Biomarker & Intervention Discovery
Polycystic Kidney Disease (ADPKD)
Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.
NIH funding$745MFY2025 · RCDC estimate
Spontaneous remissionProgressive; GFR decline averages 4-5 mL/min/year in Mayo Class 1C-1E; renal failure by 60-70 years in 50% without…
Best-intervention remissionTolvaptan (Jynarque): slows GFR decline by 35% and total kidney volume growth by 49% (TEMPO 3:4, REPRISE); dietary…
Gap sizeModerate — tolvaptan is available and effective but hepatotoxicity risk limits use; access barriers (REMS programme in…
Primary barrieraccess/practice
Full remission profile →
Remission & chronicity
Spontaneous remission
Progressive; GFR decline averages 4-5 mL/min/year in Mayo Class 1C-1E; renal failure by 60-70 years in 50% without treatment; spontaneous stabilisation rare
Best-intervention remission
Tolvaptan (Jynarque): slows GFR decline by 35% and total kidney volume growth by 49% (TEMPO 3:4, REPRISE); dietary protein restriction + hydration: modest benefit; SGLT2 inhibitors — early signal in PKD trials
Gap size
Moderate — tolvaptan is available and effective but hepatotoxicity risk limits use; access barriers (REMS programme in US) reduce prescribing
Primary barrier
access/practice
Tolvaptan REMS programme (liver toxicity monitoring) creates prescribing complexity; only patients with rapidly progressive disease (Mayo Class 1C-1E) are eligible by guideline; most patients remain on supportive care without disease-modifying therapy
Biomarker Targets & Therapeutic Agents
Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC.
Agents ranked: ● Clinical >
● Mechanistic >
● Correlative.
Pipeline results not yet available. Run: python -m biomarker_pipeline.run_for_diseases --skip-llm
Clinical Trials
Recruiting and recently completed trials from ClinicalTrials.gov.
Data retrieved 2026-07-06.
No recent clinical trials found on ClinicalTrials.gov for this condition.
Agents Found By Disease-Level Search
Searched directly from “Polycystic Kidney Disease (ADPKD)” via ClinicalTrials.gov interventions and Open Targets'
disease→drug data — independent of the 0-gene target panel above.
This surfaces agents whose mechanism doesn't route through a curated gene (combination
therapies, standard-of-care drugs, targets outside the panel).
Not yet run. python -m biomarker_pipeline.run_disease_agent_discovery --disease "..."