Gemtuzumab Ozogamicin
drugOn this page
Also known as CDP-771CMA-676CMC-676Gemtuzumab ozogamicin recombinantGemtuzumab ozogamicinaGemtuzumab ozogamicineL01XC05MylotargWAY-CMA-676GEMTUZUMAB-OZOGAMICINE
Summary
Gemtuzumab Ozogamicin (CHEMBL1201506) is an approved antibody drug conjugate (ATC L01FX02) targeting CD33; indicated across 9 conditions including acute myeloid leukemia and neoplasm; with CIViC clinical evidence for 1 variant-indication association (e.g. PML::RARA Fusion in acute promyelocytic leukemia).
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Antibody drug conjugate
- ATC class: L01FX02
- Targets: 1 (CD33)
- Indications: 9 conditions
- Clinical trials: 85
- Precision-oncology evidence (CIViC): 1 variant–indication association
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL1201506 |
| Name | Gemtuzumab Ozogamicin |
| Type | Antibody drug conjugate |
| Max phase | 4 |
| ATC | L01FX02 |
Also known as: CDP-771, CMA-676, CMC-676, Gemtuzumab ozogamicin, Gemtuzumab ozogamicin recombinant, Gemtuzumab ozogamicina, Gemtuzumab ozogamicine, L01XC05, Mylotarg, WAY-CMA-676, GEMTUZUMAB OZOGAMICIN, GEMTUZUMAB-OZOGAMICINE
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 |
|---|---|---|---|---|---|
| CD33 | CD33 | Binding | 0% | P20138 |
Bioactivity
No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).
Target pathways
Aggregated over 1 target gene(s): CD33.
Top Reactome pathways
6 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Adaptive Immune System | 1 | CD33 |
| Innate Immune System | 1 | CD33 |
| Immune System | 1 | CD33 |
| Immunoregulatory interactions between a Lymphoid and a non-Lymphoid cell | 1 | CD33 |
| Neutrophil degranulation | 1 | CD33 |
| Dengue Virus Attachment and Entry | 1 | CD33 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| immune response-inhibiting signal transduction | 1 |
| cell adhesion | 1 |
| signal transduction | 1 |
| cell-cell signaling | 1 |
| negative regulation of cell population proliferation | 1 |
| negative regulation of interleukin-1 beta production | 1 |
| negative regulation of interleukin-8 production | 1 |
| negative regulation of tumor necrosis factor production | 1 |
| Fc-gamma receptor signaling pathway | 1 |
| positive regulation of protein secretion | 1 |
| cell-cell adhesion | 1 |
| negative regulation of monocyte activation | 1 |
Indications & clinical
Indications
9 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 |
|---|---|---|---|
| acute myeloid leukemia | 4 | MONDO:0018874 | EFO:0000222 |
| neoplasm | 4 | MONDO:0005070 | EFO:0000616 |
| myeloid leukemia | 4 | MONDO:0004643 | MONDO:0004643 |
| leukemia | 3 | MONDO:0005059 | EFO:0000565 |
| myelodysplastic syndrome | 3 | MONDO:0018881 | EFO:0000198 |
| acute lymphoblastic leukemia | 2 | MONDO:0004967 | EFO:0000220 |
| acute promyelocytic leukemia | 2 | MONDO:0012883 | EFO:0000224 |
| B-cell chronic lymphocytic leukemia | 1 | MONDO:0004948 | EFO:0000095 |
| juvenile myelomonocytic leukemia | 1 | MONDO:0011908 | EFO:1000309 |
Clinical trials
Total trials: 85.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 41 |
| PHASE1 | 14 |
| PHASE3 | 11 |
| PHASE1/PHASE2 | 9 |
| PHASE2/PHASE3 | 4 |
| Not specified | 3 |
| PHASE4 | 2 |
| EARLY_PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT00304447 | PHASE4 | COMPLETED | Study Evaluating the Effect of Corticosteroids on Mylotarg® Infusion-Related Adverse Events in Patients With Leukemia |
| NCT03727750 | PHASE4 | COMPLETED | Evaluating QTc, PK, Safety of Gemtuzumab Ozogamicin (GO) in Patients With CD33+ R/R AML |
| NCT02724163 | PHASE3 | RECRUITING | International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia |
| NCT04168502 | PHASE3 | RECRUITING | Gemtuzumab Chemotherapy MRD Levels; Adult Untreated, de Novo, Fav Interm Risk AML |
| NCT04293562 | PHASE3 | RECRUITING | A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations |
| NCT05183035 | PHASE3 | RECRUITING | Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML) |
| NCT00038805 | PHASE2/PHASE3 | TERMINATED | Nonmyeloablative Preparative Regimen Using Mylotarg for Patients With High Risk Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myeloid Leukemia (CML) and Myelodysplastic Syndrome (MDS) |
| NCT00052299 | PHASE3 | COMPLETED | Chemotherapy With or Without Gemtuzumab Ozogamicin in Treating Older Patients With Acute Myeloid Leukemia |
| NCT00085709 | PHASE3 | COMPLETED | S0106 Cytarabine and Daunorubicin w/ or w/o Gemtuzumab Followed By HD Cytarabine and Either Gemtuzumab or Nothing in de Novo AML |
| NCT00091234 | PHASE2/PHASE3 | UNKNOWN | Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia |
| NCT00121303 | PHASE3 | COMPLETED | Cytarabine and Daunorubicin With or Without Gemtuzumab Ozogamicin in Treating Older Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes |
