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Drugs in development · updated 30 October 2026

Myelodysplastic–myeloproliferative diseases: drugs in development

Rare diseasePrevalence 1-9 / 1 000 000 according to Orphanet · ORPHA:98275

As of 30 October 2026 there are 18 active clinical trials (recruiting or ongoing, phases I–III) on 40 drugs for myelodysplastic–myeloproliferative diseases. 4 are experimental, that is not yet approved in the United States.

18Active trials
4Experimental drugs
0Experimental, in phase III

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.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
IadademstatII1NCT06661915
VeliparibABT-888II1NCT03289910
LP-118I1NCT04771572
OnvansertibI1NCT05549661

Sorted by most advanced phase and number of active trials.

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.

DrugMost advanced phaseActive trialsReference trial
FludarabineFludara, FLUDARABINII5NCT07228624
BusulfanBusulphan, Busulfex®II4NCT07228624
CyclophosphamideCyclophosphamid, CytoxanII4NCT07228624
DecitabineDacogen®I–II4NCT05038592
AzacitidineAzacytidine, 5-azacytidineI–II3NCT04140487
MelphalanEvomela®, Hepzato®II3NCT07228624
TacrolimusEnvarsus XR, Prograf®II3NCT07228624
CedazuridineCedaruridineII2NCT06661915
MethotrexateJylamvo®, Reditrex®II2NCT07228624
Mycophenolate MofetilMycophenolate, CellceptII2NCT02861417
RuxolitinibJakavi, Jakafi Xr®II2NCT07228624
VenetoclaxVentoclax, Venclexta®I–II2NCT04140487
Anti-Thymocyte GlobulinII1NCT02727803
AxatilimabNiktimvo®I–II1NCT06523556
CarboplatinCarboplatine, ParaplatinII1NCT03289910

Sorted by most advanced phase and number of active trials. Showing 15 of 36 drugs.

All 40 drugs, with the details

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Specific forms

Broader categories

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: Wikidata; NLM MeSH term: D054437 “Myelodysplastic-Myeloproliferative Diseases”. Prevalence: Orphanet.

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