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Drugs in development · updated 30 October 2026

Langerhans-cell histiocytosis: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:389

As of 30 October 2026 there are 28 active clinical trials (recruiting or ongoing, phases I–III) on 37 drugs for langerhans-cell histiocytosis. 6 are experimental, that is not yet approved in the United States.

28Active trials
6Experimental drugs
1Experimental, 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
FluorodeoxyglucoseIII1NCT04943211
LuvometinibFCN-159II2NCT07728201
TipifarnibII2NCT04284774
UlixertinibII2NCT03698994
CD34I–II1NCT01966367
SamotolisibLY3023414II1NCT03155620

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
CytarabineAra-C, Cytosine ArabinosideII–III4NCT02205762
TrametinibMekinist®, Spexotras®II–III4NCT07440290
PrednisonePredinisone, CortancylII–III3NCT02205762
DabrafenibTafinlar®, Finlee®II–III2NCT07440290
VinblastineVinblastin, Velban®II–III2NCT02205762
Cladribine2-chlorodeoxyadenosine, Mavenclad®II–III1NCT02205762
IndomethacinTivorbex®, Indocin®II–III1NCT02205762
Mercaptopurine6-Mercaptopurine, MecaptopurineII–III1NCT02205762
MethotrexateJylamvo®, Reditrex®II–III1NCT02205762
ClofarabineClolar®, Evoltra®II2NCT02425904
EnsartinibX-396, EnsatinibII2NCT03213652
ErdafitinibBalversa®II2NCT03210714
IvosidenibAG-120, Tibsovo®II2NCT03155620
LarotrectinibBAY2757556, Vitrakvi®II2NCT03155620
SelpercatinibLOXO-292, Retevmo®II2NCT04320888

Sorted by most advanced phase and number of active trials. Showing 15 of 31 drugs.

All 37 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: D006646 “Histiocytosis, Langerhans-Cell”. Prevalence: Orphanet.

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