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.

QUESTIONS ANSWERED

  • What role(s) will Bristol Myers Squibb(BMS) /Acceleron’s luspatercept (ACE-536) have in the management of MDS patients?
  • What is the commercial outlook for BMS’oral azacitidine (CC-486)?
  • Which emerging therapies are most likely to have an impact on the MDS therapy market in 2029 (e.g., AbbVie/Genentech’s Venclexta, Takeda’s Pevonedistat, BMS/Agios Pharmaceuticals’ Idhifa, Jazz Pharmaceuticals’ Vyxeos)? Which early-phase agents intrigue interviewed experts?
  • How will the size of the MDS population change through 2029?

PRODUCT DESCRIPTION

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

  • Myelodysplastic Syndromes - Landscape & Forecast - Disease Landscape & Forecast
    • COVID-19
      • Key Findings
        • MDS - Key Findings
      • Commercial Outlook
        • Key Findings
          • Regional Sales of Key Emerging Therapies to Treat Myelodysplastic Syndromes: 2029
          • SWOT Analysis
        • COVID-19: Areas of Potential Forecast Impact
          • 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
              • Alternative Market Scenarios
                • Alternative Market Scenarios
                • Alternative Scenarios for the Myelodysplastic Syndromes Market Through 2029
            • Forecast
              • Sales of Key Therapies in MDS
            • Etiology and Pathophysiology
              • Disease Overview
                • Etiology
                  • Primary and Secondary MDS
                  • Select Risk Factors Associated with MDS
                • Pathophysiology
                  • Documented Symptoms of MDS
                  • Characterization of MDS Pathophysiology
                  • Key Terms Used in Classifying and Understanding MDS
                  • MDS Microenvironment
                  • Molecular Mechanisms of MDS Pathology
                  • Molecular Mutations Found in Primary MDS Patients
                  • Prevalence of Cytogenetic Abnormalities
                  • Select Cytogenetic Abnormalities in MDS
                  • MDS 2008 WHO Classification System
                  • MDS 2016 WHO Classification System
                  • Expert Insight on MDS Classification Systems
                  • Pediatric MDS
                  • Characteristics of Pediatric MDS
                  • Prognostic Scoring Systems
                  • The International Prognostic Scoring System
                  • International Prognostic Scoring System for MDS
                  • Prognostic Risk Groups for MDS: IPSS Scores, Overall Survival, and Time to AML Progression
                  • Revisions to the International Prognostic Scoring System
                  • Revised International Prognostic Scoring System for MDS
                  • Prognostic Risk Groups for MDS: IPSS-R Scores, Overall Survival, and Time to AML Progression
                  • IPSS and IPSS-R: Comparing Stratification Systems
                  • Distribution of MDS Patients by Clinical Risk Category
                  • Distribution of MDS Patients by Cytogenetic Category
                  • MDS Patient Treatment Groups by Risk Category
                • Key Pathways and Drug Targets
                • Epidemiology
                  • Key Findings
                    • Epidemiology Populations
                      • Disease Definition
                      • Methods
                      • Sources Used for Diagnosed Incident Cases of MDS
                      • Number of Diagnosed Incident Cases of MDS in the Major Pharmaceutical Markets: 2019-2029
                      • Disease Definition
                      • Methods
                      • Sources Used for Diagnosed Prevalent Cases of MDS
                      • Number of Diagnosed Prevalent Cases of MDS in the Major Pharmaceutical Markets: 2019-2029
                      • Number of Drug-Treated Prevalent Cases of MDS in the Major Pharmaceutical Markets: 2019-2029
                      • Number of Drug-Treated Incident Cases of Lower-Risk MDS in the Major Pharmaceutical Markets: 2019-2029
                      • Number of Drug-Treated Incidence Cases of Higher-Risk MDS in the Major Pharmaceutical Markets: 2019-2029
                      • Disease Definition
                      • Methods
                      • Sources for Morphological Subtypes of MDS
                      • Number of Diagnosed Incident Cases of MDS in the Major Pharmaceutical Markets by Morphology: 2019-2029
                      • Number of Diagnosed Prevalent Cases of MDS in the Major Pharmaceutical Markets by Morphology: 2019-2029
                      • Disease Definition
                      • Methods
                      • Sources Used for IPSS and IPSS-R Score
                      • Number of Diagnosed Prevalent Cases of MDS in the Major Pharmaceutical Markets by IPSS Risk: 2019-2029
                      • Number of Diagnosed Prevalent Cases of MDS in the Major Pharmaceutical Markets by IPSS-R Risk: 2019-2029
                      • Number of Diagnosed Incident Cases of MDS in the Major Pharmaceutical Markets by IPSS Risk: 2019-2029
                      • Number of Diagnosed Incident Cases of MDS in the Major Pharmaceutical Markets by IPSS-R Risk: 2019-2029
                  • Current Treatment
