Myelodysplastic Syndromes – Landscape & Forecast – Niche & Rare Disease Landscape & Forecast
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 approval of Bristol Myers Squibb / Acceleron’s Reblozyl (luspatercept) for lower-risk adult MDS (2020) and Astex Pharmaceuticals’ Inqovi (decitabine and cedazuridine) for higher-risk adult MDS and refractory disease (2020) 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, intermediate-risk, and higher-risk MDS patients. Notably, emerging therapies are expected to fulfill the existing unmet need for second-line therapies for lower-risk, intermediate-risk, and higher-risk MDS.
QUESTIONS ANSWERED
- How will the launches of 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 2032?
- How will the size of the MDS population change over the forecast period?
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.
CONTENT HIGHLIGHTS
- Geographies: United States, France, Germany, Italy, Spain, and the United Kingdom.
- Primary research: Six country-specific interviews with thought-leading hematologists supported by survey data collected for this study.
- Epidemiology: Diagnosed incident and diagnosed 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 through 2032.
- Emerging therapies: Phase 3/PR: > 6 drugs; Phase 2/3 and 2: > 9 drugs; coverage of select early-phase products.
SOLUTION ENHANCEMENT
Niche & Rare Disease Landscape & Forecast introduces a new Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.
Table of contents
- Myelodysplastic Syndromes - Landscape & Forecast - Niche & Rare Disease Landscape & Forecast
- Key Findings
- Myelodysplastic Syndromes Key Findings - August 2023
- Commercial Outlook
- Key findings
- Regional sales of key emerging therapies to treat MDS: 2032
- SWOT analysis
- Drivers and constraints
- What factors are driving sales in MDS?
- What factors are constraining sales in MDS?
- Lower-risk MDS-specific trends
- Lower-risk MDS
- Higher-risk MDS-specific trends
- Higher-risk MDS
- Key findings
- Forecast
- Sales of key therapies in MDS
- Etiology and Pathophysiology
- Disease overview
- Etiology
- Primary and secondary MDS
- Select risk factors associated with MDS
- Expert insight on the etiology of MDS
- Pathophysiology
- Expert insight on pathophysiology of MDS
- Documented symptoms of MDS
- Disease progression
- Characterization of MDS pathophysiology
- Key terms used in classifying and understanding MDS
- MDS microenvironment
- MDS microenvironment and its influence on disease progression
- Molecular mechanisms of MDS pathology
- Molecular mutations found in primary MDS patients
- Prevalence of cytogenetic abnormalities
- Select cytogenetic abnormalities in MDS
- Morphological classification systems
- MDS 2008 WHO classification system
- MDS 2016 WHO classification system
- MDS 2022 WHO classification system
- Pediatric MDS
- Characteristics of pediatric MDS
- Prognostic scoring systems
- 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
- Expert insight on IPSS-R prognostic scoring system
- Distribution of MDS patients by clinical risk category
- Distribution of MDS patients by cytogenetic category
- MDS patient treatment groups by risk category
- MDS patient treatment groups by risk category
- Molecular International Prognostic Scoring System: IPSS-M
- IPSS-M MDS scoring system
- Expert insight on IPSS-M prognostic scoring system
- Disease overview
- Epidemiology
- Key findings
- Epidemiology populations
- Disease definition, methods, and sources used
- Number of diagnosed incident cases of MDS: 2022-2032
- Disease definition, methods, and sources used
- Number of diagnosed prevalent cases of MDS: 2022-2032
- Disease definition, methods, and sources used
- Number of diagnosed incident cases of MDS by morphology: 2022-2032
- Number of diagnosed prevalent cases of MDS by morphology: 2022-2032
- Disease definition, methods, and sources used
- Number of diagnosed incident cases of MDS by IPSS risk: 2022-2032
- Number of diagnosed incident cases of MDS by IPSS-R risk: 2022-2032
- Number of diagnosed prevalent cases of MDS by IPSS risk: 2022-2032
- Number of diagnosed prevalent cases of MDS by IPSS-R risk: 2022-2032
- Drug-treated cases
- Number of drug-treated incident cases of lower-risk MDS in the major pharmaceutical markets: 2022-2032
- Number of drug-treated incident cases of intermediate-risk MDS in the major pharmaceutical markets: 2022-2032
