Myelodysplastic Syndrome Market Insight
DelveInsight’s ‘Myelodysplastic Syndrome - Market Insights, Epidemiology and Market Forecast– 2030’ report delivers an in-depth understanding of the Myelodysplastic Syndrome, historical and forecasted epidemiology as well as the Myelodysplastic Syndrome market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), And Japan.
The Myelodysplastic Syndrome market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Myelodysplastic Syndrome market size from 2018 to 2030. The report also covers current Myelodysplastic Syndrome treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
Geography Covered
- The United States
- EU5 (Germany, France, Italy, Spain, and the United Kingdom)
- Japan
Study Period: 2018–2030
Myelodysplastic Syndrome Disease Understanding
Myelodysplastic Syndrome Overview
Myelodysplastic syndrome (MDS) is a heterogeneous group of hematologic neoplasms classically described as a clonal disorder of hematopoietic stem cells leading to dysplasia and ineffective hematopoiesis in the bone marrow. In MDS, also known as myelodysplasia, the bone marrow cells do not develop into mature blood cells; instead, these cells stay within the bone marrow in an immature state. There are many subtypes of MDS; some cases are mild, while others are more severe and carry a high risk of becoming acute myelogenous leukemia (AML). The subtype and the severity case depend on many factors, including how low the blood counts are and any genetic changes in bone marrow cells. MDS and aplastic anemia (AA) and Paroxysmal nocturnal hemoglobinuria (PNH), are bone marrow failure diseases. Bone marrow failure occurs when the marrow does not produce enough red cells, white cells, or platelets, or the produced blood cells are damaged or defective. This means the body cannot supply itself with the blood it needs.
MDS may be de novo or related to prior use of chemotherapeutic agents, also known as treatment-related MDS (t-MDS). The actual preceding factor(s) for de novo MDS is not entirely understood but assumed to occur from an oncogenic process resulting in one or more somatic mutations. Over recent years, much insight has been gained into mutations that are commonly altered in MDS due to advances and the rapid availability of gene sequencing. With these developments, researchers can identify one or more driver mutations in up to 80─90% of patients with some of the most common ones, including SF3B1, TET2, SRSF2, ASXL1, DNMT3A, RUNX1, U2AF1, TP53, and EZH2. RUNX1, for example, is a mutation noted to disrupt normal hematopoiesis. More than 100 genes are recurrently mutated in MDS, and these encode spliceosome components, chromatin remodeling factors, epigenetic pattern modulators, and transcription factors, among others.
Myelodysplastic Syndrome Diagnosis
MDS is generally diagnosed when a patient is evaluated for low blood counts, although in some MDS patients, the white blood count, platelet count, or both may be elevated. The main feature of MDS is a bone marrow aspirate and biopsy that reveals heavy infiltration with abnormal-looking bone marrow cells. A chromosome analysis, called cytogenetics, is performed on the bone marrow sample. In patients with MDS, immature cells called blasts make up less than 20% of the cells in the bone marrow. If blast cells make up more than 20%, the patient is diagnosed with acute myeloid leukemia.
Myelodysplastic Syndrome Treatment
The MDS is a very heterogeneous group of myeloid disorders where the prognosis of patients with MDS can be calculated using a number of scoring systems. In general, all these scoring systems include analysis of peripheral cytopenias, percentage of blasts in the bone marrow, and cytogenetic characteristics. The most commonly accepted system is the Revised International Prognostic Scoring System (IPSS-R). Somatic mutations can help define prognosis and therapy. Therapy is generally selected based on risk, transfusion needs, and percent of bone marrow blasts, cytogenetic and mutational profiles, comorbidities, the potential for allogeneic stem cell transplantation (alloSCT), and prior exposure to hypomethylating agents (HMA). The goals of therapy are different in lower-risk patients than in higher risk and in those with HMA failure. In lower risk, the goal is to decrease transfusion needs and transformation to higher risk disease or AML and improve survival. In higher risk, the goal is to prolong survival. Currently available therapies include growth factor support, lenalidomide, HMAs, intensive chemotherapy, and alloSCT. Approved in 2020 are luspatercept and the oral HMA ASTX727. At the present time, there are no approved interventions for patients with progressive or refractory disease, particularly after HMA based therapy. Options include participation in a clinical trial or cytarabine-based therapy or alloSCT.
Myelodysplastic Syndrome Epidemiology
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Incident Population of MDS, Age-specific Incident Population of MDS, Subtype-specific Incident Population of MDS, Risk-specific Incident Population of MDS, and Mutation-specific Incident Population of MDS in the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan.
Key Findings
This section provides glimpse of the MDS epidemiology in the 7MM.
- In 2020, the incident population of MDS in the 7MM was 41,586.
- Epidemiology assessed for MDS showed that the US, in 2020, accounted for approximately 20,428 incident cases of MDS.
- In the United States, in 2020, there were 240, 1,070, 3,654, 7,283 and 8,181 cases, in the age groups 0–49 years, 50–59 years, 60–69 years, 70–79 years, and 80+ years, respectively.
