PPARG peroxisome proliferator activated receptor gamma
ENSG00000132170 · protein coding · Open Targets profile
Ligand-activated transcription factor that forms obligate heterodimers with the retinoic acid receptor and acts as a key regulator of biological processes, such as adipocyte differentiation, lipid metabolism, glucose homeostasis and beta-oxidation of fatty acids (PubMed:16150867, PubMed:20829347, PubMed:23525231, PubMed:8702406, PubMed:8706692, PubMed:9065481). Activated by lipid ligands: binds peroxisome proliferators, such as hypolipidemic drugs, and fatty acids, such as prostaglandin J2 metabolites (PubMed:16150867, PubMed:20829347, PubMed:23525231, PubMed:8702406, PubMed:8706692, PubMed:9065481). Ligand-binding results in a conformational change in the receptor, promoting dissociation of repressors and recruitment of coactivators, and subsequent activation of target gene expression (PubMed:16150867, PubMed:20829347, PubMed:23525231, PubMed:8702406, PubMed:8706692, PubMed:9065481). Specifically binds to DNA specific PPAR response elements (PPRE) and modulates the transcription of its target genes, such as acyl-CoA oxidase (By similarity). Acts as a critical regulator of gut homeostasis by suppressing NF-kappa-B-mediated pro-inflammatory responses (PubMed:20829347). Plays a role in the regulation of cardiovascular circadian rhythms by regulating the transcription of BMAL1 in the blood vessels (By similarity). {ECO:0000250|UniProtKB:P37238, ECO:0000269|PubMed:16150867, ECO:0000269|PubMed:20829347, ECO:0000269|PubMed:23525231, ECO:0000269|PubMed:8702406, ECO:0000269|PubMed:8706692, ECO:0000269|PubMed:9065481}.
Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.
Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.
Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.
Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.
Tractability
- Small molecule
- Approved Drug, Structure with Ligand, High-Quality Ligand, High-Quality Pocket, Druggable Family
- PROTAC
- UniProt Ubiquitination, Database Ubiquitination, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.4334700107574463 (upper bound 0.650189995765686). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.
Who runs trials of these drugs
Principal investigators and study chairs on 196 registry trials, updated in the last three years, of the most advanced drugs against PPARG (balsalazide, olsalazine, bezafibrate, pioglitazone, saroglitazar, mesalamine, MK-0767, leriglitazone, lanifibranor, farglitazar, bardoxolone methyl, muraglitazar), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Deven ParmarZydus Therapeutics Inc. · 5 of 8 trials Phase 2+
- Deven V ParmarZydus Therapeutics Inc. · 4 of 5 trials Phase 2+
- Lynda SzczechIntercept Pharmaceuticals, Inc · 3 of 3 trials Phase 2+
- Ralph DeFronzoThe University of Texas Health Science Center at San Antonio · 2 of 3 trials Phase 2+
- Kenneth CusiUniversity of Florida · 2 of 2 trials Phase 2+
- Muhammad Abdul-GhaniThe University of Texas Health Science Center at San Antonio · 2 of 2 trials Phase 2+
- Vipul JairathWestern University; London Health Sciences Centre · 2 of 2 trials Phase 2+
- Ying HuangChildren's Hospital of Fudan University · 2 of 2 trials Phase 2+
- Khashayar SakhaeeUTSW · 1 of 2 trials Phase 2+
- Ralph A DeFronzoUniversity of Texas · 1 of 2 trials Phase 2+
- Aaron TrammellEmory University · 1 of 1 trials Phase 2+
- Abhay MuthaDiabetes Care & Research Centre · 1 of 1 trials Phase 2+
- Alexandre KarrasAssistance Publique - Hôpitaux de Paris · 1 of 1 trials Phase 2+
- Andrei SpositoUniversity of Campinas, Brazil · 1 of 1 trials Phase 2+
- Andrew A. NierenbergMassachusetts General Hospital · 1 of 1 trials Phase 2+
Registry trials of balsalazide, olsalazine, bezafibrate, pioglitazone, saroglitazar, mesalamine, MK-0767, leriglitazone, lanifibranor, farglitazar, bardoxolone methyl, muraglitazar updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 149 of 180 have all sites in one country.
Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. 16 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
128 trials of drugs against PPARG were stopped early; 22 give no reason
Terminated, withdrawn and suspended interventional trials of balsalazide, olsalazine, bezafibrate, pioglitazone, saroglitazar, mesalamine, MK-0767, leriglitazone, lanifibranor, farglitazar, bardoxolone methyl, muraglitazar, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 25 most recent of 128.
- terminated · 2026-09-18 · Gilead Sciences · matched by the registry's drug synonyms“MBX-102 did not meet the target efficacy profile of HbA1c change.”
- terminated · 2026-08-31 · Emory University · matched by the registry's drug synonyms“The study was suspended and during this time recruitment and study activities were halted. The study was ultimately terminated in April 2026 without having re-opened for recruitment additional participants.”
- terminated · 2026-07-27 · The University of Texas at Arlington · Pioglitazone“CKD participants we recruited do not have elevated ADMA, which is the main premise for this intervention. We noticed this with first cohort studied and collected blood from our other ongoing CKD studies, which further solidified and why terminated.”
- terminated · 2026-07-16 · Intercept Pharmaceuticals · Bezafibrate 200 MG, Bezafibrate 200 mg Placebo, Bezafibrate 400 MG, Bezafibrate 400 mg Placebo, Bezafibrate“Intercept made a business decision to terminate the study based on FDA's request for voluntary withdrawal of Ocaliva and the issuance of clinical hold on studies under US IND involving OCA.”