- Number of drug-treated incident cases of higher-risk MDS in the major pharmaceutical markets: 2022-2032
- Key findings
- Current Treatment
- Key findings
- Diagnosis
- Comprehensive diagnosis workup to confirm MDS
- Diagnostic tools for MDS
- Expert insight on diagnosing MDS
- Treatment goals
- Key endpoints used in clinical trials for MDS
- Expert insight on treatment goals of MDS
- Key current therapies
- Overview
- Mechanism of action of key current drugs or drug classes used for MDS
- Current treatments used for MDS
- Expert insight on current treatment: low-risk MDS
- Expert insight on current treatment: intermediate-risk MDS
- Expert insight on current treatment: high-risk 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
- Key results from select clinical trials of luspatercept for the treatment of MDS
- Key ongoing clinical trials of luspatercept in the treatment of MDS
- Advantages and disadvantages of luspatercept
- Expert insight: Luspatercept
- Advantages and disadvantages of hypomethylating agents for lower-risk patients
- Expert insight: hypomethylating agents
- Advantages and disadvantages of immunosuppressive therapies
- Expert insight: immunosuppressive therapies
- Advantages and disadvantages of iron chelation therapies
- Expert insights: iron chelation therapies
- Key results from select clinical trials of azacitidine for the treatment of MDS
- Advantages and disadvantages of azacitidine
- Expert insight: Injectable azacitidine
- Key results from select clinical trials of decitabine for the treatment of MDS
- Advantages and disadvantages of decitabine
- Key results from select clinical trials of decitabine / cedazuridine for the treatment of MDS
- Advantages and disadvantages of decitabine / cedazuridine (Inqovi)
- Expert insights: Inqovi
- Advantages and disadvantages of induction or high-intensity chemotherapies
- Advantages and disadvantages of hematopoietic stem cell transplantation
- Expert insight: hematopoietic stem cell transplantation
- Medical practice
- Overview
- Treatment guidelines
- MDS treatment guidelines
- Generalized treatment decision tree for lower-risk MDS
- Generalized treatment decision tree for higher-risk MDS
- Key findings
- Unmet Need Overview
- Current and future attainment of unmet needs in MDS
- Top unmet needs in MDS: current and future attainment
- Expert insight: unmet needs in MDS
- Expert insight: unmet needs in lower-risk MDS
- Expert insight: unmet needs in higher-risk MDS
- Drug Pipeline
- Pipeline
- Regulatory Milestones
- Indication Comparison
- Emerging Therapies
- Key findings
- 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
- Expert insight: oral azacitidine
- Expectations for launch and sales opportunity of oral azacitidine
- ASTX-030 profile
- Key ongoing clinical trials of ASTX-030 in the treatment of MDS
- Expectations for launch and sales opportunity of ASTX-030
- Venetoclax profile
- Key results from select clinical trials investigating venetoclax for the treatment of MDS
- Key clinical trials of venetoclax in the treatment of MDS
- Expert insight: venetoclax
- Expectations for launch and sales opportunity of venetoclax
- Expert insights: IDH inhibitors
- Enasidenib profile
- Key results from select clinical trials investigating enasidenib for the treatment of MDS
- Key ongoing clinical trials of enasidenib in the treatment of MDS
- Expectations for launch and sales opportunity of enasidenib
- Ivosidenib profile
- Key results from select clinical trials investigating ivosidenib for the treatment of MDS
- Key ongoing clinical trials of ivosidenib for the treatment of MDS
- Expectations for launch and sales opportunity of ivosidenib
- Eprenetapopt (APR-246) profile
- Key results from select clinical trials investigating eprenetapopt (APR-246) for the treatment of MDS
- Expert insight: eprenetapopt (APR-246)
- Expectations for launch and sales opportunity of eprenetapopt (APR-246) in MDS
- Vyxeos profile
- Key results from select clinical trials investigating Vyxeos for the treatment of MDS
- Key ongoing clinical trials of Vyxeos in the treatment of MDS
- Clinical development of Vyxeos
- Expert insight: Vyxeos
- Expectations for launch and sales opportunity of Vyxeos
- Omisirge (omidubicel) profile
- Key results from select clinical trials investigating Omisirge (omidubicel) for the treatment of MDS
- Clinical development of Omisirge (omidubicel)
- Expert insight: Omisirge (omidubicel)
