Pomalidomide
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Also known as ActimidCC-4047IMID 3IMID-3IMID3ImnovidPomalidomidaPomalidomide accordPomalidomide krkaPomalidomide viatrisPomalidomide zentivaPomalystSID174007398Cpd P
Summary
Pomalidomide (CHEMBL43452) is an approved small-molecule antineoplastic agent (ATC L04AX06) targeting CRBN; indicated across 34 conditions including immune system disorder and plasma cell myeloma.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: L04AX06
- Targets: 1 (CRBN)
- Indications: 34 conditions
- Clinical trials: 255
- Chemistry: 273.24 Da · C13H11N3O4
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL43452 |
| Name | Pomalidomide |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 134780 |
| ChEBI | CHEBI:72690 |
| ATC | L04AX06 |
| Molecular formula | C13H11N3O4 |
| Molecular weight | 273.24 |
| InChIKey | UVSMNLNDYGZFPF-UHFFFAOYSA-N |
SMILES: C1CC(=O)NC(=O)C1N2C(=O)C3=C(C2=O)C(=CC=C3)N
IUPAC name: 4-amino-2-(2,6-dioxopiperidin-3-yl)isoindole-1,3-dione
ChEBI definition: An aromatic amine that is thalidomide substituted at position 4 on the isoindole ring system by an amino group. Used for the treatment of multiple myeloma in patients who failed to respond to previous therapies.
Pharmacological roles (ChEBI): antineoplastic agent, immunomodulator, angiogenesis inhibitor.
Also known as: Actimid, CC-4047, IMID 3, IMID-3, IMID3, Imnovid, Pomalidomida, Pomalidomide, Pomalidomide accord, Pomalidomide krka, Pomalidomide viatris, Pomalidomide zentiva
Patent coverage: 5,204 distinct patent families (13,354 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 12,488 (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 |
|---|---|---|---|---|---|
| CRBN | cereblon | Binding | 5.92 | 0.2% | Q96SW2 |
Broader ChEMBL bioactivity targets: 10 (assay-derived). Sample: Tumor necrosis factor, Interleukin-1 beta, Thromboxane A2 receptor, Protein cereblon, Glyceraldehyde-3-phosphate dehydrogenase, cytosolic, Cereblon/Ikaros, Protein cereblon/Zinc finger protein Aiolos, Protein cereblon/DNA damage-binding protein 1, DNA-binding protein Ikaros, Zinc finger protein Aiolos.
Bioactivity
ChEMBL activities: 22 potent at pChembl ≥ 5 of 25 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| DDB1 | 8.05 | EC50 | 9 | nM | CHEMBL_ACT_24860457 |
| IKZF3 | 7.7 | EC50 | 20 | nM | CHEMBL_ACT_25673170 |
| CRBN | 7.66 | EC50 | 22 | nM | CHEMBL_ACT_18565668 |
| IKZF1 | 7.62 | EC50 | 24 | nM | CHEMBL_ACT_18565671 |
| TNF | 7.48 | IC50 | 33 | nM | CHEMBL_ACT_26678795 |
| IL1B | 7.33 | IC50 | 47 | nM | CHEMBL_ACT_26678780 |
| IKZF1 | 7.3 | AC50 | 50 | nM | CHEMBL_ACT_25733423 |
| IKZF1 | 7.3 | AC50 | 50 | nM | CHEMBL_ACT_25733506 |
| DDB1 | 7.2 | IC50 | 63.1 | nM | CHEMBL_ACT_28439897 |
| CRBN | 7.08 | IC50 | 83 | nM | CHEMBL_ACT_25536968 |
| CRBN | 7.08 | Kd | 83 | nM | CHEMBL_ACT_29075683 |
| CRBN | 6.92 | IC50 | 120 | nM | CHEMBL_ACT_29075709 |
| CRBN | 6.8 | Kd | 157 | nM | CHEMBL_ACT_25848529 |
| DDB1 | 6.4 | IC50 | 400 | nM | CHEMBL_ACT_25673122 |
| CRBN | 6.4 | IC50 | 400 | nM | CHEMBL_ACT_29075649 |
| CRBN | 6.13 | IC50 | 739 | nM | CHEMBL_ACT_25083008 |
| CRBN | 5.92 | IC50 | 1200 | nM | CHEMBL_ACT_18565676 |
| DDB1 | 5.82 | IC50 | 1510 | nM | CHEMBL_ACT_29056726 |
| CRBN | 5.69 | IC50 | 2060 | nM | CHEMBL_ACT_29103056 |
| Q01558 | 5.54 | IC50 | 2900 | nM | CHEMBL_ACT_26678777 |
| CRBN | 5.48 | Ki | 3300 | nM | CHEMBL_ACT_25665465 |
| CRBN | 5 | Ki | 10000 | nM | CHEMBL_ACT_22446525 |
Target pathways
Aggregated over 1 target gene(s): CRBN.
