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Protein / target

Peroxisome proliferator-activated receptor delta

Encoded byPPARDQ03181Homo sapiensSwiss-Prot
UniProt: Q03181Ensembl: ENSG00000112033HGNC: HGNC:9235NCBI Gene: 5467
Clinically validated
Therapeutic maturity
3
Approved medicines
Open Targets target-level
View by indication →
26
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Sequence-specific double-stranded DNA binding

Strongest disease association

Biliary liver cirrhosis

Via encoding gene PPARD · Clinical evidence · score 0.51

Therapeutic position

Established drug target

Small molecules

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function overview

Ligand-activated transcription factor key mediator of energy metabolism in adipose tissues.

View complete UniProt function annotation

Ligand-activated transcription factor key mediator of energy metabolism in adipose tissues (PubMed:35675826). Receptor that binds peroxisome proliferators such as hypolipidemic drugs and fatty acids. Has a preference for poly-unsaturated fatty acids, such as gamma-linoleic acid and eicosapentanoic acid. Once activated by a ligand, the receptor binds to promoter elements of target genes. Regulates the peroxisomal beta-oxidation pathway of fatty acids. Functions as transcription activator for the acyl-CoA oxidase gene. Decreases expression of NPC1L1 once activated by a ligand

Subcellular location

Nucleus
Domains and Gene Ontology detail (70)

Domains & features

NR LBD

Gene Ontology

  • Cchromatin
  • Cnucleoplasm
  • Cnucleus
  • CRNA polymerase II transcription regulator complex
  • FDNA binding
  • FDNA-binding transcription factor activity
  • FDNA-binding transcription factor activity, RNA polymerase II-specific
  • FDNA-binding transcription repressor activity, RNA polymerase II-specific
  • Flinoleic acid binding
  • Flipid binding
  • FNF-kappaB binding
  • Fnuclear receptor activity

441 aa · 50 kDa · 4 isoforms

Biological roles

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Lipid & lipoprotein metabolismUniProt · GONuclear receptor signallingUniProt · GOCell proliferation & survivalGOCell migrationGOTranscriptional regulationUniProt · GOImmune signallingGO
View supporting evidence

Lipid & lipoprotein metabolism

  • ·Ligand-activated transcription factor key mediator of energy metabolism in adipose tissu…
  • ·cholesterol metabolic process
  • ·fatty acid beta-oxidation
  • ·fatty acid catabolic process

Nuclear receptor signalling

  • ·Ligand-activated transcription factor key mediator of energy metabolism in adipose tissu…
  • ·nuclear receptor activity

Cell proliferation & survival

  • ·cell population proliferation

Cell migration

  • ·negative regulation of smooth muscle cell migration

Transcriptional regulation

  • ·Ligand-activated transcription factor key mediator of energy metabolism in adipose tissu…
  • ·RNA polymerase II transcription regulator complex
  • ·DNA-binding transcription factor activity
  • ·DNA-binding transcription factor activity, RNA polymerase II-specific

Immune signalling

  • ·negative regulation of inflammatory response
  • ·positive regulation of fat cell differentiation

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Approved medicines with mapped indications

1 medicine · 1 area

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Cholangitis1 medicine
elafibranor

3 medicines meet Open Targets' target-level approved-medicine definition; the 1 shown here are those on this page with a canonical approved disease indication in the graph. Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

2

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

elafibranor
Narrow target profileApprovedAgonist

Peroxisome proliferator-activated receptor delta agonist

Indicated for Cholangitis

Direct interaction with this protein · 1 of 2 recorded protein targets — narrow recorded profile

seladelpar
Narrow target profilePhase 3Agonist

Peroxisome proliferator-activated receptor delta agonist

Direct interaction with this protein · Only this protein recorded as a target

ChEMBL mechanism, action type, target identity and approved indications. Open Targets clinical status.

