • How will the size of the MDS population change through 2028?


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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