- As per the estimates, in the United States, there were 6,741, 1,021, 2,043, 6,333, 3,064, and 1,226 cases of RAEB/MDS-EB, del(5q) MDS, MDS-RS/RARS, MDS-MLD/RCMD, MDS-SLD/RCUD, and MDS-U subtypes in 2020, respectively.
- In 2020, there were 4,903, 2,043, 817, 2,043, 2,554, 1,839, 4,086, 2,860, 817, and 1,021 cases, for the mutation SF3B1, SRSF2, U2AF1, DNMT3A, RUNX1 TP53, TET2, ASXL1, ZRSR2, and IDH2 respectively, in the US.
- In the US, based on the IPSS scoring, Low-risk MDS and High-risk MDS accounted for the highest and lowest cases, respectively, in 2020. On the other hand, based on the IPSS-R scoring, Low-risk MDS and Very high-risk MDS accounted for the highest and lowest cases, respectively, in 2020.
- Among the EU-5 countries in 2020, Germany had the highest incident population of MDS patients with 4,515 cases, followed by France (4,371) and UK (3,302). In contrast, Spain had the lowest cases (2,010) in 2020.
- As per the estimates, Japan accounted for 3,909 incident MDS cases in 2020.
Country Wise- Myelodysplastic Syndrome Epidemiology
The epidemiology segment also provides the Myelodysplastic Syndrome epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
Myelodysplastic Syndrome Drug Chapters
The drug chapter segment of the Myelodysplastic Syndrome report encloses the detailed analysis of MDS mid and late-stage pipeline drugs. It also helps to understand the MDS clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug and the latest news and press releases.
Myelodysplastic Syndrome Marketed Drugs
Reblozyl: Bristol-Meyers Squibb
Reblozyl (luspatercept-aamt) is an erythroid maturation agent indicated for the treatment of anemia failing an erythropoiesis-stimulating agent and requiring two or more RBC units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). Bristol Myers Squibb and Acceleron Pharma are jointly developing Reblozyl as part of a global collaboration.
Product details in the report…
Inqovi: Astex Pharmaceutical/Taiho Oncology
Inqovi is a combination of decitabine (a nucleoside metabolic inhibitor) and cedazuridine (a cytidine deaminase inhibitor), indicated for the treatment of adult patients with MDS, including previously treated and untreated, de novo and secondary MDS.
Decitabine is a nucleoside metabolic inhibitor that is believed to exert its effects after phosphorylation and direct incorporation into DNA and inhibit DNA methyltransferase, causing hypomethylation of DNA and cellular differentiation and/or apoptosis.
The combination was approved in July 2020 by the US FDA. Approval was based on data from the ASCERTAIN Phase III study and supporting Phase I and II clinical studies. The ASCERTAIN Phase III study evaluated the 5-day, decitabine exposure equivalence between oral Inqovi and intravenous decitabine. The safety and efficacy of Inqovi were also assessed in the clinical studies.
Product details in the report…
Myelodysplastic Syndrome Emerging Drugs
Roxadustat: Fibrogen
Roxadustat (FG-4592) is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor. It increases hemoglobin (Hb) by mimicking the body’s natural response to low oxygen and is based on 2019 Nobel Prize-winning science. It is being developed for anemia associated with MDS.
The drug is also being developed for anemia associated with chronic kidney disease (CKD) in which is approved in Japan, China while NDA is submitted for the US and for chemotherapy-induced anemia (CIA), for which Phase II enrollment is completed.
Product details in the report…
Venclexta (Venetoclax) + Azacitidine: AbbVie
Venclexta (Venetoclax) is a first-in-class targeted medicine designed to selectively bind and inhibit the B-cell lymphoma-2 (BCL-2) protein. BCL-2 builds up and prevents cancer cells from dying or self-destructing, a process called apoptosis. Venetoclax blocks the BCL-2 protein and works to restore the process of apoptosis. It is being developed by AbbVie and Genentech. It is jointly commercialized by companies in the United States and by AbbVie outside of the United States. Venetoclax is already approved by brand name Venclexta for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) and in combination with azacitidine or decitabine or low-dose cytarabine for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adults.
Product details in the report…
Magrolimab ± Azacitidine: Gilead Sciences
Magrolimab is a first-in-class investigational monoclonal antibody against CD47 and macrophage checkpoint inhibitor designed to interfere with recognition of CD47 by the SIRPα receptor on macrophages, thus blocking the “don't eat me” signal used by cancer cells to avoid being ingested by macrophages. Azacitidine (brand name Vidaza) works together with magrolimab by inducing “eat me” signals on leukemic blasts and enhancing phagocytosis. Magrolimab, in combination with Vidaza, is clinically effective in both AML and MDS. In September 2020, FDA granted Breakthrough Therapy designation for magrolimab based on positive results of an ongoing Phase Ib study.
Product details in the report…
SY-1425 + Azacitidine: Syros pharmaceuticals
Tamibarotene (formerly SY-1425) is an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist that is being developed for genomically defined subsets of patients whose disease is characterized by the overexpression of the RARA gene. The company has initiated a Phase III trial in the newly diagnosed high-risk MDS patients in combination with azacitidine and expecting a potential NDA filing by 2024.