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene PPARD

Gene-level evidence surfaced through the gene PPARDthat encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Biliary liver cirrhosis
0.67Moderately supported

Clinical evidence dominant · Open Targets 0.51

Primary biliary cholangitis
0.57Moderately supported

Clinical evidence dominant · Open Targets 0.42

Diabetes Mellitus, Type 2
0.53Moderately supported

Clinical evidence dominant · Open Targets 0.40

Hyperlipidemias
0.49Limited support

Clinical evidence dominant · Open Targets 0.40

Cardiovascular Diseases
0.47Limited support

Clinical evidence dominant · Open Targets 0.37

View evidence synthesis (5)
Biliary liver cirrhosisModerately supported
0.67
agreement 0.54–0.80
Clinical83%Animal model15%Literature2%

Open Targets aggregate 0.51 · 3 independent evidence families

Primary biliary cholangitisModerately supported
0.57
agreement 0.44–0.70
Clinical79%Animal model18%Literature4%

Open Targets aggregate 0.42 · 3 independent evidence families

Diabetes Mellitus, Type 2Moderately supported
0.53
agreement 0.38–0.69
Clinical80%Literature20%

Open Targets aggregate 0.40 · 2 independent evidence families

HyperlipidemiasLimited support
0.49
agreement 0.34–0.65
Clinical99%Literature1%

Open Targets aggregate 0.40 · 2 independent evidence families

Cardiovascular DiseasesLimited support
0.47
agreement 0.32–0.63
Clinical95%Literature6%

Open Targets aggregate 0.37 · 2 independent evidence families

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the per-type scores above show the calculation.

Show all associations
Biliary liver cirrhosis0.51
Neurodegenerative Diseases0.44
Primary biliary cholangitis0.42
Diabetes Mellitus, Type 20.40
Hyperlipidemias0.40
Cardiovascular Diseases0.37
Non-alcoholic Fatty Liver Disease0.36

Drug development

14 compounds recorded · 3 approved · 11 in clinical development

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 2 drugs that target this protein in Forefront's canonical graph (1 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (10)
KD3010Phase 1
CER-002Phase 1
AVE0847Phase 2
GW501516Phase 2
ELAFIBRANORApproval
DB959Phase 2 3
FONADELPARPhase 3
BEZAFIBRATEApproval
INDEGLITAZARPhase 2
CHIGLITAZARPhase 3

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small molecules — Strong

Approved Drug and Structure with Ligand support this modality.

Protein degraders — Emerging

Feasibility evidence (uniprot ubiquitination and half-life data) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (8)
SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyPR · UniProt UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Safety-related annotations

regulation of transcription factor activityToxCastIncreased, Liver SteatosisAOP-Wikidrug toxicityClinPGx

Terms indexed against this target in Open Targets' safety data, with their datasource. These are annotations, not causal claims: the direction of effect (whether activation or inhibition is implicated), species and evidence strength are not captured here, so an entry does not mean that modulating this target is known to cause that condition.

Clinical trials

26

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

A Phase III, Multicentre, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elafibranor in Adult Participants With Primary Sclerosing CholangitisPHASE3

RECRUITING · via elafibranor · NCT07387549

AFFIRM: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Seladelpar on Clinical Outcomes in Patients With Primary Biliary Cholangitis (PBC) and Compensated CirrhosisPHASE3

RECRUITING · via seladelpar · NCT06051617

A Phase III, Open-label, Single Arm Study to Investigate the Efficacy and Safety of Elafibranor 80 mg in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)PHASE3

ACTIVE_NOT_RECRUITING · via elafibranor · NCT06730061

A Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants With Primary Biliary Cholangitis (PBC)PHASE3

RECRUITING · via elafibranor · NCT06016842

View all trials (22)
A Double-blind, Randomized, Placebo-Controlled Study and Open-label Long Term Extension to Evaluate the Efficacy and Safety of Elafibranor 80 mg in Patients With Primary Biliary Cholangitis With Inadequate Response or Intolerance to Ursodeoxycholic AcidPHASE3

