Myelodysplastic Syndrome Therapeutics Market Size in the 7MM was ~USD 2,800 Million in 2023, is expected to grow by 2034 | DelveInsight

DelveInsight's "Myelodysplastic Syndrome Market Insight, Epidemiology, and Market Forecast - 2034" 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, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

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Key Takeaways from the Myelodysplastic Syndrome Market Report

* In January 2025:- Keros Therapeutics Inc.:- This study (KER-050-D301) is evaluating the efficacy and safety of elritercept (KER-050) versus placebo in adult participants with transfusion-dependent anemia with very low, low, or intermediate risk MDS, or more recently defined as myelodysplastic neoplasms, with or without ring sideroblasts. The study is divided into the Screening Period, Double-blind Treatment Period, Safety Follow-Up Period and Long-term Follow-up Period.
* In January 2025:- Bristol-Myers Squibb- The purpose of the study is to compare the efficacy and safety of Luspatercept vs epoetin alfa in the treatment of anemia in adults due to IPSS-R very low, low, intermediate-risk MDS in ESA-naive participants who are non-transfusion dependent (NTD).
* In the 7MM, the US accounted for the highest Myelodysplastic Syndrome incident cases in 2023, with around 21,000 cases; these numbers are expected to increase during the forecast period.
* Among the age-specific cases, the cases from the age group 80+ accounted for the highest incident cases in 2023 in the US, with nearly 40% of total cases.
* Among the subtype-specific cases, RAEB/MDS-EB accounted for the highest number in the US in 2023.
* Amongst EU4 and the UK, Germany accounted for the highest incident cases, followed by France, while Spain occupied the bottom of the ladder in 2023.
* The leading Myelodysplastic Syndrome Companies such as 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 and others.
* Promising Myelodysplastic Syndrome Therapies such as Deferasirox, Tebapivat, KER-050, Luspatercept, Epoetin Alfa , and others.

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Myelodysplastic Syndrome Epidemiology Segmentation in the 7MM

* Total Incident Population of Myelodysplastic Syndrome
* Age-specific Incident Population of Myelodysplastic Syndrome
* Subtype-specific Incident Population of Myelodysplastic Syndrome
* Risk-specific Incident Population of Myelodysplastic Syndrome
* Mutation-specific Incident Population of Myelodysplastic Syndrome

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Myelodysplastic Syndrome Marketed Drugs

* VIDAZA (azacitidine): Bristol Myers Squibb

VIDAZA is a pyrimidine nucleoside analog of cytidine. VIDAZA is believed to exert its antineoplastic effects by causing hypomethylation of DNA and direct cytotoxicity on abnormal hematopoietic cells in the bone marrow. The concentration of azacitidine required for maximum inhibition of DNA methylation in vitro does not cause major suppression of DNA synthesis. It was first approved by the US FDA in May 2004 for the Myelodysplastic Syndrome treatment.

* DACOGEN (decitabine): Johnson & Johnson

DACOGEN is a nucleoside metabolic inhibitor indicated for the treatment of adult patients with myelodysplastic syndromes including previously treated and untreated, de novo and secondary MDS. Decitabine-induced hypomethylation in neoplastic cells may restore normal function to genes that are critical for the control of cellular differentiation and proliferation. In rapidly dividing cells, the cytotoxicity of decitabine may also be attributed to the formation of covalent adducts between DNA methyltransferase and decitabine incorporated into DNA. Non-proliferating cells are relatively insensitive to decitabine. It was first approved by the US FDA in May 2006.

Myelodysplastic Syndrome Emerging Drugs

* Venetoclax: Abbvie

Venetoclax by Abbvie is a first-in-class targeted medicine designed to selectively bind and inhibit the B-cell lymphoma-2 (BCL-2) protein. Currently, the drug is being investigated in a Phase III trial to treat adult patients with newly diagnosed higher-risk Myelodysplastic Syndrome. Moreover, in July 2021, the combination was granted Breakthrough Therapy Designation in higher risk Myelodysplastic Syndrome.

