Polycystic Kidney Disease (ADPKD) Drug Landscape
As of November 6, 2026, Drugnews tracks 14 ADPKD drug assets: one approved, 10 in clinical development and three whose late-stage trials were stopped. Of the 10 clinical-stage assets, four are from Asian sponsors (including one hospital) (2 China, 1 Japan, 1 Taiwan).
Why this disease is hard to treat
Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease. Cysts grow year after year and crowd out healthy tissue, and many patients need dialysis or a transplant in their fifties or sixties.
Only one drug is approved worldwide: Otsuka's tolvaptan (JYNARQUE), which blocks the vasopressin V2 receptor to slow cyst growth. In May 2025 Lupin launched the first US generic of tolvaptan. The only approved drug now faces generic competition, and patients are still waiting for a second mechanism.
Three late-stage trials stopped
Sanofi's venglustat Phase 2/3 trial stopped for futility at interim analysis. Reata/Biogen's bardoxolone trial stopped because Biogen discontinued all CKD programs. Palladio's lixivaptan trial stopped for commercial reasons after enrolling 12 participants. ADPKD trials track kidney volume and the rate of kidney-function decline, which makes them long and expensive.
Big pharma is coming back
After several late-stage trials stopped, five large pharmaceutical companies remain involved in clinical development:
- Novartis announced the acquisition of Regulus on April 30, 2025 for $7 per share in cash plus a $7-per-share contingent value right payable on approval of farabursen, worth up to about $1.7 billion in total. Farabursen, an oligonucleotide targeting miR-17, had completed a Phase 1b study at the time.
- Vertex's VX-407 targets patients with specific PKD1 variants; its Phase 2a has completed enrollment.
- AbbVie and Calico's ABBV-CLS-628 is enrolling a Phase 2 trial.
- AstraZeneca (AZD1613) and GSK (GSK4771261) are both in Phase 1.
A disease with a single approved drug, now facing generics, is drawing Novartis, Vertex, AbbVie, AstraZeneca and GSK at the same time. They see a market that is still largely unserved.
Asia: China, Japan and Taiwan on different routes
- China: Jemincare's JMKX003142 is in a Phase 2 trial in rapidly progressing ADPKD; Shanghai Renji Hospital has registered an early Phase 1 study of GBP-213.
- Japan: Rege Nephro is repurposing tamibarotene for ADPKD in a 52-week Phase 2a trial.
- Taiwan: Gilva Therapeutics' GV-100 is an oral selective HDAC6 inhibitor. A Phase 1 single- and multiple-ascending-dose trial in healthy volunteers started in Australia in June 2026, with 80 participants planned.
GV-100 is the only HDAC6 inhibitor in this table: tolvaptan and JMKX003142 block the vasopressin V2 receptor, farabursen uses an oligonucleotide to suppress miR-17, and ABBV-CLS-628 is an anti-PAPP-A antibody.
What to watch
- The design and timing of Novartis's next farabursen trial, following the up-to-$1.7 billion acquisition.
- Phase 2a results for Vertex's VX-407, a small molecule aimed at a PKD1-variant subgroup.
- Safety and pharmacokinetics from Gilva's GV-100 Phase 1, which will set the dose for patient trials.
Three existing drugs are also in Phase 3 trials in ADPKD: dapagliflozin (STOP-PKD), metformin (IMPEDE-PKD) and hydrochlorothiazide (HYDRO-PROTECT).
