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Disease

Myelodysplastic syndrome

Late-stage therapeutic development
Also known as MDS, MDS, unclassifiable, MDS-U, Myelodysplastic Syndromes+14 more

MDS, MDS, unclassifiable, MDS-U, Myelodysplastic Syndromes, dysmyelopoietic syndrome, hematopoeitic - myelodysplastic syndrome (MDS), myelodysplasia, myelodysplastic neoplasm, myelodysplastic syndrome, somatic, myelodysplastic syndrome, unclassifiable, myelodysplastic syndrome/neoplasm, myelodysplastic syndromes, oligoblastic leukaemia, oligoblastic leukemia, preleukemia, smoldering leukemia, smouldering leukaemia, myelodysplastic syndrome, susceptibility to.

23
Clinical trials
12
Associated genes
12
Related proteins
Current focus
Cereblon biologyErythropoietin biologyTherapeutic development
Latest activity
beta

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

Phase I Multicenter, Open-Label, First-in-Human Study of BMS-986497 (ORM-6151) as a Monotherapy, in Double Combination With Azacitidine and in Triple Combination With Azacitidine and Venetoclax in Subjects With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Clinical trial2026-10-01Status changed to Terminated · ClinicalTrials.gov

A Phase 1/2a Multicenter Ascending Dose Study to Evaluate the Safety of HA-1 Minor Histocompatibility Antigen-Reactive TCR-Modified T Cells (BSB-1001) in Patients Undergoing HLA-Matched Allogenic Hematopoietic Stem Cell Transplant for AML, ALL or MDS

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

Primary Comparison of Liposomal Anthracycline Based Treatment Versus Conventional Care Strategies Before Allogeneic Stem Cell Transplantation in Patients With Higher Risk MDS and Oligoblastic AML

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

Administration of Donor Derived Multi-Tumor-Associated Antigen (TAA)- Specific T Cells to Patients With AML or MDS (ADSPAM)

Clinical trial2026-08-26Status changed to Completed · 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

Decoy-Resistant Interleukin-18 (DR-18) for Relapse, Pre-emptive Treatment or Prophylaxis After Allogeneic Hematopoietic Cell Transplantation in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome (DR. DREAM)

Clinical trial2026-08-10Status changed to Suspended · ClinicalTrials.gov

A Phase II Randomized Study to Assess Outcomes With Treosulfan-Based Versus Clofarabine-Based Conditioning in Patients With Myelodysplastic Syndromes With Excess Blasts (MDS-EB), or Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Cell Transplantation (HCT)

Clinical trial2026-08-06Status changed to Terminated · ClinicalTrials.gov

A Pilot Study of Liposomal Cytarabine and Daunorubicin (CPX-351) in Combination With Gemtuzumab Ozogamicin (GO) in Relapsed Refractory Patients With Acute Myeloid Leukemia (AML) and Post-Hypomethylating Agent (Post-HMA) Failure High-Risk Myelodysplastic Syndrome (HR-MDS)

Clinical trial2026-07-24Status changed to Completed · ClinicalTrials.gov

A Randomized Phase II Study Evaluating the Efficacy of Hyperbaric Oxygen in Improving Engraftment in Umbilical Cord Blood Stem Cell Transplantation

Clinical trial2026-07-20Status changed to Active, not recruiting · ClinicalTrials.gov

Haploidentical Allogeneic Peripheral Blood Transplantation: Clinical Trial and Laboratory Correlates Examining Checkpoint Immune Regulators' Expression

Clinical trial2026-07-17Status changed to Completed · ClinicalTrials.gov

Phase 1b Study of Pembrolizumab, Decitabine +/- Venetoclax Combination Therapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Clinical trial2026-07-14Status changed to Suspended · ClinicalTrials.gov

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
  • 2 clinical trials expected to report results, the earliest in Q1 2028.
  • 1 industry development reported.
Upcoming Milestones
Major developments
Major trial resultsHigh impact
A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study of AG-120 in Combination With Azacitidine in Subjects ≥ 18 Years of Age With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation
Results posted2026-06-29
Major trial resultsHigh impact
A Phase III Randomized Study of Farnesyl Transferase Inhibitor R115777 in Acute Myeloid Leukemia (AML) Patients in Second or Subsequent Remission or in Remission After Primary Induction Failure or Patients Over Age 60 in First Remission
Results posted2026-06-23
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 Milestones8View
Results posted 2
Results expected 2
Recently completed 2
Other updates 2
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 timeline10

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

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

The current development programme across all trial phases.