* Tamibarotene: Syros Pharmaceuticals

Tamibarotene is an oral selective retinoic acid receptor alpha (RARA) agonist being developed for genomically defined subsets of patients whose disease is characterized by the overexpression of the RARA gene. Approximately 50% of Myelodysplastic Syndrome patients and 30% of AML patients have RARA overexpression. When RARA is expressed more than its tightly controlled natural ligand, cells in the bone marrow may not differentiate into healthy myeloid cells, which can lead to hematological malignancies. However, when oral tamibarotene is administered, tamibarotene binds to RAR, allowing for the restoration of gene expression and myeloid differentiation. It is currently being investigated in the Phase III SELECT-MDS-1 trial in newly diagnosed higher-risk myelodysplastic syndrome patients with RARA overexpression.

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Myelodysplastic Syndrome Drugs Market Insights

There is a vast pipeline of targeted agents under pre-clinical and clinical development, potentially able to modify the natural history of the diverse disease spectrum of MDS. The B-cell leukemia/lymphoma-2 (BCL-2) inhibitor, venetoclax is to date one of the most potent anti-apoptotic inhibitors, representing a novel promising agent. This small molecule blocks the binding of BH3 proteins to BCL-2, allowing the activation of BCL2 Antagonist/Killer/BCL2 associated X on the surface of mitochondria leading to cell death of cancer cells because of the release of cytochrome C through a mitochondrial outer membrane permeabilization (MOMP) process. IDH1 and IDH2 inhibitors have also recently shown an acceptable ORR in different clinical trials. FLT3 inhibitors represent another important potential group of targeted compounds in MDS patients harboring FLT3 mutations.

Myelodysplastic Syndrome Market Outlook

While for more than a decade the therapeutic scenario of Myelodysplastic Syndrome syndromes has been dominated by very few available regimens associated with dismal results, especially for patients with higher-risk disease and who failed HMAs, in the last 2 years there was a flow of an incredible variety of new targeted agents and investigational approaches that possibly will reach the clinical setting in short time. If currently available therapies still include HMAs, lenalidomide, growth factor support, chemotherapy, and allo-HSCT, there is now the possibility to choose among new investigational treatments rationally designed to exert targeted actions and increase the response rates in categories of patients that so far remained precision drug-orphans.

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Scope of the Myelodysplastic Syndrome Market Report

* Coverage- 7MM
* Study Period- 2020-2034
* Myelodysplastic Syndrome Companies- 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 and others.
* Myelodysplastic Syndrome Therapies- Deferasirox, Tebapivat, KER-050, Luspatercept, Epoetin Alfa , and others.
* Myelodysplastic Syndrome Therapeutic Assessment: Myelodysplastic Syndrome Current Marketed and Myelodysplastic Syndrome Emerging Therapies
* Myelodysplastic Syndrome Market Dynamics: Myelodysplastic Syndrome market drivers and Myelodysplastic Syndrome market barriers
* Myelodysplastic Syndrome Unmet Needs, KOL's views, Analyst's views, Myelodysplastic Syndrome Market Access and Reimbursement

Table of Content

1 Key Insights

2 Report Introduction

3 Executive Summary of Myelodysplastic Syndrome (MDS)

4 Myelodysplastic Syndrome (MDS) Market Overview at a Glance

5 Key Events

6 Epidemiology and Market Forecast Methodology

7 Disease Background and Overview

8 Diagnostic Algorithm

9 British Society for Hematology guidelines for the Diagnosis and Evaluation of Prognosis of Adult MDS

10 Treatment

11 National Comprehensive Cancer Network recommendations for myelodysplastic syndrome

12 Myelodysplastic Syndromes: ESMO Clinical Practice Guidelines for Diagnosis, Treatment, and Follow-up

13 JSH Practical Guidelines for MDS

14 British Society for Hematology Guidelines for the Management of Adult Myelodysplastic Syndromes

15 Treatment Algorithm

16 Epidemiology and Patient Population

17 Organizations contributing toward Myelodysplastic Syndrome

18 Patient Journey

19 Marketed Drugs

20 Emerging Therapies

21 Myelodysplastic Syndrome: Seven Major Market Analysis

22 Unmet Need

23 SWOT Analysis

24 KOL Views

25 Market Access and Reimbursement

26 Appendix

27 DelveInsight Capabilities

28 Disclaimer

29 About DelveInsight

About Us

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