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Drugs in development · updated 30 October 2026

Thrombocythemia, Essential: drugs in development

Rare diseasePrevalence 1-5 / 10 000 according to Orphanet · ORPHA:3318

As of 30 October 2026 there are 35 active clinical trials (recruiting or ongoing, phases I–III) on 43 drugs for thrombocythemia, Essential. 15 are experimental, that is not yet approved in the United States.

35Active trials
15Experimental drugs
2Experimental, 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
BomedemstatIMG-7289III5NCT06079879
INCA033989III3NCT07623200
IadademstatII1NCT06661915
JBI-802I–II1NCT07612280
PevonedistatI–II1NCT03862157
STP938II1NCT06786234
VeliparibABT-888II1NCT03289910
DMR-001I1NCT07849231
GLB-001I1NCT06378437
INCA035784I1NCT07008118
INCA036978I1NCT07441694
INCB057643I1NCT04279847
NavitoclaxI1NCT04041050
Poly ICLCHiltonolI1NCT05025488
ZE74-0282-0001I1NCT07527221

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
RuxolitinibJakavi, Jakafi Xr®III9NCT06079879
Ropeginterferon alfa-2bP1101, Besremi®III3NCT04285086
AnagrelideAgrylin®, Anagrelide Viatris (previously Anagrelide Mylan)®III2NCT06079879
BusulfanBusulphan, Busulfex®III2NCT06079879
Ropeginterferon alfa-2b-njftBesremi®III2NCT05481151
AspirinVazalore®, Durlaza®III1NCT05198960
HydroxyureaHydroxycarbamide, HydroxycarbamidIII1NCT06456346
CyclophosphamideCyclophosphamid, CytoxanII2NCT00544115
DecitabineDacogen®II2NCT06661915
FludarabineFludara, FLUDARABINII2NCT00544115
MelphalanEvomela®, Hepzato®II2NCT00544115
MethotrexateJylamvo®, Reditrex®II2NCT00544115
Mycophenolate MofetilMycophenolate, CellceptII2NCT00544115
PacritinibVonjo®II2NCT04282187
TacrolimusEnvarsus XR, Prograf®II2NCT00544115

Sorted by most advanced phase and number of active trials. Showing 15 of 28 drugs.

All 43 drugs, with the details

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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: NLM MeSH; NLM MeSH term: D013920 “Thrombocythemia, Essential”. Prevalence: Orphanet.

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