Product details in the report…
Pevonedistat ± Azacitidine: Takeda
Pevonedistat is the first-in-class small-molecule inhibitor of the neural precursor cell expressed, developmentally downregulated 8 (NEDD8)-activating enzyme (NAE); NAE facilitates conjugation of the small ubiquitin-like protein, NEDD8, which activates cullin-RING E3 ubiquitin ligases (CRLs). Inhibition of NAE by pevonedistat prevents degradation of CRL substrates integral to tumor cell growth, proliferation, and survival, leading to cancer cell death.
Product details in the report…
Glasdegib + Azacitidine: Pfizer
Glasdegib (PF-04449913), an oral inhibitor, is being evaluated for hematologic malignancies and solid tumors. It inhibits the smoothened (SMO) receptor, thereby disrupting the Hedgehog (Hh) pathway. SMO inhibition of Hh signaling disrupts the regulation of cancer stem cell (CSC) survival; this may inhibit the development of drug resistance and prevent relapse. Currently, the combination is being evaluated in a Phase III clinical study.
Product details in the report…
Myelodysplastic Syndrome Market Outlook
The current treatment landscape for patients with MDS comprises allo-HSCT, non-intensive therapies ranging from iron chelation and growth factors to lenalidomide and HMA. Allo-HSCT remains the only curative for this patient pool. Other approaches are non-curative, aimed at improving quality of life and delaying disease progression.
For chemotherapy, drugs such as azacitidine (Vidaza) or decitabine (Dacogen) are often the first choice, especially for patients with lower-risk forms of MDS. These drugs can often improve blood counts, and many patients need fewer transfusions and have a better quality of life, with less fatigue. These drugs can also help some people live longer. Another option for some people might be medicines to suppress the immune system, such as ATG and cyclosporine. However, for MDS del(5q) patients, lenalidomide (Revlimid), which is FDA-approved for this indication, is often used as the first treatment.
Another FDA-approved drug for supportive care for MDS is Reblozyl. Approved by the FDA in 2020, it is an erythroid maturation agent indicated for second-line treatment of MDS in patients previously treated with ESA. Bristol-Myers Squibb is anticipating getting approval for MDS patients previously untreated with ESA (ESA naïve), i.e., in the first-line setting for MDS.
The decitabine and cedazuridine combination drug, Inqovi was approved by the FDA in July 2020 for the treatment of MDS patients, including previously treated and untreated, de novo, and secondary MDS.
Other than these current therapies, the MDS pipeline is robust and possesses multiple potential drugs in the late, mid, and early stages of developments, yet to be launched. The emerging pipeline of MDS possesses several potential products developed in different stages of clinical development. Expected Launch of potential therapies, namely FibroGen (roxadustat), AbbVie (Venclexta ± azacitidine), Novartis (sabatolimab), Syros Pharmaceuticals (SY-1425 + azacitidine), Gilead Sciences (magrolimab ± azacitidine), Geron Corporation (imetelstat), Takeda (pevonedistat ± azacitidine), Pfizer (glasdegib + azacitidine), Aprea Therapeutics (eprenetapopt), Jazz Pharmaceuticals (CPX-351), Karyopharm Therapeutics (eltanexor), BerGenBio (bemcentinib), Bristol Myers Squibb (enasidenib), Sanofi (fludarabine + busulfan ± clofarabine and Medac (treosulfan ± fludarabine). The launch of these therapies may increase market size in the coming years, assisted by an increase in the MDS patient pool.
Key Findings
This section includes a glimpse of the MDS 7MM market.
- The total market size is calculated by including the market size of both emerging and current therapies. The total market size in the 7MM for MDS was estimated to be USD 1,924 million in 2020 during the study period 2018–2030.
- The United States accounted for the largest market size of MDS in comparison to the EU5 (Germany, France, Italy, the United Kingdom, and Spain) and Japan. In the United States, the market size of MDS is USD 1,301.5 million in 2020.
- Among the EU5 countries, Germany had the highest market size with USD 135.2 million in 2020, while Spain had the smallest market size of MDS with USD 60.2 million in 2020.
- In 2020, Japan accounted for market size of MDS with USD 105.8 million.
The United States Market Outlook
This section provides the total Myelodysplastic Syndrome market size and market size by therapies in the United States.
EU-5 Market Outlook
The total Myelodysplastic Syndrome market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.
Japan Market Outlook
The total Myelodysplastic Syndrome market size and market size by therapies in Japan are provided.
Myelodysplastic Syndrome Drugs Uptake
This section focuses on the rate of uptake of the potential drugs recently launched in the MDS market or expected to get launched in the market during the study period 2018–2030. The analysis covers MDS market uptake by drugs; patient uptake by therapies; and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows the comparison of the drugs based on market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
Myelodysplastic Syndrome Development Activities
The report provides insights into different therapeutic candidates in phase II, and phase III stages. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for MDS emerging therapies.
Competitive Intelligence Analysis
We perform competitive and market Intelligence analysis of the Myelodysplastic Syndrome market by using various competitive intelligence tools that include–SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.
Scope of the Report
- The report covers the descriptive overview of MDS, explaining its causes, signs and symptoms, pathogenesis and currently available therapies.