| NCT00372593 | PHASE3 | COMPLETED | Combination Chemotherapy With or Without Gemtuzumab in Treating Young Patients With Newly Diagnosed Acute Myeloid Leukemia |
| NCT00454480 | PHASE2/PHASE3 | COMPLETED | Combination Chemotherapy With or Without Gemtuzumab Ozogamicin or Tipifarnib in Treating Patients With Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndromes |
| NCT00476541 | PHASE3 | COMPLETED | NOPHO-AML 2004 Study for Children With Acute Myeloid Leukemia |
| NCT00909168 | PHASE2/PHASE3 | COMPLETED | Induction, Consolidation and Intensification Therapy for Patients Younger Than 66 Years With Previously Untreated CD33 Positive Acute Myeloid Leukemia (AML) |
| NCT00962767 | PHASE3 | COMPLETED | Comparison of Two Treatments in Intermediate and High-risk Acute Promyelocytic Leukemia (APL) Patients to Assess Efficacy in 1st Hematological Complete Remission and Molecular Remission |
| NCT04093505 | PHASE3 | TERMINATED | Gemtuzumab Ozogamicin in Induction and Glasdegib in Postremission Therapy in Patients With AML (Acute Myeloid Leukemia) |
| NCT00658814 | PHASE2 | ACTIVE_NOT_RECRUITING | Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia |
| NCT00801489 | PHASE2 | RECRUITING | Fludarabine Phosphate, Cytarabine, Filgrastim-sndz, Gemtuzumab Ozogamicin, and Idarubicin Hydrochloride in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome |
| NCT01409161 | PHASE2 | RECRUITING | Tretinoin and Arsenic Trioxide With or Without Gemtuzumab Ozogamicin in Treating Patients With Previously Untreated Acute Promyelocytic Leukemia |
| NCT03589729 | PHASE2 | RECRUITING | Dexrazoxane Hydrochloride in Preventing Heart-Related Side Effects of Chemotherapy in Participants With Blood Cancers |
| NCT03672539 | PHASE2 | ACTIVE_NOT_RECRUITING | Liposome-encapsulated Daunorubicin-Cytarabine and Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome |
| NCT03737955 | PHASE2 | RECRUITING | Fractionated Gemtuzumab Ozogamicin in Treating Measurable Residual Disease in Patients With Acute Myeloid Leukemia |
| NCT04793919 | PHASE2 | RECRUITING | Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia |
| NCT05564390 | PHASE2 | RECRUITING | MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) |
| NCT05599360 | PHASE2 | ACTIVE_NOT_RECRUITING | Vyxeos for Induction of Low- or Intermediate-risk. |
| NCT06917911 | PHASE2 | RECRUITING | Testing the Addition of Venetoclax or Gemtuzumab Ozogamicin (GO) to Usual Treatment Regimen (Cytarabine and Daunorubicin, 7+3) for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) to Improve Response (A MYELOMATCH Treatment Trial) |
| NCT00003131 | PHASE2 | COMPLETED | CMA-676 in Treating Patients With Acute Myeloid Leukemia in First Relapse |
| NCT00003673 | PHASE2 | COMPLETED | CMA-676 in Treating Older Patients With Acute Myeloid Leukemia in First Relapse |
| NCT00005962 | PHASE2 | COMPLETED | Comparison of Three Treatment Regimens in Treating Patients With Relapsed or Refractory Acute Myelogenous Leukemia |
| NCT00006122 | PHASE2 | COMPLETED | Gemtuzumab Ozogamicin With or Without Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia |
| NCT00006265 | PHASE2 | COMPLETED | Gemtuzumab Ozogamicin and High-Dose Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia |
| NCT00008151 | PHASE2 | COMPLETED | Gemtuzumab Ozogamicin, Fludarabine, and Total-body Irradiation Followed by Peripheral Stem Cell or Bone Marrow Transplantation in Treating Patients With Advanced Acute Myeloid Leukemia or Myelodysplastic Syndrome |
| NCT00017589 | PHASE2 | COMPLETED | Oblimersen and Gemtuzumab Ozogamicin in Treating Older Patients With Relapsed Acute Myeloid Leukemia |
| NCT00022321 | PHASE2 | UNKNOWN | Gemtuzumab in Treating Patients With Myelodysplastic Syndrome |
| NCT00037583 | PHASE2 | COMPLETED | Study Evaluating the Safety and Efficacy of Gemtuzumab Ozogamicin in Patients With Acute Myeloid Leukemia |
| NCT00037596 | PHASE2 | COMPLETED | Study Evaluating Gemtuzumab Ozogamicin in Acute Myeloid Leukemia |
| NCT00038831 | PHASE1/PHASE2 | COMPLETED | Allo Transplantation With Mylotarg, Fludarabine and Melphalan for AML, CML and MDS |
| NCT00049179 | PHASE2 | COMPLETED | S0117 Gemtuzumab Ozogamicin Plus Cytarabine in Treating Patients With Relapsed Acute Myeloid Leukemia |
| NCT00070174 | PHASE2 | COMPLETED | Gemtuzumab Ozogamicin in Treating Young Patients With Newly Diagnosed Acute Myeloid Leukemia Undergoing Remission Induction and Intensification Therapy |
Clinical evidence (CIViC)
Variant × indication × effect (1 predictive associations from 1 curated evidence items):
| Variant | Indication | Effect | Therapy | Level | CIViC |
|---|---|---|---|---|---|
| PML::RARA Fusion | Acute Promyelocytic Leukemia | Sensitivity/Response | Gemtuzumab Ozogamicin | CIViC B | EID8300 |
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).