Myelodysplastic Syndromes | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2020

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.


  • How will the launches of recently approved Reblozyl (luspatercept) and Inqovi (decitabine and cedazuridine) change MDS treatment?
  • What is the commercial outlook for current and emerging therapies for MDS?
  • Which emerging therapies are most likely to have an impact on the MDS therapy market by 2030 (e.g., AbbVie / Genentech’s Venclexta, Gilead’s magrolimab, Novartis’s sabatolimab, BMS / Agios Pharmaceuticals’ Idhifa, Jazz Pharmaceuticals’ Vyxeos)?
  • How will the size of the MDS population change over the forecast period?


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.


  • Geographies: United States, France, Germany, Italy, Spain, and the United Kingdom.
  • Primary research: Five country-specific interviews with thought-leading hematologists supported by survey data collected for this study.
  • Epidemiology: Diagnosed incident and prevalent cases of MDS by country, drug-treated prevalent and incident cases of MDS, diagnosed incident and prevalent cases of MDS by morphology, and diagnosed prevalent cases by prognostic risk.
  • Forecast: Drug-level sales and patient share of key MDS therapies in 2030.
  • Emerging therapies: Phase III/PR: 8 drugs; Phase II/III and II: 15 drugs; coverage of select early-phase products.
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