Top Reactome pathways
1 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Potential therapeutics for SARS | 1 | CRBN |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| protein ubiquitination | 1 |
| positive regulation of Wnt signaling pathway | 1 |
| negative regulation of protein-containing complex assembly | 1 |
| positive regulation of protein-containing complex assembly | 1 |
| negative regulation of monoatomic ion transmembrane transport | 1 |
| locomotory exploration behavior | 1 |
| proteasome-mediated ubiquitin-dependent protein catabolic process | 1 |
| limb development | 1 |
Indications & clinical
Indications
34 indications (2 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 |
|---|---|---|---|
| immune system disorder | 4 | MONDO:0005046 | EFO:0000540 |
| plasma cell myeloma | 4 | MONDO:0009693 | EFO:0001378 |
| primary myelofibrosis | 3 | MONDO:0009692 | EFO:0002430 |
| systemic sclerosis | 2 | MONDO:0005100 | EFO:0000717 |
| Kaposi’s sarcoma | 2 | MONDO:0005055 | EFO:0000558 |
| diffuse large B-cell lymphoma | 2 | MONDO:0018905 | EFO:0000403 |
| acquired polycythemia vera | 2 | MONDO:0009891 | EFO:0002429 |
| medulloblastoma | 2 | MONDO:0007959 | EFO:0002939 |
| plasma cell leukemia | 2 | MONDO:0018689 | EFO:0006475 |
| plasmacytoma | 2 | MONDO:0005615 | EFO:0006738 |
| soft tissue sarcoma | 2 | MONDO:0018078 | EFO:1001968 |
| interstitial lung disease | 2 | MONDO:0015925 | EFO:0004244 |
| pulmonary fibrosis | 2 | MONDO:0002771 | EFO:0009448 |
| graft versus host disease | 2 | MONDO:0013730 | MONDO:0013730 |
| smoldering plasma cell myeloma | 2 | MONDO:0005235 | EFO:0003073 |
| hereditary hemorrhagic telangiectasia | 2 | MONDO:0019180 | MONDO:0019180 |
| hereditary amyloidosis | 2 | MONDO:0018634 | MONDO:0019438 |
| plasma cell neoplasm | 2 | MONDO:0004959 | EFO:0000200 |
| central nervous system neoplasm | 2 | MONDO:0006130 | EFO:1000158 |
| amyloidosis | 2 | MONDO:0019065 | EFO:1001875 |
| neoplasm | 1 | MONDO:0005070 | EFO:0000616 |
| small cell lung carcinoma | 1 | MONDO:0008433 | EFO:0000702 |
| exocrine pancreatic carcinoma | 1 | MONDO:0005192 | EFO:0002618 |
| Hodgkins lymphoma | 1 | MONDO:0004952 | EFO:0000183 |
| non-Hodgkin lymphoma | 1 | MONDO:0018908 | EFO:0005952 |
| Waldenstrom macroglobulinemia | 1 | MONDO:0100280 | EFO:0009441 |
| Castleman disease | 1 | MONDO:0015564 | MONDO:0015564 |
| Burkitt lymphoma | 1 | MONDO:0007243 | EFO:0000309 |
| sickle cell disease | 1 | MONDO:0011382 | MONDO:0011382 |
| lymphoma | 1 | MONDO:0005062 | EFO:0000574 |
4 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 255.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 92 |
| PHASE1 | 55 |
| PHASE1/PHASE2 | 48 |
| PHASE3 | 44 |
| Not specified | 14 |
| PHASE4 | 1 |
| EARLY_PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT05183139 | PHASE4 | WITHDRAWN | A Multicenter In-class Transition Study of Ixazomib Combined With Pomalidomide and Dexamethasone or With Lenalidomide and Dexamethasone in Adults With Relapsed/Refractory Multiple Myeloma |