Development stage
Country
Drug assets
| Asset | Company / country | Mechanism / route | Stage | Indication | Trial ID | Deal | Latest event | Sources |
|---|---|---|---|---|---|---|---|---|
| JYNARQUE(tolvaptan) | OtsukaJapan | Vasopressin V2 receptor antagonistOral | Approved | Rapidly progressing ADPKD | — | — | First US generic launched (Lupin, 180-day exclusivity)2025-05-13 | 2025-05-13 |
| ABBV-CLS-628 | AbbVie/CalicoUnited States | anti-PAPP-A monoclonal antibodyIntravenous | Phase 2 | ADPKD | NCT06902558 | — | ANCHOR Phase 2 recruiting2026-10-06 | 2026-10-06 Accessed 2026-11-06 |
| JMKX003142 | JemincareChina | Vasopressin V2 receptor antagonistOral | Phase 2 | Rapidly progressing ADPKD | NCT06800651 | — | Phase 2 recruiting in China, with supporting Phase 1 studies2025-07-03 | 2025-07-03 2026-03-05 |
| VX-407 | VertexUnited States | small-molecule PC1 folding correctorOral | Phase 2a | ADPKD with specific PKD1 variants | NCT07161037 | — | Phase 2a enrollment completed2026-09-21 | 2026-09-21 Accessed 2026-11-06 |
| tamibarotene | Rege NephroJapan | Retinoic acid receptor agonist (repurposing)Oral | Phase 2a | ADPKD | NCT06289998 | — | Phase 2a in Japan, 52 weeks2025-04-04 | 2025-04-04 |
| farabursen(RGLS8429) | Novartis (acquired Regulus)Switzerland | miR-17 oligonucleotideSubcutaneous | Phase 1b completed | ADPKD | NCT05521191 | Novartis acquired Regulus in 2025: $7 per share in cash plus a $7-per-share CVR on approval; up to about $1.7 billion | Phase 1b multiple-ascending-dose study completed at acquisition2025-04-30 | 2025-04-30 2025-05-01 2025-06-03 |
| AZD1613 | AstraZenecaUnited Kingdom | Not disclosedInjection | Phase 1 | ADPKD | NCT07228364 | — | Phase 1 multiple-ascending-dose study in ADPKD (including China)2026-10-13 | 2026-10-13 |
| GSK4771261 | GSKUnited Kingdom | Not disclosed— | Phase 1 | ADPKD | NCT06734234 | — | Phase 1a/b in healthy participants and ADPKD patients2026-07-30 | 2026-07-30 |
| PYC-003 | PYC TherapeuticsAustralia | Peptide-conjugated PMO antisense oligonucleotideIntravenous | Phase 1 | ADPKD | NCT06714006 | — | Phase 1 (Australia, Hong Kong, New Zealand)2026-07-27 | 2026-07-27 |
| GV-100 | Gilva TherapeuticsTaiwan | Oral selective HDAC6 inhibitorOral | Phase 1 | ADPKD | NCT07788014 | — | Phase 1 single- and multiple-ascending-dose study in healthy volunteers recruiting in Australia (80 planned; started June 2026)2026-08-26 | 2026-05-04 2026-08-26 2026-05-28 |
| GBP-213 | Shanghai Renji HospitalChina | Not disclosedSubcutaneous | Early Phase 1 | ADPKD | NCT07891364 | — | Not yet recruiting2026-10-23 | 2026-10-23 |
| venglustat | SanofiFrance | Glucosylceramide synthase inhibitorOral | Phase 2/3 terminated | ADPKD | NCT03523728 NCT04705051 | — | STAGED-PKD stopped for futility at interim analysis in 20212021-08-03 | 2023-02-03 2025-09-17 |
| bardoxolone methyl | Reata/BiogenUnited States | Nrf2 activatorOral | Phase 3 terminated | ADPKD | NCT03918447 | — | FALCON Phase 3 terminated (all CKD programs discontinued)2025-06-03 | 2025-06-03 |
| lixivaptan | Palladio BiosciencesUnited States | Vasopressin V2 receptor antagonistOral | Phase 3 terminated | ADPKD | NCT04064346 | — | ACTION Phase 3 terminated (commercial reasons; 12 participants)2023-02-06 | 2023-02-06 |
Repurposing existing drugs
| Asset | Sponsor | Stage | Study | Trial ID |
|---|---|---|---|---|
| dapagliflozin | University of Cologne | Phase 3 | STOP-PKD | NCT07280585 |
| metformin XR | University of Queensland | Phase 3 | IMPEDE-PKD | NCT04939935 |
| hydrochlorothiazide | UMC Groningen | Phase 3 | HYDRO-PROTECT | NCT05373264 |
Frequently asked questions
What drugs are approved for ADPKD?
This dataset lists Otsuka’s tolvaptan (JYNARQUE) as approved for rapidly progressing ADPKD. Lupin launched the first US generic in May 2025.
How much did Novartis agree to pay for Regulus, and on what terms?
Announced on April 30, 2025. In 2025, Novartis acquired Regulus for $7 per share in cash plus a $7-per-share contingent value right payable on approval, worth up to about $1.7 billion in total.
Which Asian ADPKD assets are in clinical development?
Jemincare’s JMKX003142 (China, Phase 2), Rege Nephro’s tamibarotene (Japan, Phase 2a), Gilva Therapeutics’ GV-100 (Taiwan, Phase 1), and Shanghai Renji Hospital’s GBP-213 (China, early Phase 1; not yet recruiting).