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Drugs in development · updated 30 October 2026

Acute megakaryoblastic leukemia: drugs in development

Rare diseasePrevalence <1 / 1 000 000 according to Orphanet · ORPHA:518

As of 30 October 2026 there are 2 active clinical trials (recruiting or ongoing, phases I–III) on 3 drugs for acute megakaryoblastic leukemia. 0 are experimental, that is not yet approved in the United States.

2Active trials
0Experimental 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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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
AzacitidineAzacytidine, 5-azacytidineI–II2NCT00392353
Gemtuzumab OzogamicinMylotarg, GemtuzumabII1NCT00658814
VorinostatZolinza®, Vorinostat MSD®I–II1NCT00392353

Sorted by most advanced phase and number of active trials.

All 3 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: Wikidata; NLM MeSH term: D007947 “Leukemia, Megakaryoblastic, Acute”. Prevalence: Orphanet.

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