ACTIVE_NOT_RECRUITING · via elafibranor · NCT04526665

ASSURE: An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Seladelpar in Subjects With Primary Biliary Cholangitis (PBC)PHASE3

ACTIVE_NOT_RECRUITING · via seladelpar · NCT03301506

The SELIC Trial Seladelpar for the Treatment of Ischemic Cholangiopathy: An Open-Label, Single-Arm, Investigator-Initiated StudyPHASE2

NOT_YET_RECRUITING · via seladelpar · NCT07305363

A Phase II, Multicenter, Double-Blind, Randomised, Placebo-Controlled Study and Open Label Long Term Extension to Evaluate the Safety and Efficacy of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis (PSC).PHASE2

ACTIVE_NOT_RECRUITING · via elafibranor · NCT05627362

A Phase IIIb Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Effect of Elafibranor 80 mg on Normalisation of Alkaline Phosphatase in Adult Participants With Primary Biliary Cholangitis (PBC) and Inadequate Response or Intolerance to Ursodeoxycholic Acid.PHASE3

COMPLETED · via elafibranor · NCT06383403

IDEAL: A 52-week, Double-blind, Placebo-controlled, Randomized, Phase 3 Study Intended to Determine the Effects of Seladelpar on Normalization of Alkaline Phosphatase Levels in Subjects With Primary Biliary Cholangitis (PBC) and an Incomplete Response or Intolerance to Ursodeoxycholic Acid (UDCA)PHASE3

COMPLETED · via seladelpar · NCT06060665

RESPONSE: A Placebo-controlled, Randomized, Phase 3 Study to Evaluate the Efficacy and Safety of Seladelpar in Patients With Primary Biliary Cholangitis (PBC) and an Inadequate Response to or an Intolerance to Ursodeoxycholic Acid (UDCA)PHASE3

COMPLETED · via seladelpar · NCT04620733

A 52-week, Placebo-controlled, Randomized, Phase 3 Study to Evaluate the Safety and Efficacy of Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and an Inadequate Response to or an Intolerance to Ursodeoxycholic Acid (UDCA)PHASE3

COMPLETED · via seladelpar · NCT03602560

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Evaluate the Efficacy and Safety of Elafibranor in Patients With Nonalcoholic Steatohepatitis (NASH) and FibrosisPHASE3

TERMINATED · via elafibranor · NCT02704403

A Phase 2, Randomized, Double Blind, Placebo Controlled, Multiple Center Study to Evaluate the Safety, Tolerability, and Efficacy of Seladelpar Administered for 24 Weeks in Adult Patients With Primary Sclerosing Cholangitis (PSC)PHASE2

TERMINATED · via seladelpar · NCT04024813

A Double-Blind, Placebo-Controlled, Cross-over Phase II Study to Evaluate the Effect of a 6-week Elafibranor (120mg) Treatment Administered Once Daily on Hepatic Lipid Composition in Subjects With Nonalcoholic Fatty Liver (NAFL)PHASE2

TERMINATED · via elafibranor · NCT03953456

An Open Label, Randomized, Multicenter Study to Assess the Pharmacokinetic and Pharmacodynamic Profile and the Safety and Tolerability of Two Dose Levels of Elafibranor (80 mg and 120 mg) in Children and Adolescents, 8 to 17 Years of Age, With Nonalcoholic Steatohepatitis (NASH)PHASE2

TERMINATED · via elafibranor · NCT03883607

A Phase 2, Double-Blind, Randomized, Placebo-Controlled Study Followed by an Open-Label Extension Period to Evaluate the Activity of Seladelpar in Subjects With Nonalcoholic Steatohepatitis (NASH)PHASE2

TERMINATED · via seladelpar · NCT03551522

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate the Efficacy and Safety of Elafibranor at Doses of 80 mg and 120mg After 12 Weeks of Treatment in Patients With Primary Biliary Cholangitis and Inadequate Response to Ursodeoxycholic AcidPHASE2

