Phuturdex › Diseases › Myelodysplastic Syndromes
Drugs in development · updated 30 October 2026
Myelodysplastic Syndromes: drugs in development
As of 30 October 2026 there are 259 active clinical trials (recruiting or ongoing, phases I–III) on 203 drugs for myelodysplastic Syndromes. 94 are experimental, that is not yet approved in the United States.
Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.
Sign in free →Experimental drugs
Not yet approved by the US FDA for any indication.
Sorted by most advanced phase and number of active trials. Showing 40 of 94 drugs.
Approved drugs in new trials
Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| FludarabineFludara, FLUDARABIN | II–III | 58 | NCT04708054 |
| AzacitidineAzacytidine, 5-azacytidine | II–III | 45 | NCT04256317 |
| CyclophosphamideCyclophosphamid, Cytoxan | III | 45 | NCT05457556 |
| VenetoclaxVentoclax, Venclexta® | II–III | 36 | NCT04256317 |
| DecitabineDacogen® | III | 29 | NCT05883956 |
| TacrolimusEnvarsus XR, Prograf® | III | 27 | NCT05457556 |
| MelphalanEvomela®, Hepzato® | III | 24 | NCT05457556 |
| BusulfanBusulphan, Busulfex® | II–III | 23 | NCT04708054 |
| Mycophenolate MofetilMycophenolate, Cellcept | III | 21 | NCT05457556 |
| CytarabineAra-C, Cytosine Arabinoside | III | 19 | NCT02521493 |
| ThiotepaTepylute®, Tepadina And Sodium Chloride® | II–III | 14 | NCT04708054 |
| LuspaterceptACE-536, Reblozyl | III | 13 | NCT07894861 |
| CedazuridineCedaruridine | II–III | 12 | NCT04256317 |
| MethotrexateJylamvo®, Reditrex® | III | 9 | NCT05457556 |
| Epoetin AlfaEprex, Epoetin alga | III | 8 | NCT07422480 |
Sorted by most advanced phase and number of active trials. Showing 15 of 109 drugs.
All 203 drugs, with the details
In Phuturdex every drug has all its trials, sponsors and countries, FDA and EMA approvals, orphan designations and safety alerts. Free until 31 March 2027.
Open in Phuturdex →Specific forms
- Paroxysmal nocturnal hemoglobinuria 36 drugs
- Acute myeloid leukemia 314 drugs
- Refractory anemia with excess of blasts 35 drugs
- GATA2 deficiency 18 drugs
- Chronic myelomonocytic leukemia 90 drugs
- Precursor lymphoblastic lymphoma/leukemia 163 drugs
- Acute biphenotypic leukaemia 80 drugs
- Aplastic anemia 43 drugs
- Anemia 52 drugs
- Hemolysis 3 drugs
- Hematologic neoplasm 216 drugs
- Lymphoid leukemia 18 drugs
Broader categories
- Bone marrow disease 14 drugs
- Hematologic Diseases 57 drugs
- Neoplasm 671 drugs
- Myeloid leukemia 26 drugs
- Leukemia 153 drugs
- Immune System Diseases 54 drugs
Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: NLM MeSH; NLM MeSH term: D009190 “Myelodysplastic Syndromes”. Prevalence: Orphanet.