Avacopan
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Also known as CCX-168Ccx168TavneosAVACOPAN (CCX168)
Summary
Avacopan (CHEMBL3989871) is an approved small molecule (ATC L04AJ05) targeting C5AR1; indicated across 10 conditions including anti-neutrophil antibody associated vasculitis and granulomatosis with polyangiitis.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: L04AJ05
- Targets: 1 (C5AR1)
- Indications: 10 conditions
- Clinical trials: 22
- Chemistry: 581.6 Da · C33H35F4N3O2
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL3989871 |
| Name | Avacopan |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 49841217 |
| ATC | L04AJ05 |
| Molecular formula | C33H35F4N3O2 |
| Molecular weight | 581.6 |
| InChIKey | PUKBOVABABRILL-YZNIXAGQSA-N |
SMILES: CC1=C(C(=CC=C1)F)C(=O)N2CCC[C@@H]([C@@H]2C3=CC=C(C=C3)NC4CCCC4)C(=O)NC5=CC(=C(C=C5)C)C(F)(F)F
IUPAC name: (2R,3S)-2-[4-(cyclopentylamino)phenyl]-1-(2-fluoro-6-methylbenzoyl)-N-[4-methyl-3-(trifluoromethyl)phenyl]piperidine-3-carboxamide
Also known as: Avacopan, CCX-168, Ccx168, CCX168, Tavneos, AVACOPAN, TAVNEOS, AVACOPAN (CCX168), avacopan
Patent coverage: 222 distinct patent families (581 SureChEMBL compound mentions), from 4 matched compound structure(s). One matched structure accounts for 547 (94%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.
Targets
Targets
Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).
| Gene | Target | Action | pAffinity | Cancer dependency | UniProt |
|---|---|---|---|---|---|
| C5AR1 | C5a1 receptor | Antagonist | 9.7 | 0.1% | P21730 |
Bioactivity
No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).
Target pathways
Aggregated over 1 target gene(s): C5AR1.
Top Reactome pathways
12 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Signal Transduction | 1 | C5AR1 |
| Complement cascade | 1 | C5AR1 |
| Innate Immune System | 1 | C5AR1 |
| Immune System | 1 | C5AR1 |
| Signaling by GPCR | 1 | C5AR1 |
| Class A/1 (Rhodopsin-like receptors) | 1 | C5AR1 |
| Peptide ligand-binding receptors | 1 | C5AR1 |
| GPCR downstream signalling | 1 | C5AR1 |
| G alpha (i) signalling events | 1 | C5AR1 |
| GPCR ligand binding | 1 | C5AR1 |
| Neutrophil degranulation | 1 | C5AR1 |
| Regulation of Complement cascade | 1 | C5AR1 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| microglial cell activation | 1 |
| complement receptor mediated signaling pathway | 1 |
| chemotaxis | 1 |
| inflammatory response | 1 |
| immune response | 1 |
| cellular defense response | 1 |
| signal transduction | 1 |
| adenylate cyclase-inhibiting G protein-coupled receptor signaling pathway | 1 |
| phospholipase C-activating G protein-coupled receptor signaling pathway | 1 |
| positive regulation of cytosolic calcium ion concentration | 1 |
| sensory perception of chemical stimulus | 1 |
| positive regulation of vascular endothelial growth factor production | 1 |
| positive regulation of macrophage chemotaxis | 1 |
| neutrophil chemotaxis | 1 |
| response to peptidoglycan | 1 |
Indications & clinical
Indications
10 indications (4 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| anti-neutrophil antibody associated vasculitis | 4 | MONDO:0005435 | EFO:0004826 |
| granulomatosis with polyangiitis | 4 | MONDO:0012105 | EFO:0005297 |
| microscopic polyangiitis | 4 | MONDO:0019124 | EFO:1000784 |
| anti-neutrophil cytoplasmic antibody-associated vasculitis | 4 | MONDO:0015492 | MONDO:0015492 |
| vasculitis | 3 | MONDO:0018882 | EFO:0006803 |
| IgA glomerulonephritis | 2 | MONDO:0005342 | EFO:0004194 |
| hidradenitis suppurativa | 2 | MONDO:0006559 | EFO:1000710 |
| atypical hemolytic-uremic syndrome | 2 | MONDO:0016244 | MONDO:0016244 |
