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Drugs in development · updated 30 October 2026

Pachyonychia congenita: drugs in development

Rare diseasePrevalence <1 / 1 000 000 according to Orphanet · ORPHA:2309

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

3Active trials
2Experimental 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
PTX-022III1NCT05643872
KM-023II1NCT07893483

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
ErlotinibTarceva®, Erlotinib Accord®I–II1NCT06545695

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: D053549 “Pachyonychia Congenita”. Prevalence: Orphanet.

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