PPARA peroxisome proliferator activated receptor alpha
ENSG00000186951 · protein coding · Open Targets profile
Ligand-activated transcription factor. Key regulator of lipid metabolism. Activated by the endogenous ligand 1-palmitoyl-2-oleoyl-sn- glycerol-3-phosphocholine (16:0/18:1-GPC). Activated by oleylethanolamide, a naturally occurring lipid that regulates satiety. Receptor for peroxisome proliferators such as hypolipidemic drugs and fatty acids. Regulates the peroxisomal beta-oxidation pathway of fatty acids. Functions as a transcription activator for the ACOX1 and P450 genes. Transactivation activity requires heterodimerization with RXRA and is antagonized by NR2C2. May be required for the propagation of clock information to metabolic pathways regulated by PER2. {ECO:0000269|PubMed:10195690, ECO:0000269|PubMed:24043310, ECO:0000269|PubMed:7629123, ECO:0000269|PubMed:7684926, ECO:0000269|PubMed:9556573}.
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 Withdrawn means the drug was pulled from the market or from development.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, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.5213099718093872 (upper bound 0.7444000244140625). 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 112 registry trials, updated in the last three years, of the most advanced drugs against PPARA (choline fenofibrate, pemafibrate, elafibranor, ciprofibrate, fenofibric acid, bezafibrate, saroglitazar, gemfibrozil, fenofibrate, chiglitazar, tesaglitazar, K-877), 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+
- Channa JayasekeraMayo Clinic · 2 of 2 trials Phase 2+
- Ying HanThe First Affiliated Hospital of Air Force Medical University · 2 of 2 trials Phase 2+
- Bing XuThe First Affiliated Hospital of Xiamen University · 1 of 2 trials Phase 2+
- Alexandra ErshovaNational Medical Research Centre for Therapy and Preventive Medicine Ministry of Health of Russia · 1 of 1 trials Phase 2+
- Alicia JenkinsNHMRC Clinical Trials Centre, The University of Sydney · 1 of 1 trials Phase 2+
- Amedeo A AziziMedical University of Vienna · 1 of 1 trials Phase 2+
- Andre BelousKowa Pharma Development Co. · 1 of 1 trials Phase 2+
- Andreas PeyrlMedical University of Vienna · 1 of 1 trials Phase 2+
- Andrew A. NierenbergMassachusetts General Hospital · 1 of 1 trials Phase 2+
- Anthony KeechNHMRC Clinical Trials Centre, The University of Sydney · 1 of 1 trials Phase 2+
- Aruna PradhanCCVDP & The Brigham and Women's Hospital · 1 of 1 trials Phase 2+
- Ashraf M Alaaclinical pharmacy departement - Faculty of Pharmacy - Tanta University · 1 of 1 trials Phase 2+
Registry trials of choline fenofibrate, pemafibrate, elafibranor, ciprofibrate, fenofibric acid, bezafibrate, saroglitazar, gemfibrozil, fenofibrate, chiglitazar, tesaglitazar, K-877 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; 82 of 102 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. 10 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
57 trials of drugs against PPARA were stopped early; 4 give no reason
Terminated, withdrawn and suspended interventional trials of choline fenofibrate, pemafibrate, elafibranor, ciprofibrate, fenofibric acid, bezafibrate, saroglitazar, gemfibrozil, fenofibrate, chiglitazar, tesaglitazar, K-877, 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 57.
- withdrawn · 2026-08-19 · Polaryx Therapeutics, Inc. · matched by the registry's drug synonyms“Study has yet to begin and removing to avoid further participant outreach to sponsor”
- withdrawn · 2026-08-19 · Forest Hills Lab · Gemfibrozil“Has yet to begin and may not so halting to avoid participant outreach”
- 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 · 2025-12-18 · Zydus Therapeutics Inc. · Saroglitazar Magnesium 4 mg Tablet“Sponsor Decision”
- terminated · 2025-10-22 · Mayo Clinic · Fenofibrate“Low Enrollment”
- terminated · 2025-09-25 · Kowa Research Institute, Inc. · K-877“The study was stopped for futility based on efficacy results at the interim analysis; no unexpected safety findings were observed.”
