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.
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
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
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.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
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.