| NCT03539744 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study Designed to Evaluate the Safety and Efficacy of Venetoclax Plus Dexamethasone (VenDex) Compared With Pomalidomide Plus Dexamethasone (PomDex) in Participants With t(11;14)-Positive Relapsed or Refractory Multiple Myeloma. |
| NCT03651128 | PHASE3 | ACTIVE_NOT_RECRUITING | Efficacy and Safety Study of bb2121 Versus Standard Regimens in Subjects With Relapsed and Refractory Multiple Myeloma (RRMM) |
| NCT04181827 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study Comparing JNJ-68284528, a CAR-T Therapy Directed Against B-cell Maturation Antigen (BCMA), Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple Myeloma |
| NCT04484623 | PHASE3 | ACTIVE_NOT_RECRUITING | Belantamab Mafodotin Plus Pomalidomide and Dexamethasone (Pd) Versus Bortezomib Plus Pd in Relapsed/Refractory Multiple Myeloma |
| NCT05020236 | PHASE3 | RECRUITING | A Study to Learn About the Study Medicine Elranatamab Alone and With Daratumumab in People With Multiple Myeloma Who Have Received Other Treatments |
| NCT05028348 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study of Combination of Selinexor, Pomalidomide, and Dexamethasone (SPd) Versus Elotuzumab, Pomalidomide, and Dexamethasone (EloPd) in Subject With Previously Treated Multiple Myeloma |
| NCT05083169 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study of Teclistamab in Combination With Daratumumab Subcutaneously (SC) (Tec-Dara) Versus Daratumumab SC, Pomalidomide, and Dexamethasone (DPd) or Daratumumab SC, Bortezomib, and Dexamethasone (DVd) in Participants With Relapsed or Refractory Multiple Myeloma |
| NCT05405166 | PHASE3 | ACTIVE_NOT_RECRUITING | SC Versus IV Isatuximab in Combination With Pomalidomide and Dexamethasone in RRMM |
| NCT05438043 | PHASE3 | RECRUITING | A Study of Daratumumab |
| NCT05455320 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study Comparing Talquetamab in Combination With Daratumumab or in Combination With Daratumumab and Pomalidomide Versus Daratumumab in Combination With Pomalidomide and Dexamethasone in Participants With Multiple Myeloma That Returns After Treatment or is Resistant to Treatment |
| NCT05519085 | PHASE3 | RECRUITING | A Study to Evaluate Mezigdomide, Bortezomib and Dexamethasone (MEZIVd) Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) in Participants With Relapsed or Refractory Multiple Myeloma (RRMM) |
| NCT05572515 | PHASE3 | ACTIVE_NOT_RECRUITING | A Study Comparing Teclistamab Monotherapy Versus Pomalidomide, Bortezomib, Dexamethasone (PVd) or Carfilzomib, Dexamethasone (Kd) in Participants With Relapsed or Refractory Multiple Myeloma |
| NCT05730036 | PHASE3 | RECRUITING | A Trial to Learn How Well Linvoseltamab Works Compared to the Combination of Elotuzumab, Pomalidomide and Dexamethasone for Adult Participants With Relapsed/Refractory Multiple Myeloma |
| NCT06152575 | PHASE3 | RECRUITING | MagnetisMM-32: A Study to Learn About the Study Medicine Called Elranatamab in People With Multiple Myeloma (MM) That Has Come Back After Taking Other Treatments (Including Prior Treatment With an Anti-CD38 Antibody and Lenalidomide) |