- Comprehensive insight has been provided into the MDS epidemiology and treatment.
- Additionally, an all-inclusive account of both the current and emerging therapies for MDS are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
- A detailed review of the MDS; historical and forecasted is included in the report, covering the 7MM drug outreach.
- The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MM MDS Injury market.
Report Highlights
- In the coming years, the MDS market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
- The companies and academics are working to assess challenges and seek opportunities that could influence MDS R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
- Delvelnsight has analysed incident population of MDS in the 7MM which suggests that the cases of MDS will increase during the forecast period of 2021–2030.
- Age-specific data of MDS suggests that incidence of MDS in the US, was highest in the age group of 80+ years, followed by 70–79 years and 60–69 years.
- The scope of the report also encompasses another major segment, i.e., mutation-specific incidence MDS, wherein the number of patients suffering from mutations of SF3B1, SRSF2, U2AF1, DNMT3A, RUNX1 TP53, TET2, ASXL1, ZRSR2, and IDH2 were calculated.
- There are sub- types of MDS considered in this report include MDS can be broadly categorized into the following subtypes: RAEB/MDS-EB, del(5q) MDS, MDS-RS/RARS, MDS-MLD/RCMD, MDS-SLD/RCUD, and MDS-U.
- Currently available therapies include growth factor support, lenalidomide, Reblozyl, HMAs, intensive chemotherapy, and alloSCT along with the drugs approved in 2020, luspatercept and the oral HMA ASTX727.
- Expected Launch of potential therapies by key players like , FibroGen (roxadustat), AbbVie (Venclexta ± azacitidine), Novartis (sabatolimab), Syros Pharmaceuticals (SY-1425 + azacitidine), Gilead Sciences (magrolimab ± azacitidine), Geron Corporation (imetelstat), Takeda (pevonedistat ± azacitidine), Pfizer (glasdegib + azacitidine), Aprea Therapeutics (eprenetapopt), Jazz Pharmaceuticals (CPX-351), Karyopharm Therapeutics (eltanexor), BerGenBio (bemcentinib), Bristol Myers Squibb (enasidenib), Sanofi (fludarabine + busulfan ± clofarabine), Medac (treosulfan ± fludarabine) and others may increase the market size in the coming years, assisted by an increase in the incident population of Myelodysplastic Syndrome (MDS). It is expected that these therapies will help thrive the market of Myelodysplastic Syndrome (MDS) post-launch, in the United States. Furthermore, these novel therapies will mark there entry in the EU5 countries and Japan in subsequent years.
Analyst Comments
- Companies like Bristol Myers Squibb, Aprea Therapeutics, Geron Corporation, and others are developing therapies targeting certain mutations like TP53, IDH2, RARA, etc. This will provide more precise treatment for MDS patients not responding or having poor outcomes toward the current standard of treatment.
- Various therapies for second-line settings are also being developed, which will provide more treatment options for the patients, as there are currently no approved second-line treatments for MDS.
- Therapies like imetelstat, treosulfan, and SY-1425 + azacitidine, are expected to enter the market sooner than the other therapies, as their respective companies are planning to file for NDA soon.
Myelodysplastic Syndrome Report Insights
- Patient Population
- Therapeutic Approaches
- Myelodysplastic Syndrome Pipeline Analysis
- Myelodysplastic Syndrome Market Size and Trends
- Market Opportunities
- Impact of upcoming Therapies
Myelodysplastic Syndrome Report Key Strengths
- Ten Years Forecast
- 7MM Coverage
- Myelodysplastic Syndrome Epidemiology Segmentation
- Key Cross Competition
- Highly Analyzed Market
- Drugs Uptake
Myelodysplastic Syndrome Report Assessment
- Current Treatment Practices
- Unmet Needs
- Pipeline Product Profiles
- Market Attractiveness
- Market Drivers and Barriers
Key Questions
Market Insights:
- What was the Myelodysplastic Syndrome market share (%) distribution in 2018 and how it would look like in 2030?
- What would be the Myelodysplastic Syndrome total market size as well as market size by therapies across the 7MM during the forecast period (2021–2030)?
- What are the key findings pertaining to the market across the 7MM
- Which country will have the largest Myelodysplastic Syndrome market size during the forecast period (2021–2030)?
- At what CAGR, the Myelodysplastic Syndrome market is expected to grow in the 7MM during the forecast period (2021–2030)?
- What would be the Myelodysplastic Syndrome market outlook across the 7MM during the forecast period (2021–2030)?
- What would be the Myelodysplastic Syndrome market growth till 2030 and what will be the resultant market size in the year 2030?
- How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
Epidemiology Insights:
- What is the disease risk, burden and unmet needs of Myelodysplastic Syndrome?
- What is the historical Myelodysplastic Syndrome patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK) and Japan?
- What would be the forecasted patient pool of Myelodysplastic Syndrome at the 7MM?
- What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Myelodysplastic Syndrome?
- Out of the above-mentioned countries, which country would have the highest incident population of Myelodysplastic Syndrome during the forecast period (2021–2030)?
- At what CAGR the population is expected to grow across the 7MM during the forecast period (2021–2030)?