                    • Key Findings
                      • Diagnosis
                        • Diagnostic Tools for MDS
                        • Expert Insight on Diagnosis of MDS
                      • Treatment Goals
                        • Key End Points Used in Clinical Trials for MDS
                      • Key Current Therapies
                        • Overview
                        • Mechanism of Action of Key Current Drugs or Drug Classes Used for MDS
                        • Current Treatments Used for MDS
                        • Market Events Impacting the Use of Key Current Therapies for MDS
                        • Advantages and Disadvantages of Erythropoiesis-Stimulating Agents
                        • Expert Insight: Erythropoiesis-Stimulating Agents
                        • Lenalidomide
                        • Key Results from Select Clinical Trials of Lenalidomide for the Treatment of MDS
                        • Advantages and Disadvantages of Lenalidomide
                        • Expert Insight: Lenalidomide
                        • Luspatercept
                        • Advantages and Disadvantages of Hypomethylating Agents for Lower-Risk Patients
                        • Expert Insight: Hypomethylating Agents
                        • Advantages and Disadvantages of Immunosuppressive Therapy
                        • Expert Insight: Immunosuppressive Therapy
                        • Advantages and Disadvantages of Iron Chelation Therapy
                        • Expert Insight: Iron Chelation Therapy
                        • Key Results from Select Clinical Trials of Azacitidine for the Treatment of MDS
                        • Advantages and Disadvantages of Azacitidine
                        • Expert Insight: Injectable Azacitidine
                        • Key Results of Select Clinical Trials of Decitabine for the Treatment of MDS
                        • Advantages and Disadvantages of Decitabine
                        • Expert Insight: Decitabine
                        • Advantages and Disadvantages of Induction or High-Intensity Chemotherapy
                        • Expert Insight: Induction or High-Intensity Chemotherapy
                        • Advantages and Disadvantages of Hematopoietic Stem Cell Transplantation
                        • Expert Insight on Hematopoietic Stem Cell Transplantation
                      • Medical Practice
                        • Overview
                        • Treatment Guidelines
                        • Myelodysplastic Syndromes Treatment Guidelines
                        • Generalized Treatment Decision Tree for Lower-Risk MDS
                        • Generalized Treatment Decision Tree for Higher-Risk MDS
                    • Unmet Need Overview
                      • Current and Future Attainment of Unmet Needs in MDS
                      • Top Unmet Needs in MDS: Current and Future Attainment
                    • Emerging Therapies
                      • Key Findings
                        • Pipeline Trends for MDS
                      • Key Emerging Therapies
                        • Key Therapies in Development for Myelodysplastic Syndromes
                        • Estimated Launch Dates of Key Emerging Therapies for the Treatment of Myelodysplastic Syndromes
                        • Oral Azacitidine Profile
                        • Key Results from Select Clinical Trials Investigating Oral Azacitidine for the Treatment of MDS
                        • Clinical Development of Oral Azacitidine
                        • Expectations for Launch and Sales Opportunity of Oral Azacitidine
                        • Guadecitabine Profile
                        • Clinical Development of Guadecitabine
                        • Key Ongoing Clinical Trials of Guadecitabine in the Treatment of MDS
                        • Expert Insight: Guadecitabine
                        • Expectations for Launch and Sales Opportunity of Guadecitabine
                        • ASTX-727 (Inqovi) Profile
                        • Clinical Development of ASTX-727 (Inqovi)
                        • Key Ongoing Clinical Trials of ASTX-727 (Inqovi) in the Treatment of MDS
                        • Expert Insight on ASTX-727 (Inqovi)
                        • Expectations for Launch and Sales Opportunity of ASTX-727 (Inqovi)
                        • Venetoclax Profile
                        • Key Results from Select Clinical Trials Investigating Venetoclax for the Treatment of MDS
                        • Clinical Development of Venetoclax
                        • Key Ongoing Clinical Trials of Venetoclax in the Treatment of MDS
                        • Expert Insight: Venetoclax
                        • Expectations for Launch and Sales Opportunity of Venetoclax
                        • Pevonedistat Profile
                        • Clinical Development of Pevonedistat
                        • Key Ongoing Clinical Trials of Pevonedistat in the Treatment of MDS
                        • Expert Insight: Pevonedistat
                        • Expectations for Launch and Sales Opportunity of Pevonedistat
                        • Expert Insight on IDH Inhibitors
                        • Enasidenib Profile
                        • Clinical Development of Enasidenib