| NCT06158841 | PHASE3 | RECRUITING | Study Assessing Activity of Intravenous (IV) Etentamig Monotherapy Versus Standard Available Therapies in Adult Participants With Relapsed or Refractory Multiple Myeloma |
| NCT06208150 | PHASE3 | RECRUITING | A Study Comparing Talquetamab Plus Pomalidomide, Talquetamab Plus Teclistamab, and Elotuzumab, Pomalidomide, and Dexamethasone or Pomalidomide, Bortezomib, and Dexamethasone in Participants With Relapsed or Refractory Myeloma Who Have Received an Anti-CD38 Antibody and Lenalidomide |
| NCT06413498 | PHASE3 | RECRUITING | A Study Comparing Anitocabtagene Autoleucel to Standard of Care Therapy in Participants With Relapsed/ Refractory Multiple Myeloma |
| NCT06464991 | PHASE3 | RECRUITING | A Phase III Study of Eque-cel in Subjects With Len-refractory RRMM (FUMANBA-03) |
| NCT06508983 | PHASE3 | RECRUITING | A Clinical Study Comparing SG301 Plus Pomalidomide and Dexamethasone to Placebo Plus Pomalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma Patients |
| NCT06615479 | PHASE3 | RECRUITING | A Study to Compare the Efficacy and Safety of BMS-986393 Versus Standard Regimens in Adult Participants With Relapsed or Refractory and Lenalidomide-exposed Multiple Myeloma (QUINTESSENTIAL-2) |
| NCT06742138 | PHASE3 | NOT_YET_RECRUITING | To Evaluate the Efficacy and Safety of QL2109 and DARZALEX FASPRO® in Multiple Myeloma |
| NCT06956170 | PHASE3 | ACTIVE_NOT_RECRUITING | All Japanese Population: Belantamab Mafodotin Plus Pomalidomide and Dexamethasone (Pd) Versus Bortezomib Plus Pd in Relapsed/Refractory Multiple Myeloma |
| NCT07138209 | PHASE3 | RECRUITING | A Study Comparing QLS32015 Monotherapy Versus Pomalidomide, Dexamethasone (Pd) or Selinexor, Dexamethasone (Sd) in Participants With Relapsed or Refractory Multiple Myeloma |
| NCT07222761 | PHASE3 | RECRUITING | A Study to Compare Linvoseltamab Monotherapy and Linvoseltamab + Carfilzomib Combination Therapy With Standard-of-Care Combination Regimens in Adult Participants With Relapsed/Refractory Multiple Myeloma (RRMM) |
| NCT07391657 | PHASE3 | RECRUITING | A Study Comparing AZD0120, a Dual-targeted CAR-T Against B-cell Maturation Antigen (BCMA) and CD19, Versus Standard Regimens in Participants With Relapsed Refractory Multiple Myeloma (DURGA-4) |
| NCT07452198 | PHASE3 | RECRUITING | A Study of GR1803 Injection Versus Daratumumab, Pomalidomide, and Dexamethasone (DPd) in Participants With Relapsed or Refractory Multiple Myeloma |
| NCT07555938 | PHASE3 | RECRUITING | Cevostamab in Combination With Pomalidomide and Dexamethasone Versus Standard of Care in Participants With Previously Treated Multiple Myeloma |
| NCT07569757 | PHASE3 | NOT_YET_RECRUITING | Clinical Study of TQB2934 Injection in Relapsed/Refractory Multiple Myeloma |
| NCT07579234 | PHASE3 | RECRUITING | A Multicenter, Randomized, Open-label, Parallel-group, Controlled, Superiority Phase III Clinical Study Comparing the Efficacy and Safety of F182112 Versus Standard of Care in Patients With Relapsed or Refractory Multiple Myeloma |
| NCT01178281 | PHASE3 | COMPLETED | Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence |
