Pomalidomide

drug
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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

FieldValue
ChEMBL IDCHEMBL43452
NamePomalidomide
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID134780
ChEBICHEBI:72690
ATCL04AX06
Molecular formulaC13H11N3O4
Molecular weight273.24
InChIKeyUVSMNLNDYGZFPF-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).

GeneTargetActionpAffinityCancer dependencyUniProt
CRBNcereblonBinding5.920.2%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):

TargetpChemblTypeValueUnitActivity ID
DDB18.05EC509nMCHEMBL_ACT_24860457
IKZF37.7EC5020nMCHEMBL_ACT_25673170
CRBN7.66EC5022nMCHEMBL_ACT_18565668
IKZF17.62EC5024nMCHEMBL_ACT_18565671
TNF7.48IC5033nMCHEMBL_ACT_26678795
IL1B7.33IC5047nMCHEMBL_ACT_26678780
IKZF17.3AC5050nMCHEMBL_ACT_25733423
IKZF17.3AC5050nMCHEMBL_ACT_25733506
DDB17.2IC5063.1nMCHEMBL_ACT_28439897
CRBN7.08IC5083nMCHEMBL_ACT_25536968
CRBN7.08Kd83nMCHEMBL_ACT_29075683
CRBN6.92IC50120nMCHEMBL_ACT_29075709
CRBN6.8Kd157nMCHEMBL_ACT_25848529
DDB16.4IC50400nMCHEMBL_ACT_25673122
CRBN6.4IC50400nMCHEMBL_ACT_29075649
CRBN6.13IC50739nMCHEMBL_ACT_25083008
CRBN5.92IC501200nMCHEMBL_ACT_18565676
DDB15.82IC501510nMCHEMBL_ACT_29056726
CRBN5.69IC502060nMCHEMBL_ACT_29103056
Q015585.54IC502900nMCHEMBL_ACT_26678777
CRBN5.48Ki3300nMCHEMBL_ACT_25665465
CRBN5Ki10000nMCHEMBL_ACT_22446525

Target pathways

Aggregated over 1 target gene(s): CRBN.

Top Reactome pathways

1 total, by targets touching each:

PathwayTargetsGenes
Potential therapeutics for SARS1CRBN

Dominant GO biological processes

GO termTargets
protein ubiquitination1
positive regulation of Wnt signaling pathway1
negative regulation of protein-containing complex assembly1
positive regulation of protein-containing complex assembly1
negative regulation of monoatomic ion transmembrane transport1
locomotory exploration behavior1
proteasome-mediated ubiquitin-dependent protein catabolic process1
limb development1

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).

IndicationTrial phaseMONDOEFO
immune system disorder4MONDO:0005046EFO:0000540
plasma cell myeloma4MONDO:0009693EFO:0001378
primary myelofibrosis3MONDO:0009692EFO:0002430
systemic sclerosis2MONDO:0005100EFO:0000717
Kaposi’s sarcoma2MONDO:0005055EFO:0000558
diffuse large B-cell lymphoma2MONDO:0018905EFO:0000403
acquired polycythemia vera2MONDO:0009891EFO:0002429
medulloblastoma2MONDO:0007959EFO:0002939
plasma cell leukemia2MONDO:0018689EFO:0006475
plasmacytoma2MONDO:0005615EFO:0006738
soft tissue sarcoma2MONDO:0018078EFO:1001968
interstitial lung disease2MONDO:0015925EFO:0004244
pulmonary fibrosis2MONDO:0002771EFO:0009448
graft versus host disease2MONDO:0013730MONDO:0013730
smoldering plasma cell myeloma2MONDO:0005235EFO:0003073
hereditary hemorrhagic telangiectasia2MONDO:0019180MONDO:0019180
hereditary amyloidosis2MONDO:0018634MONDO:0019438
plasma cell neoplasm2MONDO:0004959EFO:0000200
central nervous system neoplasm2MONDO:0006130EFO:1000158
amyloidosis2MONDO:0019065EFO:1001875
neoplasm1MONDO:0005070EFO:0000616
small cell lung carcinoma1MONDO:0008433EFO:0000702
exocrine pancreatic carcinoma1MONDO:0005192EFO:0002618
Hodgkins lymphoma1MONDO:0004952EFO:0000183
non-Hodgkin lymphoma1MONDO:0018908EFO:0005952
Waldenstrom macroglobulinemia1MONDO:0100280EFO:0009441
Castleman disease1MONDO:0015564MONDO:0015564
Burkitt lymphoma1MONDO:0007243EFO:0000309
sickle cell disease1MONDO:0011382MONDO:0011382
lymphoma1MONDO:0005062EFO: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

