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Disease

Myelodysplastic Syndromes

Late-stage therapeutic developmentEmerging researchSteady momentum
MeSHMyelodysplastic Syndromes
4
Publications
20
Clinical trials
2
Related conditions
2019
Latest publication
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

Safety and Efficacy of Anti-CD123 Chimeric Antigen Receptor-Modified Autologous T Cells (CART123) in Patients With Relapsed/Refractory CD123+ Hematologic Malignancies: A Dose Escalation, Open-Label, Phase I Study

Clinical trial2026-09-14Status changed to Active, not recruiting · ClinicalTrials.gov

ABBA CORD: Double Umbilical Cord Blood Transplants With Abatacept for Graft Versus Host Disease Prophylaxis

Clinical trial2026-09-10Status changed to Suspended · ClinicalTrials.gov

A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies

Clinical trial2026-08-20Status changed to Active, not recruiting · ClinicalTrials.gov

A Phase 1b Dose Escalation Study Evaluating the Safety and Pharmacokinetics of Venetoclax in Combination With Azacitidine in Subjects With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

Clinical trial2026-08-12Status changed to Completed · ClinicalTrials.gov

A Phase 1, First-in-Human, Dose Escalation Study of MGD024, a CD123 x CD3 Bispecific DART Molecule, in Patients With Select Relapsed or Refractory Hematologic Malignancies

Clinical trial2026-07-28Status changed to Active, not recruiting · ClinicalTrials.gov

Marketing authorisation: Rytelo (EMA)

Regulatory2025-03-07EMA

Generic medicine authorisation: Azacitidine Kabi (EMA)

Regulatory2024-01-05EMA

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 5 clinical trials expected to report results, the earliest in Q4 2027.
  • 1 industry development reported.
Upcoming Milestones
Major developments
Upcoming trial readoutHigh impact
A Phase 3, Multicenter, Open Label, Randomized, Non-comparative Two-arm Study of Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Adult Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an Isocitrate Dehydrogenase-1 (IDH1) Mutation (PyramIDH Study)
Results expected Q4 20272026-09-03
Upcoming trial readoutHigh impact
A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate-Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-Stimulating Agent (ESA)-Naive Participants Who Are Non-Transfusion Dependent (NTD): The "ELEMENT-MDS" Trial
Results expected Q1 20282026-06-30
Regulatory developmentImportant
Biosimilar approval — Pegfilgrastim
Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).2026-09-25
Clinical Milestones7View
Results expected 5
Recently completed 1
Other updates 1
Industry & Market1View
Regulatory Updates1View
  • 2026-09-25Biosimilar approval — PegfilgrastimReduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).
Activity timeline9

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Pegfilgrastimapproved

Biosimilar approval — Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adul… (2026)

Imetelstatapproved

Original application approval — Treatment of transfusion-dependent anaemia in adults with low- to intermediate-1 risk mye… (2025)

Azacitidineapproved

Generic approval — Azacitidine Kabi is indicated for the treatment of adult patients who are not eligible fo… (2024)

Efbemalenograstim alfaapproved

Original application approval — Ryzneuta is indicated for the reduction in the duration of neutropenia and the incidence… (2024)

Lenalidomideapproved

Generic approval — Multiple myelomaLenalidomide Krka as monotherapy is indicated for the maintenance treatme… (2021)

Clinical trials

15 sponsors · 0 new · 1 completed in the last 12 months (net -1)

The current development programme across all trial phases.

Clinical programme
20
All trials
6
Active
11
Late-stage
6
Completed
Late-stage studies

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) (RENEW)

Phase 3 · Recruiting · Takeda

A Phase 3, Multicenter, Open Label, Randomized, Non-comparative Two-arm Study of Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Adult Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an Isocitrate Dehydrogenase-1 (IDH1) Mutation (PyramIDH Study)

Phase 3 · Recruiting · Institut de Recherches Internationales Servier

A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate-Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-Stimulating Agent (ESA)-Naive Participants Who Are Non-Transfusion Dependent (NTD): The "ELEMENT-MDS" Trial

Phase 3 · Active, not recruiting · Bristol-Myers Squibb

A Randomized Trial Testing Early vs Late Onset of EPO Alfa Treatment in Lower Risk MDS With Non RBC Transfusion Dependent Anemia and Without Del 5q

Phase 3 · Active, not recruiting · Groupe Francophone des Myelodysplasies

A Phase 2/3, Multicenter, Randomized, Dose Optimization (Part I), Double-blind (Part II) Study to Compare the Efficacy and Safety of Oral Azacitidine (Oral-Aza, ONUREG®) Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With IPSS-R Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)

Phase 2/3 · Active, not recruiting · Bristol-Myers Squibb

Efficiency of Antibacterial Prophylaxis (Levofloxacin) in Azacitidine Treated Patients

Phase 3 · Completed · Centre Henri Becquerel
Recruiting

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) (RENEW)

Phase 3 · Recruiting · Takeda

A Phase 3, Multicenter, Open Label, Randomized, Non-comparative Two-arm Study of Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Adult Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an Isocitrate Dehydrogenase-1 (IDH1) Mutation (PyramIDH Study)

Phase 3 · Recruiting · Institut de Recherches Internationales Servier

A Phase 2 Trial of Interferon-γ (IFN-γ) in Combination With Donor Leukocyte Infusion (DLI) to Treat Relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS) After Allogeneic Hematopoietic Stem Cell Transplantation (alloSCT)