| liver disorder | 1 | MONDO:0005154 | EFO:0001421 |
| chronic kidney disease | 1 | MONDO:0005300 | EFO:0003884 |
Clinical trials
Total trials: 22.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE1 | 9 |
| PHASE2 | 7 |
| PHASE3 | 3 |
| PHASE4 | 1 |
| PHASE2/PHASE3 | 1 |
| Not specified | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT06072482 | PHASE4 | RECRUITING | A Study to Evaluate Avacopan in Participants With ANCA-associated Vasculitis |
| NCT06321601 | PHASE3 | RECRUITING | Study to Evaluate Avacopan in Combination With a Rituximab or Cyclophosphamide-containing Regimen, in Children From 6 Years to < 18 Years of Age With AAV. |
| NCT07176546 | PHASE2/PHASE3 | NOT_YET_RECRUITING | TAVNEOS for Otolaryngologic Manifestations of Granulomatosis With Polyangiitis |
| NCT07373262 | PHASE3 | NOT_YET_RECRUITING | Avacopan Added to Standard-of-care Therapy in ANCA-associated Vasculitis With Severe Kidney Involvement |
| NCT02994927 | PHASE3 | COMPLETED | A Phase 3 Clinical Trial of CCX168 (Avacopan) in Patients With ANCA-Associated Vasculitis |
| NCT06676579 | PHASE2 | RECRUITING | Avacopan in Crescentic Immunoglobulin A Nephropathy (IgAN) |
| NCT01363388 | PHASE2 | COMPLETED | A Study to Evaluate the Safety and Efficacy of CCX168 in Subjects With ANCA-Associated Vasculitis |
| NCT02222155 | PHASE2 | COMPLETED | Clinical Trial to Evaluate Safety and Efficacy of CCX168 in ANCA-Associated Vasculitis |
| NCT02384317 | PHASE2 | COMPLETED | Open-Label Study to Evaluate Safety and Efficacy of CCX168 in Subjects With IGA Nephropathy on Stable RAAS Blockade |
| NCT02464891 | PHASE2 | TERMINATED | Complement Inhibition in aHUS Dialysis Patients |
| NCT03301467 | PHASE2 | COMPLETED | Controlled Trial Evaluating Avacopan in C3 Glomerulopathy |
| NCT03852472 | PHASE2 | COMPLETED | Evaluation of Safety and Efficacy of Avacopan in Subjects With Moderate to Severe Hidradenitis Suppurativa (AURORA) |
| NCT07556484 | PHASE1 | RECRUITING | Pharmacokinetics Of Emulsified Avacopan Applied By NG Tube |
| NCT05984251 | PHASE1 | COMPLETED | A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CCX168 in Healthy Participants |
| NCT05988008 | PHASE1 | COMPLETED | A Study of CCX168 in Japanese and Caucasian Healthy Adult Males |
| NCT05988021 | PHASE1 | COMPLETED | A Study in Healthy Volunteers to Evaluate the Pharmacokinetic Food Effect and Cardiac Safety of CCX168 |
| NCT05988034 | PHASE1 | COMPLETED | A Study to Assess the Effect of Avacopan at Therapeutic and Supratherapeutic Doses on the QT/QTc Interval in Healthy Participants |
| NCT06004934 | PHASE1 | COMPLETED | A Study to Evaluate the Pharmacokinetics of Avacopan (CCX168) in Participants With Mild or Moderate Hepatic Impairment |
| NCT06004947 | PHASE1 | COMPLETED | A Study in Healthy Volunteers to Evaluate the Drug-Drug Interaction Potential of CCX168 With Concomitant Medications |
| NCT06207682 | PHASE1 | COMPLETED | Effect of Repeated Oral Doses of Avacopan on the Pharmacokinetics (PK) of a Single Dose of Simvastatin |
| NCT06468826 | PHASE1 | COMPLETED | A Study of Avacopan in Participants With Normal Renal Function and Participants With End-Stage Renal Disease (ESRD) |
| NCT06758271 | Not specified | RECRUITING | Special Drug Use-results Survey for Long-term Use（Avacopan） |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.
Related molecules
Related molecules
Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.
2 molecules share ≥1 primary target. Top 2 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| LOPERAMIDE | ChEMBL | Phase 4 (approved) | C5AR1 |
| Belzutifan | PubChem | Approved | C5AR1 |