Clinical programme
23
All trials
6
Active
14
Late-stage
6
Completed
Late-stage studies

A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy Followed by Maintenance Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome With Excess Blasts-2, With an IDH1 or IDH2 Mutation, Respectively, Eligible for Intensive Chemotherapy.

Phase 3 · Active, not recruiting · Stichting Hemato-Oncologie voor Volwassenen Nederland

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 Phase 3, Multicenter, Open-label, Randomized, Study of Gilteritinib Versus Midostaurin in Combination With Induction and Consolidation Therapy Followed by One-year Maintenance in Patients With Newly Diagnosed Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes With Excess Blasts-2 (MDS-EB2) With FLT3 Mutations Eligible for Intensive Chemotherapy (HOVON 156 AML / AMLSG 28-18)

Phase 3 · Active, not recruiting · Stichting Hemato-Oncologie voor Volwassenen Nederland

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

A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study of AG-120 in Combination With Azacitidine in Subjects ≥ 18 Years of Age With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation

Phase 3 · Active, not recruiting · Institut de Recherches Internationales Servier

A Phase 3b, Randomized, Open-Label, Double Crossover Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Adult Patients With IPSS R Intermediate Myelodysplastic Syndrome, Low Blast Acute Myeloid Leukemia, IPSS Intermediate-2 or High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

Phase 3 · Active, not recruiting · Otsuka Australia Pharmaceutical Pty Ltd
Recently completed

Efficacy and Safety of Ustekinumab for Intestinal Behçet's Syndrome Complicated by Myelodysplastic Syndrome

Phase 2 · Completed · Liu Tian

A Phase III Randomized Study of Farnesyl Transferase Inhibitor R115777 in Acute Myeloid Leukemia (AML) Patients in Second or Subsequent Remission or in Remission After Primary Induction Failure or Patients Over Age 60 in First Remission

Phase 3 · Completed · National Cancer Institute (NCI)

A Multi-Center Study of Conditioning With Treosulfan, Fludarabine and Escalating Doses of TBI for Allogeneic Hematopoietic Cell Transplantation in Patients With Acute Myeloid Leukemia (AML) Myelodysplastic Syndrome (MDS), and Acute Lymphoblastic Leukemia (ALL)

Phase 2 · Completed · Fred Hutchinson Cancer Center

A Programme of Treatment Development for Older Patients With Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome

Phase 2/3 · Completed · The University of New South Wales

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

A PHASE III STUDY IN CHILDREN WITH UNTREATED ACUTE MYELOGENOUS LEUKEMIA (AML) OR MYELODYSPLASTIC SYNDROME (MDS)

Phase 3 · Completed · National Cancer Institute (NCI)

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.

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

TET20.80GATA20.77ASXL10.76DNMT3A0.74SRSF20.68DDX410.68SETBP10.66CSF3R0.64SF3B10.64DNMT10.62TGFB10.58CRBN0.57

Disease biology

12 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

DNA (cytosine-5)-methyltransferase 3Avia DNMT3A0.74Granulocyte colony-stimulating factor receptorvia CSF3R0.64DNA (cytosine-5)-methyltransferase 1via DNMT10.62Transforming growth factor beta-1 proproteinvia TGFB10.58Protein cereblonvia CRBN0.57Erythropoietin receptorvia EPOR0.57DNA damage-binding protein 1via DDB10.56E3 ubiquitin-protein ligase RBX1via RBX10.56Transforming growth factor beta-2 proproteinvia TGFB20.56DNA polymerase delta subunit 3via POLD30.52Breakpoint cluster region proteinvia BCR0.52Cytidine deaminasevia CDA0.51

Reference

Authoritative identity, definition & identifiers.

Synonyms

MDS, MDS, unclassifiable, MDS-U, Myelodysplastic Syndromes, dysmyelopoietic syndrome, hematopoeitic - myelodysplastic syndrome (MDS), myelodysplasia, myelodysplastic neoplasm, myelodysplastic syndrome, somatic, myelodysplastic syndrome, unclassifiable, myelodysplastic syndrome/neoplasm, myelodysplastic syndromes, oligoblastic leukaemia, oligoblastic leukemia, preleukemia, smoldering leukemia, smouldering leukaemia, myelodysplastic syndrome, susceptibility to

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.