Current Treatment Scenario, Marketed Drugs and Emerging Therapies:
- What are the current options for the treatment of Myelodysplastic Syndrome along with the approved therapy?
- What are the current treatment guidelines for the treatment of Myelodysplastic Syndrome in the US, Europe and Japan?
- What are the Myelodysplastic Syndrome marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
- How many companies are developing therapies for the treatment of Myelodysplastic Syndrome?
- How many therapies are developed by each company for the treatment of Myelodysplastic Syndrome?
- How many emerging therapies are in the mid-stage and late stage of development for the treatment of Myelodysplastic Syndrome?
- What are the key collaborations (Industry–Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Myelodysplastic Syndrome therapies?
- What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Myelodysplastic Syndrome and their status?
- What are the key designations that have been granted for the emerging therapies for Myelodysplastic Syndrome?
- What are the 7MM historical and forecasted market of Myelodysplastic Syndrome?
Reasons to buy
- The report will help in developing business strategies by understanding trends shaping and driving Myelodysplastic Syndrome.
- To understand the future market competition in the Myelodysplastic Syndrome market and an Insightful review of the key market drivers and barriers.
- Organize sales and marketing efforts by identifying the best opportunities for Myelodysplastic Syndrome in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), And Japan.
- Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
- Organize sales and marketing efforts by identifying the best opportunities for the Myelodysplastic Syndrome market.
- To understand the future market competition in the Myelodysplastic Syndrome market.
1. Key Insights
2. Report Introduction
3. Myelodysplastic Syndrome (MDS) Market Overview at a Glance
3.1. Market Size Distribution by Therapies in 2018 (%)
3.2. Market Size Distribution by Therapies in 2030 (%)
4. Key Events
5. SWOT Analysis
6. Disease Background and Overview
6.1. Introduction
6.2. Classification
6.2.1. WHO Classification System (2016)
6.2.2. French American British (FAB) Classification System
6.3. Sign and Symptoms
6.3.1. Low Red Blood Cell Counts
6.3.2. Low White Blood
6.3.3. Low Platelet Count
6.4. Causes and Risk factors
6.5. Prevention
6.6. Clinical Presentation
6.7. Risk Stratification
6.7.1. International Prognostic Scoring System (IPSS)
6.7.2. Revised International Prognostic Scoring System (IPSS-R)
6.8. Pathophysiology
6.8.1. Haploinsufficiency through Chromosomal Deletion
6.8.2. Recurrently Mutated Genes
6.8.3. Pre-MDS Clonal Mutations
6.8.4. Post-MDS Clonal Mutations
6.8.5. Epigenetic Changes
6.8.6. Ineffective Hematopoiesis
6.8.7. Microenvironment and Immune Cells
6.9. Prognosis
6.10. Mutations
6.11. Differential Diagnosis
6.12. Diagnosis
6.12.1. History and Physical Examination
6.12.2. Blood Examination
6.12.3. Bone Marrow Examination
6.12.4. Cytochemistry and Immunocytochemistry
6.12.5. Cytogenetic and molecular features
7. Diagnostic Algorithm
8. British Society for Hematology guidelines for the Diagnosis and Evaluation of Prognosis of Adult MDS (2021)
9. Treatment
9.1. Risk adapted therapy
9.1.1. Treatment of ICUS/CHIP/CCUS
9.1.2. Options for lower-risk MDS
9.1.3. Options for high-risk MDS
10. National Comprehensive Cancer Network recommendations for myelodysplastic syndrome (MDS)
10.1. Recommendations for Supportive care
10.2. Recommended Treatment Approaches for Lower-Risk Patients (IPSS Low, Intermediate-1; IPSS-R Very Low, Low, Intermediate; or WPSS Very Low, Low, Intermediate)
10.3. Recommended Treatment Approaches for Higher-Risk Patients (IPSS Intermediate-2, High; IPSS-R Intermediate, High, Very High; or WPSS High, Very High)
10.3.1. Intensive Therapy
10.3.2. Non-intensive Therapy
10.3.3. Supportive Care Only
11. Myelodysplastic Syndromes: ESMO Clinical Practice Guidelines for Diagnosis, Treatment, and Follow-up (2020)
11.1. Recommendations for Diagnosis of MDS
11.2. Recommendations for Staging and Risk Assessment
11.3. Recommendations for Treatment
12. JSH Practical Guidelines for MDS (2018)
13. British Society for Hematology Guidelines for the Management of Adult Myelodysplastic Syndromes (2021)
13.1. Recommendations on Supportive Care for MDS
13.2. Recommendations on Management of Low-risk MDS
13.3. Recommendation for Management of MDS Associated with Del(5q)