                        • Key Ongoing Clinical Trials of Enasidenib in the Treatment of MDS
                        • Expectations for Launch and Sales Opportunity of Enasidenib
                        • Ivosidenib Profile
                        • Key Ongoing Clinical Trials of Ivosidenib for the Treatment of MDS
                        • Expectations for Launch and Sales Opportunity of Ivosidenib
                        • Eprenetapopt (APR-246) Profile
                        • Clinical Development of Eprenetapopt (APR-246)
                        • Key Ongoing Clinical Trials of Eprenetapopt (APR-246) in the Treatment of MDS
                        • Expert Insight: Eprenetapopt (APR-246)
                        • Expectations for Launch and Sales Opportunity of Eprenetapopt (APR-246) in MDS
                        • Vyxeos Profile
                        • Clinical Development of Vyxeos
                        • Key Ongoing Clinical Trials of Vyxeos in the Treatment of MDS
                        • Expert Insight: Vyxeos
                        • Expectations for Launch and Sales Opportunity of Vyxeos
                        • Omidubicel Profile
                        • Clinical Development of Omidubicel
                        • Expert Insight: Omidubicel
                        • Expectations for Launch and Sales Opportunity of Omidubicel
                        • Rigosertib (IV) Profile
                        • Key Results from Select Clinical Trials Investigating Rigosertib (PO) for the Treatment of MDS
                        • Clinical Development of Rigosertib
                        • Key Ongoing Clinical Trials of Rigosertib
                        • Expectations for Launch and Sales Opportunity of Rigosertib
                        • Magrolimab Profile
                        • Clinical Development of Magrolimab
                        • Key Ongoing Clinical Trials of Magrolimab in the Treatment of MDS
                        • Expert Insight: Magrolimab
                        • Expectations for Launch and Sales Opportunity of Magrolimab
                        • MBG-453 Profile
                        • Clinical Development of MBG-453
                        • Key Ongoing Clinical Trials of MBG-453 in the Treatment of MDS
                        • Expert Insight: MBG-453
                        • Expectations for Launch and Sales Opportunity of MBG-453
                        • Key Results from Select Clinical Trials Investigating Luspatercept for the Treatment of MDS
                        • Key Ongoing Clinical Trials of Luspatercept in the Treatment of MDS
                        • Expert Insight: Luspatercept
                        • Expectations for Launch and Sales Opportunity of Luspatercept
                        • Imetelstat Profile
                        • Clinical Development of Imetelstat
                        • Key Ongoing Clinical Trials of Imetelstat in the Treatment of MDS
                        • Expert Insight: Imetelstat
                        • Expectations for Launch and Sales Opportunity of Imetelstat
                        • H3B-8800 Profile
                        • H3B-8800 Clinical Development
                        • Expert Insight on H3B-8800
                        • Expectations for Launch and Sales Opportunity of H3B-8800
                      • Early- and Mid-Phase Pipeline Analysis
                        • Select Compounds in Early-/Mid-Phase Development for Lower-Risk MDS
                        • Clinical Development Strategy for Early-Phase Therapies for Lower-Risk MDS
                        • Select Compounds in Early-/Mid-Phase Development for Higher-Risk MDS
                        • Clinical Development Strategy for Early-Phase Emerging Therapies in Higher-Risk MDS Patients
                        • Expert Insight on the Early- and Mid-Phase Pipeline for MDS
                      • Key Discontinuations and Failures in MDS
                        • Key Discontinuations and Failures
                      • Patient Registries
                        • Patient Registries for MDS
                        • Prominent MDS Patient Organizations
                      • Orphan-Drug Designation
                      • Access and Reimbursement Overview
                        • Region-Specific Reimbursement Practices
                          • Key Market Access Considerations in MDS: United States
                          • General Reimbursement Environment: United States
                          • Key Market Access Considerations in Myelodysplastic Syndromes: EU5
                          • General Reimbursement Environment: EU5
                      • Appendix
                        • Key Abbreviations Related to MDS
                        • Brands, Marketers, and Generic Availability of Key Therapies for Myelodysplastic Syndromes by Market
                        • Bibliography
                        • Bibliography (Epidemiology)

                    Author(s): Rachit Singhal; Ullas Ulahannan

                    Nuno T. Antunes, Ph.D., 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 Ph.D. in animal health from the Universidad de las Palmas de Gran Canaria, Spain, and a D.V.M. 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.


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