Danicopan
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Also known as Ach-0144471ACH-4471Voydeya
Summary
Danicopan (CHEMBL4250860) is an approved small molecule (ATC L04AJ09) targeting CFD; indicated across 10 conditions including hemoglobinuria and membranoproliferative glomerulonephritis.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: L04AJ09
- Targets: 1 (CFD)
- Indications: 10 conditions
- Clinical trials: 24
- Chemistry: 580.4 Da · C26H23BrFN7O3
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL4250860 |
| Name | Danicopan |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 118323590 |
| ATC | L04AJ09 |
| Molecular formula | C26H23BrFN7O3 |
| Molecular weight | 580.4 |
| InChIKey | PIBARDGJJAGJAJ-NQIIRXRSSA-N |
SMILES: CC1=NC=C(C=N1)C2=CC3=C(C=C2)N(N=C3C(=O)C)CC(=O)N4C[C@@H](C[C@H]4C(=O)NC5=NC(=CC=C5)Br)F
IUPAC name: (2S,4R)-1-[2-[3-acetyl-5-(2-methylpyrimidin-5-yl)indazol-1-yl]acetyl]-N-(6-bromo-2-pyridinyl)-4-fluoropyrrolidine-2-carboxamide
Also known as: Ach-0144471, ACH-0144471, ACH-4471, Danicopan, Voydeya, DANICOPAN
Patent coverage: 104 distinct patent families (290 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 210 (72%) 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 |
|---|---|---|---|---|---|
| CFD | complement factor D | Inhibition | 9 | 0.7% | P00746 |
Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Complement factor D.
Bioactivity
ChEMBL activities: 3 potent at pChembl ≥ 5 of 3 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| CFD | 8.4 | IC50 | 4 | nM | CHEMBL_ACT_18652345 |
| CFD | 8.4 | IC50 | 4 | nM | CHEMBL_ACT_18652378 |
| CFD | 7.82 | IC50 | 15 | nM | CHEMBL_ACT_18652379 |
Target pathways
Aggregated over 1 target gene(s): CFD.
Top Reactome pathways
3 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Platelet degranulation | 1 | CFD |
| Alternative complement activation | 1 | CFD |
| Neutrophil degranulation | 1 | CFD |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| proteolysis | 1 |
| complement activation | 1 |
| complement activation, alternative pathway | 1 |
| response to bacterium | 1 |
| zymogen activation | 1 |
| protein maturation | 1 |
| immune system process | 1 |
| innate immune response | 1 |
Indications & clinical
Indications
10 indications (3 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 |
|---|---|---|---|
| hemoglobinuria | 3 | MONDO:0003656 | MONDO:0100244 |
| membranoproliferative glomerulonephritis | 2 | MONDO:0002461 | MONDO:0019736 |
| severe acute respiratory syndrome | 2 | MONDO:0005091 | MONDO:0100096 |
| liver disorder | 1 | MONDO:0005154 | EFO:0001421 |
| kidney disorder | 1 | MONDO:0005240 | EFO:0003086 |
5 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 24.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE1 | 10 |
| PHASE2 | 8 |
| PHASE3 | 3 |
| Not specified | 3 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT05389449 | PHASE3 | ACTIVE_NOT_RECRUITING | A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH |
| NCT06449001 | PHASE3 | RECRUITING | Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis |
| NCT04469465 | PHASE3 | COMPLETED | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) |
| NCT03053102 | PHASE2 | COMPLETED | Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| NCT03124368 | PHASE2 | COMPLETED | A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN |
| NCT03181633 | PHASE2 | COMPLETED | A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| NCT03369236 | PHASE2 | COMPLETED | A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G) |
| NCT03459443 | PHASE2 | TERMINATED | A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471 |
| NCT03472885 | PHASE2 | COMPLETED | Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab |
| NCT04988035 | PHASE2 | COMPLETED | ACTIV-5 / Big Effect Trial (BET-C) for the Treatment of COVID-19 |
| NCT05019521 | PHASE2 | TERMINATED | A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration |
| NCT03108274 | PHASE1 | COMPLETED | A Drug Interaction Study Between Danicopan and Midazolam, Fexofenadine, and Mycophenolate Mofetil in Healthy Participants |
| NCT03555539 | PHASE1 | COMPLETED | Study of Danicopan in Participants With Hepatic Impairment |
| NCT04451434 | PHASE1 | COMPLETED | Study of Danicopan in Participants of Japanese Descent |
| NCT04551599 | PHASE1 | COMPLETED | A Study of the Effects of Food and Age on Danicopan |
| NCT04709094 | PHASE1 | COMPLETED | A Drug Interaction Study of Danicopan |
| NCT04889677 | PHASE1 | COMPLETED | Study of a Single Dose of Danicopan in Healthy Participants |
| NCT04889690 | PHASE1 | COMPLETED | Study of Multiple Doses of Danicopan in Healthy Participants |
| NCT04935294 | PHASE1 | COMPLETED | Study of Danicopan in Participants With Normal Kidney Function and Participants With Kidney Dysfunction |
| NCT05016206 | PHASE1 | COMPLETED | A Study of the Cardiac Effects of Danicopan in Healthy Adults |
| NCT05109390 | PHASE1 | COMPLETED | A Study of the Drug Interactions Between Danicopan and Cyclosporine, Tacrolimus, Antacids, and Omeprazole in Healthy Adults |
| NCT05982938 | Not specified | AVAILABLE | Danicopan Early Access Program |
| NCT07413250 | Not specified | ACTIVE_NOT_RECRUITING | Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data |
| NCT07413679 | Not specified | ACTIVE_NOT_RECRUITING | Long-term Safety of Danicopan: IPIG Registry-based Cohort Study |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
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.
1 molecules share ≥1 primary target. Top 1 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| Iptacopan | PubChem | Approved | CFD |