Crofelemer
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Also known as FulyzaqMytesiNP-303ProvirSP-303TRN-002Virend
Summary
Crofelemer (CHEMBL2108184) is an approved small molecule (ATC A07XA06) targeting CFTR and ANO1; indicated across 3 conditions including diarrheal disease and irritable bowel syndrome.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: A07XA06
- Targets: 2 (CFTR, ANO1)
- Indications: 3 conditions
- Clinical trials: 12
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL2108184 |
| Name | Crofelemer |
| Type | Small molecule |
| Max phase | 4 |
| ATC | A07XA06 |
Also known as: Crofelemer, Fulyzaq, Mytesi, NP-303, Provir, SP-303, TRN-002, Virend, CROFELEMER
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 |
|---|---|---|---|---|---|
| CFTR | CFTR | Inhibition | 5.15 | 0.1% | P13569 |
| ANO1 | CaCC | Inhibition | 5.19 | 1.2% | Q5XXA6 |
Bioactivity
No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).
Target pathways
Aggregated over 2 target gene(s): CFTR, ANO1.
Top Reactome pathways
21 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Disease | 1 | ANO1 |
| Stimuli-sensing channels | 1 | ANO1 |
| Transport of small molecules | 1 | ANO1 |
| ABC-family protein mediated transport | 1 | CFTR |
| RHO GTPases regulate CFTR trafficking | 1 | CFTR |
| Infectious disease | 1 | ANO1 |
| Defective CFTR causes cystic fibrosis | 1 | CFTR |
| Ub-specific processing proteases | 1 | CFTR |
| Cargo recognition for clathrin-mediated endocytosis | 1 | CFTR |
| Clathrin-mediated endocytosis | 1 | CFTR |
| RHOQ GTPase cycle | 1 | CFTR |
| Chaperone Mediated Autophagy | 1 | CFTR |
| Late endosomal microautophagy | 1 | CFTR |
| Aggrephagy | 1 | CFTR |
| SARS-CoV Infections | 1 | ANO1 |
| SARS-CoV-2 Infection | 1 | ANO1 |
| Induction of Cell-Cell Fusion | 1 | ANO1 |
| Late SARS-CoV-2 Infection Events | 1 | ANO1 |
| Viral Infection Pathways | 1 | ANO1 |
| Ion channel transport | 1 | ANO1 |
| Developmental Lineage of Pancreatic Ductal Cells | 1 | CFTR |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| chloride transmembrane transport | 2 |
| monoatomic ion transport | 2 |
| chloride transport | 2 |
| monoatomic ion transmembrane transport | 2 |
| cholesterol biosynthetic process | 1 |
| water transport | 1 |
| bicarbonate transport | 1 |
| cholesterol transport | 1 |
| response to endoplasmic reticulum stress | 1 |
| transepithelial water transport | 1 |
| sperm capacitation | 1 |
| multicellular organismal-level water homeostasis | 1 |
| intracellular pH elevation | 1 |
| establishment of localization in cell | 1 |
| transmembrane transport | 1 |
Indications & clinical
Indications
3 indications (1 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 |
|---|---|---|---|
| diarrheal disease | 4 | MONDO:0001673 | HP:0002014 |
| irritable bowel syndrome | 2 | MONDO:0005052 | EFO:0000555 |
| breast neoplasm | 2 | MONDO:0021100 | MONDO:0007254 |
Clinical trials
Total trials: 12.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 4 |
| PHASE3 | 3 |
| PHASE4 | 2 |
| Not specified | 2 |
| EARLY_PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT03898856 | PHASE4 | COMPLETED | Yield of Diagnostic Tests and Effects of Crofelemer for Chronic Idiopathic Diarrhea In Non-HIV Patients |
| NCT04486326 | PHASE4 | TERMINATED | Crofelemer for Functional Diarrhea |
| NCT00002408 | PHASE3 | COMPLETED | A Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Efficacy and Safety of Orally Administered SP-303 for the Treatment of Diarrhea in Acquired Immunodeficiency Syndrome (AIDS) Patients |
| NCT00547898 | PHASE3 | COMPLETED | Safety and Effectiveness of 3 Doses of Crofelemer Compared to Placebo in the Treatment of HIV Associated Diarrhea |
| NCT01374490 | PHASE3 | COMPLETED | Safety and Tolerability of Crofelemer for HIV-Associated Diarrhea |
| NCT00101725 | PHASE2 | COMPLETED | A Study of Crofelemer to Treat Diarrhea Irritable Bowel Syndrome |
| NCT00461526 | PHASE2 | COMPLETED | Diarrhea Predominant Irritable Bowel Syndrome in Females |
| NCT02910219 | PHASE2 | COMPLETED | Diarrhea Prevention and Prophylaxis With Crofelemer in HER2 Positive Breast Cancer Patients |
| NCT03094052 | PHASE2 | COMPLETED | Incidence and Severity of Diarrhea in Patients With HER2 Positive Breast Cancer Treated With Trastuzumab and Neratinib |
| NCT06326645 | EARLY_PHASE1 | RECRUITING | Open-Label Pilot Study With Crofelemer in Patients With Short Bowel Syndrome |
| NCT00002186 | Not specified | COMPLETED | Double-Blind Placebo-Controlled Study Comparing the Combination of 15% SP-303 Gel With Acyclovir Versus Acyclovir Alone for the Treatment of Recurrent Herpes Simplex Virus (HSV) Infections in Subjects With Acquired Immunodeficiency Syndrome (AIDS) |
| NCT00002310 | Not specified | COMPLETED | SP-303T Applied to the Skin of Patients With Herpes Simplex Virus (HSV) Infection and AIDS Who Have Not Had Success With Acyclovir |
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.
17 molecules share ≥1 primary target. Top 17 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| IVACAFTOR | ChEMBL + PubChem | Phase 4 (approved) | CFTR |
| NITAZOXANIDE | ChEMBL + PubChem | Phase 4 (approved) | ANO1 |
| ELEXACAFTOR | ChEMBL | Phase 4 (approved) | CFTR |
| GLYBURIDE | ChEMBL | Phase 4 (approved) | CFTR |
| LUMACAFTOR | ChEMBL | Phase 4 (approved) | CFTR |
| NICLOSAMIDE | ChEMBL | Phase 4 (approved) | ANO1 |
| TEZACAFTOR | ChEMBL | Phase 4 (approved) | CFTR |
| BAMOCAFTOR | ChEMBL | Phase 3 | CFTR |
| QUERCETIN | ChEMBL | Phase 3 | CFTR |
| RUTIN | ChEMBL | Phase 3 | CFTR |
| GALICAFTOR | ChEMBL | Phase 2 | CFTR |
| GENISTEIN | ChEMBL | Phase 2 | CFTR |
| GLPG-2737 | ChEMBL | Phase 2 | CFTR |
| ICENTICAFTOR | ChEMBL | Phase 2 | CFTR |
| NAVOCAFTOR | ChEMBL | Phase 2 | CFTR |
| RISELCAFTOR | ChEMBL | Phase 2 | CFTR |
| Tadalafil | PubChem | Approved | CFTR |
Related Atlas pages
- Genes: CFTR, ANO1
- Diseases: diarrheal disease
- Drugs: Ivacaftor, Nitazoxanide, Elexacaftor, Glyburide, Lumacaftor, Niclosamide, Tezacaftor, Bamocaftor, Quercetin, Rutin, Tadalafil