- terminated · 2025-09-02 · Zydus Therapeutics Inc. · Saroglitazar Magnesium 4 mg“Sponsor Decision”
- withdrawn · 2024-12-18 · Piedmont Healthcare · matched by the registry's drug synonyms“Lack of patient population”
- withdrawn · 2023-06-23 · St. Louis University · matched by the registry's drug synonyms“No participants were recruited and the study was deemed not feasible”
- withdrawn · 2023-05-15 · St. Louis University · matched by the registry's drug synonyms“End of grant funding”
- suspended · 2023-02-08 · Zydus Therapeutics Inc. · Saroglitazar Magnesium 1 mg, Saroglitazar Magnesium 2 mg, Saroglitazar Magnesium 4 mg“Management decision to suspend the study due to recruitment issues”
- terminated · 2022-03-23 · Genfit · Elafibranor“Study did not meet the predefined primary surrogate efficacy endpoint, no safety issues identified”
- terminated · 2021-10-29 · The Mind Research Network · Gemfibrozil 600 MG“Funds for the study expired”
- terminated · 2021-10-28 · Genfit · Elafibranor 80mg, Elafibranor 120mg“Due to lack of efficacy (but not due to safety) in a Phase 3 trial of elafibranor in adult participants with NASH and fibrosis, this study in pediatric NASH was prematurely terminated”
- withdrawn · 2021-09-17 · Beijing 302 Hospital · Fenofibrate“No participants enrolled”
- terminated · 2020-09-16 · Jaeb Center for Health Research · Pemafibrate“recruitment for the substudy did not meet the goals”
- terminated · 2020-08-31 · Genfit · elafibranor 120mg“Continuation of the trial cannot serve a scientific purpose”
- terminated · 2019-11-29 · The University of Texas Medical Branch, Galveston · Fenofibrate“Shriner's Burn Hospital closed the study and access to study-related data is unavailable. We are unable to submit the additional information or results-data”
- terminated · 2019-11-29 · The University of Texas Medical Branch, Galveston · Fenofibrate“lost funding; Shriner Burn Hosp has revoked access to study records. No updates or results can be submitted”
- terminated · 2018-02-06 · University of Miami · fenofibrateNo reason given
- terminated · 2017-08-17 · VA Office of Research and Development · fenofibrate“Low recruitment in intervention study. Baseline data published.”
- terminated · 2017-06-14 · University of Miami · Fenofibrate“Lack of adequate accrual.”
- withdrawn · 2016-09-01 · Essentialis, Inc. · 135 mg fenofibric acid“Did not continue with development of DCCR in Very High Triglycerides”
- terminated · 2016-09-01 · University of Utah · matched by the registry's drug synonyms“Collaborator/corporate sponsor withdrew funding and permission to continue.”
Paediatric incentives and obligations for PPARA's drugs
For choline fenofibrate, pemafibrate, elafibranor, ciprofibrate, fenofibric acid, bezafibrate, saroglitazar, gemfibrozil, fenofibrate, chiglitazar, tesaglitazar, K-877: 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 · PPARA 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-003591-PIP01-24 (fenofibrate;rosuvastatin calcium, Treatment of elevated cholesterol with elevated triglycerides): W: decision granting a waiver in all age groups for all conditions or indications, 14 Jun 2024; EMEA-003563-PIP01-23 (atorvastatin calcium;fenofibrate, Treatment of hyperlipidaemia): W: decision granting a waiver in all age groups for all conditions or indications, 6 May 2024; EMEA-003562-PIP01-23 (ezetimibe;fenofibrate;rosuvastatin calcium, Treatment of hyperlipidaemia): W: decision granting a waiver in all age groups for all conditions or indications, 6 May 2024; EMEA-001573-PIP03-23 (pemafibrate, Treatment of primary biliary cholangitis): W: decision granting a waiver in all age groups for all conditions or indications, 8 Sept 2023; +9 moreRule: 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/3/25/3100 positive 18 Jul 2025: Treatment of primary sclerosing cholangitis; EU/3/23/2798 positive 25 Jul 2023: Treatment of primary biliary cholangitis; EU/3/22/2675 positive 10 Aug 2022: Treatment of primary sclerosing cholangitis; EU/3/19/2182 positive 25 Jul 2019: Treatment of primary biliary cholangitis; +1 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 · Cholib: authorised since 26 Aug 2013; Iqirvo: authorised since 19 Sept 2024, orphan medicine, conditional; Pravafenix: authorised since 14 Apr 2011; Synordia: application withdrawnRule: 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 choline fenofibrate, pemafibrate, elafibranor, ciprofibrate, fenofibric acid, bezafibrate, saroglitazar, gemfibrozil, fenofibrate, chiglitazar, tesaglitazar, k-877Rule: 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 · 25 interventional studies of choline fenofibrate, pemafibrate, elafibranor, ciprofibrate, fenofibric acid, bezafibrate, saroglitazar, gemfibrozil, fenofibrate, chiglitazar, tesaglitazar, k-877 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 27 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.