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 / 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 and higher-risk MDS patients. Notably, emerging therapies are expected to fulfill the existing unmet need for second-line therapies for both lower-risk and higher-risk MDS.
QUESTIONS ANSWERED
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 2031?
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 2031.
Emerging therapies: Phase III/PR: > 7 drugs; Phase II/III and II: > 10 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.
Regional sales of key emerging therapies to treat myelodysplastic syndromes: 2031
SWOT analysis
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 2031
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
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
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
Molecular International Prognostic Scoring System: IPSS-M
Expert insight on IPSS-M prognostic scoring system
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: 2021-2031
Disease definition
Methods
Sources used for diagnosed prevalent cases of MDS
Number of diagnosed prevalent cases of MDS: 2021-2031
Number of drug-treated incident cases of lower-risk MDS in the major pharmaceutical markets: 2021-2031
Number of drug-treated incident cases of higher-risk MDS in the major pharmaceutical markets: 2021-2031
Disease definition
Methods
Sources for morphological subtypes of MDS
Number of diagnosed incident cases of MDS by morphology: 2021-2031
Number of diagnosed prevalent cases of MDS by morphology: 2021-2031
Disease definition
Methods
Sources used for IPSS and IPSS-Ru00a0score
Number of diagnosed prevalent cases of MDS by IPSS risk: 2021-2031
Number of diagnosed prevalent cases of MDS by IPSS-R risk: 2021-2031
Number of diagnosed incident cases of MDS by IPSS risk: 2021-2031
Number of diagnosed incident cases of MDS by IPSS-R risk: 2021-2031
Current treatment
Key findings
Diagnosis
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 of 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
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
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)
Advantages and disadvantages of induction or high-intensity chemotherapy
Advantages and disadvantages of 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
Unmet need overview
Current and future attainment of unmet needs in MDS
Expert insight: unmet needs in MDS
Expert insight: unmet needs in lower-risk MDS
Expert insight: unmet needs in higher-risk MDS
Top unmet needs in MDS: current and future attainment
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
Expectations for launch and sales opportunity of oral azacitidine
ASTX-030 profile
Clinical development of ASTX-030
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
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
Expert insight on IDH inhibitors
Enasidenib profile
Key results from select clinical trials investigating enasidenib for the treatment of MDS
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 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
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
Key results from select clinical trials investigating Vyxeos for the treatment of MDS
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
Terminated trial of IV rigosertib
Expectations for launch and sales opportunity of rigosertib
Magrolimab profile
Key results from select clinical trials investigating magrolimab for the treatment of MDS
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
Sabatolimab (MBG-453) profile
Key results from select clinical trials investigating sabatolimab for the treatment of MDS
Clinical development of sabatolimab (MBG-453)
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)
Tamibarotene (SY-1425) profile
Clinical development of tamibarotene (SY-1425)
Key ongoing clinical trials of tamibarotene (SY-1425) in the treatment of MDS
Expert insight: tamibarotene
Expectations for launch and sales opportunity of tamibarotene (SY-1425)
Orca-T (TRGFT-201)
Orca-T (TRGFT-201) profile
Clinical development of Orca-T (TRGFT-201)
Key ongoing clinical trials of Orca-T (TRGFT-201) in the treatment of MDS
Expectations for launch and sales opportunity of Orca-T (TRGFT-201)
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: H3B-8800
Expectations for launch and sales opportunity of H3B-8800
Roxadustat profile
Clinical development of roxadustat
Key ongoing clinical trials of roxadustat in the treatment of MDS
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-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
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
Appendix
Key Abbreviations Related to MDS
Bibliography
Bibliography (Epidemiology)
Vidhi Khosla
Vidhi Khosla, M.Sc., is an analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. She has several years of experience providing consulting, research, and analytic support in the pharmaceutical and healthcare domain and has worked in numerous therapy areas, including neurology, women’s health, metabolic disorders, and rare diseases. Prior to joining Clarivate, Ms. Khosla 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.
Ullas Ulahannan
Ullas Ulahannan, M.P.H., is an associate epidemiologist at Clarivate. Previously, he worked at Jhpiego (a John Hopkins University affiliate) as a senior program officer evaluating, implementing, and monitoring various health programs in collaboration with the state and central governments of India. He earned a B.S. in nursing from Rajiv Gandhi University and an M.P.H. from Manipal University.