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Drugs in development · updated 30 October 2026

Congenital adrenal hyperplasia: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:418

As of 30 October 2026 there are 13 active clinical trials (recruiting or ongoing, phases I–III) on 7 drugs for congenital adrenal hyperplasia. 4 are experimental, that is not yet approved in the United States.

13Active trials
4Experimental 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
AtumelnantII–III3NCT07159841
AsedebartII1NCT07859293
NBIP-01435II1NCT07850739
OMS1620I1NCT07811089

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
CrinecerfontCrenessity®III4NCT04490915
MetyraponeMetopirone®II2NCT06106295
OsilodrostatLCI699, Isturisa®II1NCT07104812

Sorted by most advanced phase and number of active trials.

All 7 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: D000312 “Adrenal Hyperplasia, Congenital”. Prevalence: Orphanet.

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