| NCT01311687 | PHASE3 | COMPLETED | A Phase 3, Multicenter, Randomized, Open-Label Study to Compare the Efficacy and Safety of Pomalidomide in Combination With Low-Dose Dexamethasone Versus High-Dose Dexamethasone in Subjects With Refractory Multiple Myeloma or Relapsed and Refractory Multiple Myeloma and Companion Study |
| NCT01324947 | PHASE3 | COMPLETED | Study to Evaluate the Safety and Efficacy of Pomalidomide Monotherapy in Subjects With Refractory or Relapsed Refractory Multiple Myeloma |
| NCT01712789 | PHASE3 | COMPLETED | Evaluation of Safety of Pomalidomide in Combination With Dexamethasone (Low Dose) in Patients With Refractory or Relapsed and Refractory Multiple Myeloma |
| NCT01734928 | PHASE3 | COMPLETED | Safety and Efficacy of Pomalidomide, Bortezomib and Low-dose Dexamethasone in Subjects With Relapsed or Refractory Multiple Myeloma |
| NCT02576977 | PHASE3 | TERMINATED | Study of Pomalidomide and Low Dose Dexamethasone With or Without Pembrolizumab (MK-3475) in Refractory or Relapsed and Refractory Multiple Myeloma (rrMM) (MK-3475-183/KEYNOTE-183) |
| NCT02726581 | PHASE3 | COMPLETED | An Investigational Immuno-therapy Study of Nivolumab, Pomalidomide and Dexamethasone Combinations in Patients With Multiple Myeloma |
| NCT02916420 | PHASE3 | UNKNOWN | Efficacy and Safety of Pomalidomide in Combination With Low-dose Dexamethasone in Chinese Patients With Relapsed and Refractory Multiple Myeloma |
| NCT02990338 | PHASE3 | COMPLETED | Multinational Clinical Study Comparing Isatuximab, Pomalidomide, and Dexamethasone to Pomalidomide and Dexamethasone in Refractory or Relapsed and Refractory Multiple Myeloma Patients |
| NCT03151811 | PHASE3 | TERMINATED | A Study of Melphalan Flufenamide (Melflufen)-Dex or Pomalidomide-dex for RRMM Patients Refractory to Lenalidomide |
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.
18 molecules share ≥1 primary target. Top 18 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| CRIZOTINIB | ChEMBL + PubChem | Phase 4 (approved) | CRBN |
| ASCIMINIB | ChEMBL | Phase 4 (approved) | CRBN |
| BRIGATINIB | ChEMBL | Phase 4 (approved) | CRBN |
| DASABUVIR | ChEMBL | Phase 4 (approved) | CRBN |
| LENALIDOMIDE | ChEMBL | Phase 4 (approved) | CRBN |
| PONATINIB | ChEMBL | Phase 4 (approved) | CRBN |
| THALIDOMIDE | ChEMBL | Phase 4 (approved) | CRBN |
| IBERDOMIDE | ChEMBL | Phase 3 | CRBN |
| MEZIGDOMIDE | ChEMBL | Phase 3 | CRBN |
| URACIL | ChEMBL | Phase 3 | CRBN |
| VEPDEGESTRANT | ChEMBL | Phase 3 | CRBN |
| ADAVOSERTIB | ChEMBL | Phase 2 | CRBN |
| AVADOMIDE | ChEMBL | Phase 2 | CRBN |
| E-7820 | ChEMBL | Phase 2 | CRBN |
| FORETINIB | ChEMBL | Phase 2 | CRBN |
| INDISULAM | ChEMBL | Phase 2 | CRBN |
| Fulvestrant | PubChem | Approved | CRBN |
| Sotorasib | PubChem | Approved | CRBN |
Related Atlas pages
- Genes: CRBN
- Diseases: immune system disorder, plasma cell myeloma, primary myelofibrosis
- Drugs: Crizotinib, Asciminib, Brigatinib, Dasabuvir, Lenalidomide, Ponatinib, Thalidomide, Iberdomide, Mezigdomide, Uracil, Vepdegestrant, Fulvestrant, Sotorasib