13.4. Recommendations on Management of High-risk MDS
13.5. Recommendations for Allogeneic Hematopoietic Stem Cell Transplant in MDS
14. Treatment Algorithm
15. Epidemiology and Patient Population
15.1. Key Findings
15.2. Epidemiology Methodology
15.3. Total Incident Population of Myelodysplastic Syndrome (MDS) in the 7MM
15.4. Epidemiology of Myelodysplastic Syndrome (MDS)
15.4.1. US
15.4.2. EU-5
15.4.3. Japan
15.5. The United States
15.5.1. Total Incident Population of Myelodysplastic Syndrome (MDS) in the United States
15.5.2. Age-specific Incident Population of Myelodysplastic Syndrome (MDS) in the United States
15.5.3. Subtype-specific Incident Population of Myelodysplastic Syndrome (MDS) in the United States
15.5.4. Risk-specific incident population of Myelodysplastic Syndrome (MDS) in the United States
15.5.5. Mutation-specific incident population of Myelodysplastic Syndrome (MDS) in the United States
15.6. EU5
15.6.1. Total Incident Population of Myelodysplastic Syndrome (MDS) in EU-5
15.6.2. Age-specific Incident Population of Myelodysplastic syndrome (MDS) in EU-5
15.6.3. Subtype-specific Incident Population of Myelodysplastic syndrome (MDS) in EU-5
15.6.4. Risk-specific incident population of Myelodysplastic syndrome (MDS) in EU5
15.6.5. Mutation-specific incident population of Myelodysplastic syndrome (MDS) in EU5
15.7. Japan
15.7.1. Total Incident Population of Myelodysplastic Syndrome (MDS) in Japan
15.7.2. Age-specific Incident Population of Myelodysplastic Syndrome (MDS) in Japan
15.7.3. Subtype-specific Incident Population of Myelodysplastic Syndrome (MDS) in Japan
15.7.4. Risk-specific incident population of Myelodysplastic syndrome (MDS) in Japan
15.7.5. Mutation-specific incident population of Myelodysplastic syndrome (MDS) in Japan
16. Organizations contributing towards Myelodysplastic Syndrome (MDS)
17. Patient Journey
19. Marketed Drugs
19.1. Reblozyl: Bristol-Myers Squibb
19.1.1. Product Description
19.1.2. Clinical Development
19.1.2.1. Clinical Trials Information
19.1.3. Safety and Efficacy
19.2. Inqovi (decitabine and cedazuridine): Astex Pharmaceutical/Taiho Oncology
19.2.1. Product Description
19.2.2. Clinical Development
19.2.2.1. Clinical Trials Information
19.2.3. Safety and Efficacy
20. Emerging Therapies
20.1. Key Competitors
20.2. Roxadustat: Fibrogen
20.2.1. Product Description
20.2.2. Clinical Development
20.2.2.1. Clinical Trials Information
20.2.3. Safety and Efficacy
20.3. Venclexta (Venetoclax) + Azacitidine: AbbVie
20.3.1. Product Description
20.3.2. Clinical Development
20.3.2.1. Clinical Trials Information
20.3.3. Safety and Efficacy
20.4. Magrolimab ± Azacitidine: Gilead Sciences
20.4.1. Product Description
20.4.2. Clinical Development
20.4.2.1. Clinical Trials Information
20.4.3. Safety and Efficacy
20.5. Sabatolimab + Azacitidine: Novartis
20.5.1. Product Description
20.5.2. Clinical Development
20.5.2.1. Clinical Trials Information
20.5.3. Safety and Efficacy
20.6. SY-1425 + Azacitidine: Syros Pharmaceuticals
20.6.1. Product Description
20.6.2. Clinical Development
20.6.2.1. Clinical Trials Information
20.6.3. Safety and Efficacy
20.7. Pevonedistat ± Azacitidine: Takeda
20.7.1. Product Description
20.7.2. Clinical Development
20.7.2.1. Clinical Trials Information
20.7.3. Safety and Efficacy
20.8. Glasdegib + Azacitidine/Cytarabine: Pfizer
20.8.1. Product Description
20.8.2. Clinical Development
20.8.2.1. Clinical Trials Information
20.8.3. Safety and Efficacy
20.9. Imetelstat: Geron Corporation
20.9.1. Product Description
20.9.2. Clinical Development
20.9.2.1. Clinical Trials Information
20.9.3. Safety and Efficacy
20.10. Eltanexor (ATG 016; KPT-8602): Karyopharm Therapeutics/Antengene Corporation
20.10.1. Product Description
20.10.2. Clinical Development
20.10.2.1. Clinical Trials Information
20.10.3. Safety and Efficacy
20.11. Bemcentinib: BerGenBio ASA
20.11.1. Product Description
20.11.2. Clinical Development
20.11.2.1. Clinical Trials Information
20.11.3. Safety and Efficacy
20.12. Enasidenib: Bristol Myers Squibb
20.12.1. Product Description
20.12.2. Clinical Development
20.12.2.1. Clinical Trials Information
20.12.3. Safety and Efficacy
20.13. CPX-351: Jazz Pharmaceuticals
20.13.1. Product Description
20.13.2. Clinical Development
20.13.2.1. Clinical Trials Information
20.13.3. Safety and Efficacy
20.14. Eprenetapopt (APR-246): Aprea Therapeutics
20.14.1. Product Description
20.14.2. Clinical Development
20.14.2.1. Clinical Trials Information