PhaseTrials
PHASE292
PHASE155
PHASE1/PHASE248
PHASE344
Not specified14
PHASE41
EARLY_PHASE11

Top trials by phase / activity

NCTPhaseStatusTitle
NCT05183139PHASE4WITHDRAWNA Multicenter In-class Transition Study of Ixazomib Combined With Pomalidomide and Dexamethasone or With Lenalidomide and Dexamethasone in Adults With Relapsed/Refractory Multiple Myeloma
NCT03539744PHASE3ACTIVE_NOT_RECRUITINGA 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.
NCT03651128PHASE3ACTIVE_NOT_RECRUITINGEfficacy and Safety Study of bb2121 Versus Standard Regimens in Subjects With Relapsed and Refractory Multiple Myeloma (RRMM)
NCT04181827PHASE3ACTIVE_NOT_RECRUITINGA 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
NCT04484623PHASE3ACTIVE_NOT_RECRUITINGBelantamab Mafodotin Plus Pomalidomide and Dexamethasone (Pd) Versus Bortezomib Plus Pd in Relapsed/Refractory Multiple Myeloma
NCT05020236PHASE3RECRUITINGA Study to Learn About the Study Medicine Elranatamab Alone and With Daratumumab in People With Multiple Myeloma Who Have Received Other Treatments
NCT05028348PHASE3ACTIVE_NOT_RECRUITINGA Study of Combination of Selinexor, Pomalidomide, and Dexamethasone (SPd) Versus Elotuzumab, Pomalidomide, and Dexamethasone (EloPd) in Subject With Previously Treated Multiple Myeloma
NCT05083169PHASE3ACTIVE_NOT_RECRUITINGA 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
NCT05405166PHASE3ACTIVE_NOT_RECRUITINGSC Versus IV Isatuximab in Combination With Pomalidomide and Dexamethasone in RRMM
NCT05438043PHASE3RECRUITINGA Study of Daratumumab
NCT05455320PHASE3ACTIVE_NOT_RECRUITINGA 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
NCT05519085PHASE3RECRUITINGA Study to Evaluate Mezigdomide, Bortezomib and Dexamethasone (MEZIVd) Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) in Participants With Relapsed or Refractory Multiple Myeloma (RRMM)
NCT05572515PHASE3ACTIVE_NOT_RECRUITINGA Study Comparing Teclistamab Monotherapy Versus Pomalidomide, Bortezomib, Dexamethasone (PVd) or Carfilzomib, Dexamethasone (Kd) in Participants With Relapsed or Refractory Multiple Myeloma
NCT05730036PHASE3RECRUITINGA Trial to Learn How Well Linvoseltamab Works Compared to the Combination of Elotuzumab, Pomalidomide and Dexamethasone for Adult Participants With Relapsed/Refractory Multiple Myeloma
NCT06152575PHASE3RECRUITINGMagnetisMM-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)
NCT06158841PHASE3RECRUITINGStudy Assessing Activity of Intravenous (IV) Etentamig Monotherapy Versus Standard Available Therapies in Adult Participants With Relapsed or Refractory Multiple Myeloma
NCT06208150PHASE3RECRUITINGA 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
NCT06413498PHASE3RECRUITINGA Study Comparing Anitocabtagene Autoleucel to Standard of Care Therapy in Participants With Relapsed/ Refractory Multiple Myeloma
NCT06464991PHASE3RECRUITINGA Phase III Study of Eque-cel in Subjects With Len-refractory RRMM (FUMANBA-03)
NCT06508983PHASE3RECRUITINGA Clinical Study Comparing SG301 Plus Pomalidomide and Dexamethasone to Placebo Plus Pomalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma Patients
NCT06615479PHASE3RECRUITINGA 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)