Phase 2 · Recruiting · Sawa Ito, MD
Recently completed

Phase I Trial of the Combination of Bortezomib and Clofarabine in Adults With Refractory Solid Tumors, Lymphomas, or Myelodysplastic Syndromes

Phase 1 · Completed · National Cancer Institute (NCI)

Efficiency of Antibacterial Prophylaxis (Levofloxacin) in Azacitidine Treated Patients

Phase 3 · Completed · Centre Henri Becquerel

A Phase 2, Multicenter, Single-Arm Bridging Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes(MDS) in Chinese and Japanese Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions

Phase 2 · Completed · Celgene

A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 in Patients Diagnosed With Low and INT-1 Risk Myelodysplastic Syndrome (MDS) and Transfusion-dependent Iron Overload

Phase 4 · Completed · Novartis Pharmaceuticals

Phase II Study of Thalidomide in the Treatment of Myelodysplastic Syndromes in Adults: A Clinical and Biologic Study

Phase 2 · Completed · National Cancer Institute (NCI)

A Randomized, Multi-Center, Double-Blind, Placebo-Controlled Trial Assessing The Safety And Efficacy Of Thalidomide (THALOMID) For The Treatment Of Anemia In Red Blood Cell Transfusion-Dependent Patients With Myelodysplastic Syndromes

Phase 2 · Completed · Roswell Park Cancer Institute

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

Product approvals and authorisations
2026emaBiosimilar approval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2025emaBiosimilar approval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2025emaBiosimilar approval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2025emaOriginal application approval — Imetelstat· Treatment of transfusion-dependent anaemia in adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have failed to respond or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA). source ↗
2024emaOriginal application approval — Efbemalenograstim alfa· Ryzneuta is indicated for the reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2024emaGeneric approval — Azacitidine· Azacitidine Kabi is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation(HSCT) with: Intermediate-2 and high-risk myelodysplastic syndromes (MDS) according to the international prognostic scoring system (IPSS), Chronic myelomonocytic leukaemia (CMML) with 10-29% marrow blasts without myeloproliferative disorder, Acute myeloid leukaemia (AML) with 20-30% blasts and multi-lineage dysplasia, according to World Health Organization (WHO) classification, AML with > 30% marrow blasts according to the WHO classification. source ↗
2021emaGeneric approval — Lenalidomide· Multiple myelomaLenalidomide Krka as monotherapy is indicated for the maintenance treatment of adult patients with newly diagnosed multiple myeloma who have undergone autologous stem cell transplantation. Lenalidomide Krka as combination therapy with dexamethasone, or bortezomib and dexamethasone, or melphalan and prednisone (see section 4.2) is indicated for the treatment of adult patients with previously untreated multiple myeloma who are not eligible for transplant. Lenalidomide Krka in combination with dexamethasone is indicated for the treatment of multiple myeloma in adult patients who have received at least one prior therapy. Myelodysplastic syndromesLenalidomide Krka as monotherapy is indicated for the treatment of adult patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.Mantle cell lymphomaLenalidomide Krka as monotherapy is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (see sections 4.4 and 5.1). Follicular lymphomaLenalidomide Krka in combination with rituximab (anti-CD20 antibody) is indicated for the treatment of adult patients with previously treated follicular lymphoma (Grade 1 – 3a). Multiple myelomaLenalidomide Krka as monotherapy is indicated for the maintenance treatment of adult patients with newly diagnosed multiple myeloma who have undergone autologous stem cell transplantation. Lenalidomide Krka as combination therapy with dexamethasone, or bortezomib and dexamethasone, or melphalan and prednisone (see section 4.2) is indicated for the treatment of adult patients with previously untreated multiple myeloma who are not eligible for transplant. Lenalidomide Krka in combination with dexamethasone is indicated for the treatment of multiple myeloma in adult patients who have received at least one prior therapy. Myelodysplastic syndromesLenalidomide Krka as monotherapy is indicated for the treatment of adult patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate. Mantle cell lymphomaLenalidomide Krka as monotherapy is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (see sections 4.4 and 5.1). Follicular lymphomaLenalidomide Krka in combination with rituximab (anti-CD20 antibody) is indicated for the treatment of adult patients with previously treated follicular lymphoma (Grade 1 – 3a). source ↗
2020emaBiosimilar approval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

4 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
2008–2019
Leading journals3
  • Blood2
  • Journal of clinical oncology : official journal of the American Society of Clinical Oncology1
  • The New England journal of medicine1
Leading researchers8
  • Arber DA2
  • Bloomfield CD2
  • Borowitz MJ2
  • Le Beau MM2
  • Thiele J2
  • Vardiman JW2
  • Bloom PP1
  • Brunning RD1
Affiliations (unnormalised)6
  • University of Chicago2
  • Comprehensive Cancer Center1
  • Erasmus Medical Center1
  • Institute of Pathology1
  • John Hopkins Medical Institutions1
  • Massachusetts General Hospital1

Related conditions

2 matches

Diseases frequently studied alongside this one. Number shows shared papers.

Eosinophilia2Myeloproliferative Disorders2

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Clonal hematopoietic stem cell disorders characterized by dysplasia in one or more hematopoietic cell lineages. They predominantly affect patients over 60, are considered preleukemic conditions, and have high probability of transformation into ACUTE MYELOID LEUKEMIA.

Identifiers
MeSHMyelodysplastic Syndromes
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.