20.14.3. Safety and Efficacy
20.15. Fludarabine + Busulfan ± Clofarabine: Sanofi
20.15.1. Product Description
20.15.2. Clinical Development
20.15.2.1 Clinical Trials Information
20.15.3. Safety and Efficacy
20.16. Treosulfan ± fludarabine: Medac
20.16.1. Product Description
20.16.2. Clinical Development
20.16.2.1. Clinical Trials Information
20.16.3. Safety and Efficacy
21. Potential of Current Therapies and Emerging Therapies
22. Myelodysplastic Syndrome (MDS): Seven Major Market Analysis
22.1. Key Findings
22.2. Market Methodology
22.3. Total Market Size of Myelodysplastic Syndrome (MDS) in the 7MM
22.4. Market Size of Myelodysplastic Syndrome (MDS) by First Line Therapies in the 7MM
22.5. Market Size of Myelodysplastic Syndrome (MDS) by Emerging Therapies in the 7MM
22.6. Market Outlook
22.7. Key Market Forecast Assumptions
22.8. The United States Market Size
22.8.1. Total Market Size of Myelodysplastic Syndrome (MDS) in the United States
22.8.2. Market Size of Myelodysplastic Syndrome (MDS) by First Line Therapies in the United States
22.8.3. Market Size of Myelodysplastic Syndrome (MDS) by Second Line Therapies in the United States
22.9. EU-5 Market Size
22.9.1. Total Market size of Myelodysplastic Syndrome (MDS) in Europe
22.9.2. Market Size of Myelodysplastic Syndrome (MDS) by First Line Therapies in Europe
22.9.3. Market Size of Myelodysplastic Syndrome (MDS) by Second Line Therapies in Europe
22.10. Japan
22.10.1. Total Market size of Myelodysplastic Syndrome (MDS) in Japan
22.10.2. Market Size of Myelodysplastic Syndrome (MDS) by Therapies in Japan
22.10.3. Market Size of Myelodysplastic Syndrome (MDS) by Second Line Therapies in Japan
23. KOL Views
24. Market Drivers
25. Market Barriers
26. Unmet Needs
27. Reimbursement and Market Access
28. Appendix
28.1. Bibliography
28.2. Report Methodology
29. DelveInsight Capabilities
30. Disclaimer
31. About DelveInsight
List of Table
Table 1: Summary of Myelodysplastic Syndrome (MDS) Market, and Epidemiology (2018–2030)
Table 2: Key Events
Table 3: Total Incident Population of MDS in the 7MM (2018–2030)
Table 4: Total Incident Population of MDS in the US (2018–2030)
Table 5: Age-specific Incident Population of MDS in the United States (2018–2030)
Table 6: Subtype-specific Incident Population of MDS in the US (2018–2030)
Table 7: Risk-specific incident population of MDS in the United States (2018–2030)
Table 8: Mutation-specific incident population of MDS in the United States (2018–2030)
Table 9: Total Incidence of MDS in EU-5 (2018–2030)
Table 10: Age-specific Incident Population of MDS in EU-5 (2018–2030)
Table 11: Subtype-specific Incident Population of MDS in EU-5 (2018–2030)
Table 12: Risk-specific incident population of MDS in EU5 (2018–2030)
Table 13: Mutation-specific incident population of MDS in EU5 (2018–2030)
Table 14: Total Incident Population of MDS in Japan (2018–2030)
Table 15: Age-specific Incident Population of MDS in Japan (2018–2030)
Table 16: Subtype-specific Incident Population of MDS in Japan (2018–2030)
Table 17: Risk-specific incident population of MDS in Japan (2018–2030)
Table 18: Mutation-specific incident population of MDS in Japan (2018–2030)
Table 19: Organizations Contributing Toward the Fight Against Myelodysplastic Syndrome
Table 20: Reblozyl, Clinical Trial Description, 2021
Table 21: Inqovi, Clinical Trial Description, 2021
Table 22: Comparison of emerging drugs under development
Table 23: Comparison of emerging drugs under development
Table 24: Roxadustat (FG-4592), Clinical Trial Description, 2021
Table 25: Venclexta (Venetoclax) + Azacitidine, Clinical Trial Description, 2021
Table 26: Magrolimab ± Azacitidine, Clinical Trial Description, 2021
Table 27: Sabatolimab (MBG453) + Azacitidine), Clinical Trial Description, 2021
Table 28: SY-1425 + Azacitidine, Clinical Trial Description, 2021
Table 29: Pevonedistat + Azacitidine, Clinical Trial Description, 2021
Table 30: Glasdegib + Azacitidine, Clinical Trial Description, 2021
Table 31: Imetelstat, Clinical Trial Description, 2021
Table 32: Eltanexor, Clinical Trial Description, 2021
Table 33: Bemcentinib, Clinical Trial Description, 2021
Table 34: Enasidenib, Clinical Trial Description, 2021
Table 35: CPX-351, Clinical Trial Description, 2021
Table 36: APR-246 + Azacitidine, Clinical Trial Description, 2021
Table 37: Fludarabine + Busulfan ± Clofarabine, Clinical Trial Description, 2021
Table 38: Treosulfan ± fludarabine, Clinical Trial Description, 2021
Table 39: Market Size of MDS in the 7MM in USD Million (2018–2030)