- terminated · 2026-06-08 · Intercept Pharmaceuticals · matched by the registry's drug synonyms“Intercept made a business decision to terminate the study based on FDA's request for voluntary withdrawal of Ocaliva and the issuance of clinical hold on studies under US IND involving OCA.”
- terminated · 2026-04-28 · University of Maryland, Baltimore · Pioglitazone 15mg“Loss of Financial Sponsor”
- terminated · 2025-12-18 · Zydus Therapeutics Inc. · Saroglitazar Magnesium 4 mg Tablet“Sponsor Decision”
- terminated · 2025-09-02 · Zydus Therapeutics Inc. · Saroglitazar Magnesium 4 mg“Sponsor Decision”
- withdrawn · 2025-08-12 · AbbVie · Mesalamine“strategic considerations”
- terminated · 2025-06-11 · Biogen · Bardoxolone methyl“The company determined that continued exposure of these high-risk PH patients to clinic or in-person visits during the COVID pandemic presented an unacceptable risk.”
- terminated · 2025-06-04 · Biogen · Bardoxolone methyl capsules“Exposure of these high-risk patients to clinic or in-person visits during the pandemic presented an unacceptable risk to their health”
- terminated · 2025-06-03 · Biogen · Bardoxolone methyl“Discontinuation of all bardoxolone chronic kidney disease programs”
- terminated · 2025-06-03 · Biogen · Bardoxolone methyl oral capsule“Discontinuation of all bardoxolone chronic kidney disease programs”
- terminated · 2025-05-30 · Biogen · matched by the registry's drug synonyms“Enrollment discontinued after second cohort completed.”
- withdrawn · 2025-05-29 · Biogen · Bardoxolone Methyl“IDMC recommendation for safety concerns”
- withdrawn · 2025-05-29 · Biogen · Bardoxolone Methyl 20 mg“IDMC recommendation for safety concerns”
- terminated · 2025-05-29 · Biogen · Bardoxolone methyl“To pursue other indications”
- terminated · 2025-05-29 · Biogen · Bardoxolone Methyl: 20 mg“IDMC recommendation for safety concerns”
- terminated · 2025-05-29 · Biogen · Bardoxolone Methyl“IDMC recommendation for safety concerns”
- withdrawn · 2025-05-29 · Biogen · Bardoxolone Methyl“IDMC recommendation for safety concerns”
- terminated · 2025-03-14 · Kinarus AG · matched by the registry's drug synonyms“Sponsor went bankrupt - trial was discontinued due to lack of financing”
- terminated · 2025-02-21 · Boston Medical Center · matched by the registry's drug synonyms“Unable to recruit adequate numbers of participants to answer the research question.”
- terminated · 2024-05-08 · University Hospital Regensburg · Pioglitazone“Due to financial reasons.”
- terminated · 2023-10-27 · Biogen · 20 mg bardoxolone methyl“IDMC recommendation for safety concerns”
- terminated · 2023-09-26 · Kyowa Kirin Co., Ltd. · Bardoxolone methyl“Based on results of double-blind parts, it was determined that it would be difficult to submit a marketing authorization application for RTA 402 for diabetic kidney disease.”
Paediatric incentives and obligations for PPARG's drugs
For balsalazide, olsalazine, bezafibrate, pioglitazone, saroglitazar, mesalamine, MK-0767, leriglitazone, lanifibranor, farglitazar, bardoxolone methyl, muraglitazar: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.
- FDA pediatric target list: no · PPARG is not on the listRule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
- EU paediatric investigation plan: yes · EMEA-002106-PIP01-16-M03 (leriglitazone, Treatment of adrenoleukodystrophy): PM: decision on the application for modification of an agreed PIP, 13 Sept 2024; EMEA-002488-PIP01-18 (Bardoxolone methyl, Treatment of Alport syndrome): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 16 Jul 2021; EMEA-002187-PIP01-17 (pioglitazone hydrochloride;spironolactone metformin hydrochloride, Treatment of polycystic ovary syndrome): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 16 Apr 2021Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
- EU orphan designation: yes · EU/3/21/2480 positive 20 Aug 2021: Treatment of autosomal dominant polycystic kidney disease; EU/3/18/2019 positive 25 May 2018: Treatment of Alport syndrome; EU/3/22/2675 positive 10 Aug 2022: Treatment of primary sclerosing cholangitis; EU/3/14/1361 withdrawn 19 Nov 2014: Treatment of systemic sclerosis; +4 moreRule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
- Authorised in the EU: yes · Nezglyal: authorised since 21 Sept 2026, orphan medicine, exceptional circumstances; Competact: authorised since 28 Jul 2006; Nezglyal: refused, orphan medicine; Pioglitazone Accord: authorised since 21 Mar 2012; +17 moreRule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
- FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of balsalazide, olsalazine, bezafibrate, pioglitazone, saroglitazar, mesalamine, mk-0767, leriglitazone, lanifibranor, farglitazar, bardoxolone methyl, muraglitazarRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-12
- Registered studies open to children: yes · 47 interventional studies of balsalazide, olsalazine, bezafibrate, pioglitazone, saroglitazar, mesalamine, mk-0767, leriglitazone, lanifibranor, farglitazar, bardoxolone methyl, muraglitazar include patients under 18; 3 are recruiting, not yet recruiting or activeRule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-12
Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs
All 40 drugs and clinical candidates as a table
Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.