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Drugs in development · updated 30 October 2026

Autosomal dominant polycystic kidney: drugs in development

Rare diseasePrevalence 1-5 / 10 000 according to Orphanet · ORPHA:730

As of 30 October 2026 there are 23 active clinical trials (recruiting or ongoing, phases I–III) on 30 drugs for autosomal dominant polycystic kidney. 11 are experimental, that is not yet approved in the United States.

23Active trials
11Experimental 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
CAM2029II–III1NCT05281328
JMKX003142II5NCT06800651
VX-407II2NCT07161037
PlcaceboII1NCT06800651
TamibaroteneII1NCT06289998
14C-VX-407I1NCT07868926
[14C]JMKX003142I1NCT07463053
GSK4771261I1NCT06734234
GV-100I1NCT07788014
PYC-003I1NCT06714006
GBP-213Early I1NCT07891364

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
DapagliflozinForxiga, Dapagliflozin PropanediolIII1NCT07280585
HydrochlorothiazideInzirqo®, Microzide®III1NCT05373264
MetforminRiomet Er®, Glumetza®III1NCT04939935
AtorvastatinLipitor, Atorvaliq®II1NCT05870007
Bempedoic acidNexletol®, Nilemdo®II1NCT07282821
EmpagliflozinJardianceII1NCT05510115
Leuprolide AcetateLeuprorelin, LupronII1NCT05478083
Sodium BicarbonateSodium Bicarbonate In Plastic Container®II1NCT05870007
TirzepatideZepbound, MounjaroII1NCT06582875
AmiodaroneNexterone®, Cordarone®I1NCT07181265
CyclosporineCyclosporine A, Cyclosporin CapsulesI1NCT07565441
DigoxinLanoxin®, Lanoxicaps®I1NCT07565441
DrospirenoneDRSP, Slynd®I1NCT07792408
EfavirenzSustiva®, Stocrin®I1NCT07565441
FebuxostatAdenuric, Uloric®I1NCT07181265

Sorted by most advanced phase and number of active trials. Showing 15 of 19 drugs.

All 30 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: D016891 “Polycystic Kidney, Autosomal Dominant”. Prevalence: Orphanet.

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