Table 40: Market Size of MDS by First Line therapies in the 7MM, in USD Million (2018–2030)
Table 41: Market Size of MDS by Second Line therapies in the 7MM, in USD Million (2018–2030)
Table 42: Key Market Forecast Assumptions for FG-4592 (Roxadustat) (for 1LOT)
Table 43: Key Market Forecast Assumptions for Venclexta ± Azacitidine (for 1LOT)
Table 44: Key Market Forecast Assumptions for Magrolimab +Azacitidine (for 1LOT)
Table 45: Key Market Forecast Assumptions for Pevonedistat ± Azacitidine (for 1LOT)
Table 46: Key Market Forecast Assumptions for SY-1425 + Azacitidine (for 1LOT)
Table 47: Key Market Forecast Assumptions for Glasdegib + Azacitidine (for 1LOT)
Table 48: Key Market Forecast Assumptions for Imetelstat (for 2LOT)
Table 49: Key Market Forecast Assumptions for Bemcentinib (for 2LOT)
Table 50: Key Market Forecast Assumptions for Eltanexor (ATG 016/KPT-8602) (for 2LOT)
Table 51: Key Market Forecast Assumptions for Enasidenib + Azacitidine (for 2LOT)
Table 52: Key Market Forecast Assumptions for CPX-351 (for 2LOT)
Table 53: Key Market Forecast Assumptions for Eprenetapopt
Table 54: Market Size of MDS in the US, USD Million (2018–2030)
Table 55: Market Size of MDS by First Line therapies in the US, USD Million (2018–2030)
Table 56: Market Size of MDS by therapies in the US, USD Million (2018–2030)
Table 57: EU5 Market Size of MDS in USD Million (2018–2030)
Table 58: Market Size of MDS by First Line Therapies in EU5, USD Million (2018–2030)
Table 59: EU5 Market Size of MDS by Second Line Therapies in USD Million (2018–2030)
Table 60: Japan Market Size of MDS in Japan, USD Million (2018–2030)
Table 61: Market Size of MDS by First Line Therapies in Japan, USD Million (2018–2030)
Table 62: Market Size of MDS by Second Line Therapies in Japan, USD Million (2018–2030)
List of Figures
Figure 1: Total Incident Population of MDS in the 7MM (2018–2030)
Figure 2: Total Incident Population of MDS in the US (2018–2030)
Figure 3: Age-specific Incident Population of MDS in the United States (2018–2030)
Figure 4: Subtype-specific Incident Population of MDS in the US (2018–2030)
Figure 6: IPSS-R Risk-specific incident population of MDS in the United States (2018–2030)
Figure 5: IPSS Risk-specific incident population of MDS in the United States (2018–2030)
Figure 7: Mutation-specific incident population of MDS in the United States (2018–2030)
Figure 8: Total Incident Population of MDS in EU5 (2018–2030)
Figure 9: Age-specific Incident Population of MDS in EU-5 (2018–2030)
Figure 10: Subtype-specific Incident Population of MDS in EU-5 (2018–2030)
Figure 11: IPSS-R Risk-specific incident population of MDS in EU5 (2018–2030)
Figure 12: IPSS Risk-specific incident population of MDS in EU5 (2018–2030)
Figure 13: Mutation-specific incident population of MDS in EU-5 (2018–2030)
Figure 14: Total Incident Population of MDS in Japan (2018–2030)
Figure 15: Age-specific Incident Population of MDS in Japan (2018–2030)
Figure 16: Subtype-specific Incident Population of MDS in Japan (2018–2030)
Figure 17: IPSS-R Risk-specific incident population of MDS in Japan (2018–2030)
Figure 18: IPSS Risk-specific incident population of MDS in Japan (2018–2030)
Figure 19: Mutation-specific incident population of MDS in Japan (2018–2030)
Figure 20: Market Size of MDS in the 7MM in USD Million (2018–2030)
Figure 21: Market Size of MDS by First Line Therapies in the 7MM, in USD Million (2018–2030)
Figure 22: Market Size of MDS by Second Line Therapies in the 7MM, in USD Million (2018–2030)
Figure 22: Market Size of MDS in the US, USD Millions (2018–2030)
Figure 23: Market Size of MDS by First Line therapies in the US, USD Million (2018–2030)
Figure 24: Market Size of MDS by therapies in the US, USD Million (2018–2030)
Figure 25: Market Size of MDS in EU5, USD Million (2018–2030)
Figure 26: EU5 Market Size of MDS by First Line Therapies in USD Million (2018–2030)
Figure 27: EU5 Market Size of MDS by Second Line Therapies in USD Million (2018–2030)
Figure 28: Market Size of MDS in Japan, USD Million (2018–2030)
Figure 29: Japan Market Size of MDS by First Line Therapies, USD Million (2018–2030)
Figure 30: Japan Market Size of MDS by Second Line Therapies, USD Million (2018–2030)
Bristol-Myers Squibb
Astex Pharmaceutical
Taiho Oncology
Fibrogen
AbbVie
Gilead Sciences
Novartis
Syros Pharmaceuticals
Takeda
Pfizer
Geron Corporation
Karyopharm Therapeutics
Antengene Corporation
BerGenBio ASA
Jazz Pharmaceuticals
Aprea Therapeutics
Sanofi
Medac