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.

Table of contents

  • Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
      • Drivers and Constraints
        • What Factors Are Driving Sales in MDS?
        • What Factors Are Constraining Sales in MDS?
      • Lower-Risk MDS-Specific Trends
      • Higher-Risk MDS-Specific Trends
    • Forecast
    • Etiology and Pathophysiology
      • Disease Overview
      • Etiology
      • Pathophysiology
        • Symptoms of MDS
        • Disease Progression
        • Mechanisms of MDS Pathology
        • Morphological Classification of MDS
        • Assessing Prognostic Risk in MDS
      • Key Pathways and Drug Targets
    • Epidemiology
      • Key Findings
      • Epidemiology Populations
        • Diagnosed Incident Cases of MDS
        • Diagnosed Prevalent Cases of MDS
        • Drug-Treated Prevalent Cases of MDS
        • Morphological Subtypes of MDS
        • IPSS and IPSS-R Score
    • Current Treatment
      • Key Findings
      • Diagnosis
      • Treatment Goals
      • Key Current Therapies
        • Overview
        • Current Therapies for Lower-Risk MDS
        • Current Therapies for Higher-Risk Myelodysplastic Syndromes
      • Medical Practice
        • Overview
        • Treatment Guidelines
        • Region-Specific Treatment Practices
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in MDS
    • Emerging Therapies
      • Key Findings
      • Key Emerging Therapies
        • Notable Developments Among Key Emerging Therapies for Myelodysplastic Syndromes
        • Emerging Therapies for Higher-Risk MDS Patients
        • Emerging Therapies for Lower-Risk MDS Patients
      • Early- and Mid-Phase Pipeline Analysis
        • Notable Developments in the Mid-Phase Pipeline for MDS
      • Patient Registries
        • Patient Organizations
      • Orphan-Drug Designation
        • Orphan-Drug Provisions: United States
        • Orphan-Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
      • Looking for More?
    • Methodology
      • Bottom-Up Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • General Statements About Pricing
        • Generic Erosion
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Bibliography
      • Bibliography (Epidemiology)

Author(s): Nuno Tiago Giao Antunes, PhD; Atul Sharma, MPH

Nuno T. Antunes, ,  is senior business insights analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing in antibacterial agents. Previously, he was a Latin America Market Access senior analyst in the Global Market Access Insights Team, where he developed expertise in market access, pricing and reimbursement, health technology assessment, and health policy. Nuno holds a in animal health from the Universidad de las Palmas de Gran Canaria, Spain, and a degree from the Universidade de Trás-os-Montes e Alto Douro, Portugal. Prior to joining DRG, he conducted research in antimicrobial resistance and antimicrobial development, and worked in the medical devices industry as a scientist.

Atul Sharma started working in Decision Resources Group as an intern in early 2016 and currently works as an associate epidemiologist. He performs fully documented systematic reviews of both published and grey literature on the epidemiology of assigned diseases and their risk factors to estimate incidence/prevalence over a 10-30 year period. He produces analyses for pharmaceutical drug developers on the descriptive epidemiology of major drug indications in mature and developing markets. He holds a Master’s in Public Health degree from School of Public Health, Post-Graduate Institute of Medical Education and Research and a Bachelor’s in dental surgery from MN DAV Dental College & Hospital.