COMPLETED · via elafibranor · NCT03124108

A Phase I, Open-Label, Randomised, Balanced, Single-Dose, Two-Period, Two-Sequence Crossover-Design Study to Evaluate Effects of Food on the Bioavailability of 80 mg Elafibranor (IPN60190) To-be-marketed Tablet Formulation After Single Oral Administration in Healthy Adult ParticipantsPHASE1

COMPLETED · via elafibranor · NCT05564208

A Phase I, Single-Centre, Open-Label, Repeat Dose Study to Assess the Pharmacokinetics, Safety and Tolerability Following Administration of Elafibranor in Healthy Japanese and Non-Asian Participants.PHASE1

COMPLETED · via elafibranor · NCT05543369

The Effect of Hepatic Impairment on The Pharmacokinetics of Seladelpar: An Open-Label Study Following Oral Dosing of Seladelpar to Subjects With Primary Biliary Cholangitis (PBC) and Hepatic ImpairmentPHASE1

COMPLETED · via seladelpar · NCT04950764

Open-label, Phase 1, Single-dose Study to Evaluate the Pharmacokinetics of Elafibranor 120 mg in Healthy Elderly and Young Adults VolunteersPHASE1

COMPLETED · via elafibranor · NCT04171752

Phase 1, Open-label Study, in Healthy Male Volunteers to Evaluate the Potential Effect of Indomethacin on the Pharmacokinetics Parameters of ElafibranorPHASE1

COMPLETED · via elafibranor · NCT03985969

Open Label, Phase I Study to Assess and Compare the Pharmacokinetic Parameters After Single Oral Administration of Elafibranor 120 mg in Renal Impaired Patients and Healthy Subjects With Normal Renal FunctionPHASE1

COMPLETED · via elafibranor · NCT03844555

An Open-label, Phase 1, Single-dose Study to Evaluate the Pharmacokinetics of Elafibranor 120 mg in Adult Subjects With Hepatic Impairment and Adult Healthy Control SubjectsPHASE1

COMPLETED · via elafibranor · NCT03765671

A Phase 1, Open Label, Non-Randomized Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Oral Seladelpar in Subjects With Varying Degrees of Hepatic Impairment and Healthy Matched Control Subjects With Normal Hepatic FunctionPHASE1

COMPLETED · via seladelpar · NCT03369002

ClinicalTrials.gov via the drug-target graph.

What's happening now

3

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for a drug that targets this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

  1. Label change2026-08-07

    Label change: ELAFIBRANOR (NDA218860)

    fda · regulatory · fda · via elafibranor

  2. Regulatory approval2024-09-19

    Approval: Iqirvo (EMA)

    ema · regulatory · ema · via elafibranor

  3. Regulatory approval2024-06-10

    Approval: ELAFIBRANOR (NDA218860)

    fda · regulatory · fda · via elafibranor

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.

Related family literature

4

Papers about “Peroxisome Proliferator-Activated Receptors” — a broader family this protein belongs to. Shown as context; not counted as papers specifically about this protein.

Peroxisome proliferator-activated receptors as targets to treat metabolic diseases: Focus on the adipose tissue, liver, and pancreas.

Souza-Tavares H · World journal of gastroenterology · 2023

via Peroxisome Proliferator-Activated Receptors

Redox regulation of the immune response.

Morris G · Cellular & molecular immunology · 2022

via Peroxisome Proliferator-Activated Receptors

PPARs as Metabolic Regulators in the Liver: Lessons from Liver-Specific PPAR-Null Mice.

Wang Y · International journal of molecular sciences · 2020

via Peroxisome Proliferator-Activated Receptors

Inflammatory links between obesity and metabolic disease.

Lumeng CN · The Journal of clinical investigation · 2011

via Peroxisome Proliferator-Activated Receptors

Europe PMC literature, reached through curated HGNC family membership. Membership is a taxonomic relationship — it does not imply this protein participates in every mechanism these papers discuss.