NCT06742138PHASE3NOT_YET_RECRUITINGTo Evaluate the Efficacy and Safety of QL2109 and DARZALEX FASPRO® in Multiple Myeloma
NCT06956170PHASE3ACTIVE_NOT_RECRUITINGAll Japanese Population: Belantamab Mafodotin Plus Pomalidomide and Dexamethasone (Pd) Versus Bortezomib Plus Pd in Relapsed/Refractory Multiple Myeloma
NCT07138209PHASE3RECRUITINGA Study Comparing QLS32015 Monotherapy Versus Pomalidomide, Dexamethasone (Pd) or Selinexor, Dexamethasone (Sd) in Participants With Relapsed or Refractory Multiple Myeloma
NCT07222761PHASE3RECRUITINGA 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)
NCT07391657PHASE3RECRUITINGA 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)
NCT07452198PHASE3RECRUITINGA Study of GR1803 Injection Versus Daratumumab, Pomalidomide, and Dexamethasone (DPd) in Participants With Relapsed or Refractory Multiple Myeloma
NCT07555938PHASE3RECRUITINGCevostamab in Combination With Pomalidomide and Dexamethasone Versus Standard of Care in Participants With Previously Treated Multiple Myeloma
NCT07569757PHASE3NOT_YET_RECRUITINGClinical Study of TQB2934 Injection in Relapsed/Refractory Multiple Myeloma
NCT07579234PHASE3RECRUITINGA 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
NCT01178281PHASE3COMPLETEDStudy of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence
NCT01311687PHASE3COMPLETEDA 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
NCT01324947PHASE3COMPLETEDStudy to Evaluate the Safety and Efficacy of Pomalidomide Monotherapy in Subjects With Refractory or Relapsed Refractory Multiple Myeloma
NCT01712789PHASE3COMPLETEDEvaluation of Safety of Pomalidomide in Combination With Dexamethasone (Low Dose) in Patients With Refractory or Relapsed and Refractory Multiple Myeloma
NCT01734928PHASE3COMPLETEDSafety and Efficacy of Pomalidomide, Bortezomib and Low-dose Dexamethasone in Subjects With Relapsed or Refractory Multiple Myeloma
NCT02576977PHASE3TERMINATEDStudy 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)
NCT02726581PHASE3COMPLETEDAn Investigational Immuno-therapy Study of Nivolumab, Pomalidomide and Dexamethasone Combinations in Patients With Multiple Myeloma
NCT02916420PHASE3UNKNOWNEfficacy and Safety of Pomalidomide in Combination With Low-dose Dexamethasone in Chinese Patients With Relapsed and Refractory Multiple Myeloma
NCT02990338PHASE3COMPLETEDMultinational Clinical Study Comparing Isatuximab, Pomalidomide, and Dexamethasone to Pomalidomide and Dexamethasone in Refractory or Relapsed and Refractory Multiple Myeloma Patients
NCT03151811PHASE3TERMINATEDA 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.

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:

MoleculeSourceStatusShared targets
CRIZOTINIBChEMBL + PubChemPhase 4 (approved)CRBN
ASCIMINIBChEMBLPhase 4 (approved)CRBN
BRIGATINIBChEMBLPhase 4 (approved)CRBN
DASABUVIRChEMBLPhase 4 (approved)CRBN
LENALIDOMIDEChEMBLPhase 4 (approved)CRBN
PONATINIBChEMBLPhase 4 (approved)CRBN
THALIDOMIDEChEMBLPhase 4 (approved)CRBN
IBERDOMIDEChEMBLPhase 3CRBN
MEZIGDOMIDEChEMBLPhase 3CRBN
URACILChEMBLPhase 3CRBN
VEPDEGESTRANTChEMBLPhase 3CRBN
ADAVOSERTIBChEMBLPhase 2CRBN
AVADOMIDEChEMBLPhase 2CRBN
E-7820ChEMBLPhase 2CRBN
FORETINIBChEMBLPhase 2CRBN
INDISULAMChEMBLPhase 2CRBN
FulvestrantPubChemApprovedCRBN
SotorasibPubChemApprovedCRBN