- Expectations for launch and sales opportunity of Omisirge (omidubicel)
- Rigosertib profile
- Key results from select clinical trials investigating rigosertib for the treatment of MDS
- Terminated trial of IV rigosertib
- Key trials of PO rigosertib
- Clinical development of rigosertib
- Expectations for launch and sales opportunity of rigosertib
- Magrolimab profile
- Key results from select clinical trials investigating magrolimab for the treatment of MDS
- Key ongoing clinical trials of magrolimab in the treatment of MDS
- Clinical development of magrolimab
- Expert insight: magrolimab
- Expectations for launch and sales opportunity of magrolimab
- Sabatolimab (MBG-453) profile
- Key results from select clinical trials investigating sabatolimab for the treatment of MDS
- Key ongoing clinical trials of sabatolimab (MBG-453) in the treatment of MDS
- Expert insight: sabatolimab (MBG-453)
- Expectations for launch and sales opportunity of sabatolimab (MBG-453)
- Tamibarotene (SY-1425) profile
- Key results from select clinical trials investigating tamibarotene for the treatment of MDS
- Key ongoing clinical trials of tamibarotene (SY-1425) in the treatment of MDS
- Clinical development of tamibarotene (SY-1425)
- Expert insight: tamibarotene
- Expectations for launch and sales opportunity of tamibarotene (SY-1425)
- Orca-T (TRGFT-201) profile
- Key results from select clinical trials investigating Orca-T for the treatment of MDS
- Key ongoing clinical trials of Orca-T (TRGFT-201) in the treatment of MDS
- Expert insight: Orca-T
- Expectations for launch and sales opportunity of Orca-T (TRGFT-201)
- Imetelstat profile
- Key results from select clinical trials investigating imetelstat for the treatment of MDS
- Key ongoing clinical trials of imetelstat in the treatment of MDS
- Expert insight: imetelstat
- Expectations for launch and sales opportunity of imetelstat
- RVT-2001 (H3B-8800) profile
- Key results from select clinical trials investigating RVT-2001 (H3B-8800) for the treatment of MDS
- RVT-2001 (H3B-8800) clinical development
- Expectations for launch and sales opportunity of RVT-2001 (H3B-8800)
- Roxadustat profile
- Key results from select clinical trials investigating roxadustat for the treatment of MDS
- Key clinical trials of roxadustat in the treatment of MDS
- Clinical development of roxadustat
- Expert insight: roxadustat
- Expectations for launch and sales opportunity of roxadustat
- Early- and mid-phase pipeline analysis
- Select compounds in early- / mid-phase development for lower-risk MDS
- Clinical development strategy for early- / mid-phase therapies for lower-risk MDS
- Select compounds in early- / mid-phase development for higher-risk MDS
- Clinical development strategy for early-phase therapies for higher-risk MDS
- Key discontinuations and failures in MDS
- Patient registries
- Patient registries for MDS
- Prominent MDS patient organizations
- Orphan drug designation
- Key findings
- Access and Reimbursement Overview
- Region-specific reimbursement practices
- Key market access considerations in MDS: United States
- Expert insight on market access and reimbursement of MDS: United States
- General reimbursement environment: United States
- Key market access considerations in MDS: EU5
- Expert insight on market access and reimbursement of MDS: EU5
- General reimbursement environment: EU5
- Region-specific reimbursement practices
- Appendix
- Key abbreviations related to MDS
- Bibliography
- Bibliography (epidemiology)
- Key Findings
Vidhi Khosla, M.B.A., M.Sc., Healthcare Research & Data Analyst, Infectious, Niche, and Rare Diseases. Ms. Khosla has several years of experience providing consulting, research, and analytic support in the pharmaceutical and healthcare domains and has worked in numerous therapy areas, including neurology, women’s health, metabolic disorders, and rare diseases. Prior to joining Clarivate, she worked at WNS Global Services and DelveInsight Business Research LLP. She earned her bachelor’s and master’s (gold medalist) degrees in biochemistry from Delhi University and Kurukshetra University, respectively, and an M.B.A. from Symbiosis International University.
Aishwarya S. Ambat, M.P.H., Epidemiologist , Epidemiology. She received her M.P.H. from the Manipal Academy of Higher Education in India and holds a bachelor’s degree in physiotherapy. Her areas of interest are rheumatic disease and cancer epidemiology.