Everolimus
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Also known as AfinitorAfinitor disperzCerticanRAD 666RAD-001RAD-666RAD001Sdz radSDZ-RADVotubiaZortressSID144206063
Summary
Everolimus (CHEMBL1908360) is an approved small-molecule antineoplastic agent (ATC L01EG02) targeting MTOR; indicated across 147 conditions including breast carcinoma and renal cell carcinoma; with CIViC clinical evidence for 42 variant-indication associations (e.g. TSC1 Loss-of-function in tuberous sclerosis).
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: L01EG02 (+1 more)
- Targets: 1 (MTOR)
- Indications: 147 conditions
- Clinical trials: 712
- Precision-oncology evidence (CIViC): 42 variant–indication associations
- Chemistry: 958.2 Da · C53H83NO14
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL1908360 |
| Name | Everolimus |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 6442177 |
| ChEBI | CHEBI:68478 |
| ATC | L01EG02, L04AH02 |
| Molecular formula | C53H83NO14 |
| Molecular weight | 958.2 |
| InChIKey | HKVAMNSJSFKALM-GKUWKFKPSA-N |
SMILES: C[C@@H]1CC[C@H]2C[C@@H](/C(=C/C=C/C=C/[C@H](C[C@H](C(=O)[C@@H]([C@@H](/C(=C/[C@H](C(=O)C[C@H](OC(=O)[C@@H]3CCCCN3C(=O)C(=O)[C@@]1(O2)O)[C@H](C)C[C@@H]4CC[C@H]([C@@H](C4)OC)OCCO)C)/C)O)OC)C)C)/C)OC
IUPAC name: (1R,9S,12S,15R,16E,18R,19R,21R,23S,24E,26E,28E,30S,32S,35R)-1,18-dihydroxy-12-[(2R)-1-[(1S,3R,4R)-4-(2-hydroxyethoxy)-3-methoxycyclohexyl]propan-2-yl]-19,30-dimethoxy-15,17,21,23,29,35-hexamethyl-11,36-dioxa-4-azatricyclo[30.3.1.04,9]hexatriaconta-16,24,26,28-tetraene-2,3,10,14,20-pentone
ChEBI definition: A macrocyclic lactone that is rapamycin in which the hydroxy group attached to the cyclohexyl moiety has been converted into the corresponding 2-hydroxyethyl ether. It is an immunosuppressant and antineoplastic agent.
Pharmacological roles (ChEBI): antineoplastic agent, immunosuppressive agent, mTOR inhibitor, anticoronaviral agent, geroprotector.
Also known as: Afinitor, Afinitor disperz, Certican, Everolimus, RAD 666, RAD-001, RAD-666, RAD001, Sdz rad, SDZ-RAD, Votubia, Zortress
Patent coverage: 18,736 distinct patent families (73,430 SureChEMBL compound mentions), from 1 matched compound structure(s). 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 |
|---|---|---|---|---|---|
| MTOR | mechanistic target of rapamycin kinase | Inhibition | 8.7 | 98.3% (common-essential) | P42345 |
Broader ChEMBL bioactivity targets: 7 (assay-derived). Sample: ATP-binding cassette sub-family C member 4, Voltage-gated potassium channel, IKs; KCNQ1(Kv7.1)/KCNE1(MinK), Serine/threonine-protein kinase mTOR, mTORC1, ATP-binding cassette sub-family C member 2, ATP-binding cassette sub-family C member 3, Bile salt export pump.
Bioactivity
ChEMBL activities: 11 potent at pChembl ≥ 5 of 15 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| MTOR | 10.3 | IC50 | 0.05 | nM | CHEMBL_ACT_24962479 |
| MTOR | 10.15 | IC50 | 0.07 | nM | CHEMBL_ACT_25441981 |
| MTOR | 8.8 | IC50 | 1.6 | nM | CHEMBL_ACT_24788805 |
| MTOR | 8.7 | Kd | 2 | nM | CHEMBL_ACT_18813394 |
| MTOR | 8.3 | IC50 | 5 | nM | CHEMBL_ACT_20681616 |
| MTOR | 8.3 | IC50 | 5 | nM | CHEMBL_ACT_24984326 |
| MTOR | 8.3 | IC50 | 5 | nM | CHEMBL_ACT_25044904 |
| MTOR | 8.3 | IC50 | 5 | nM | CHEMBL_ACT_29205858 |
| MTOR | 6.82 | IC50 | 150 | nM | CHEMBL_ACT_25872954 |
| MTOR | 6.28 | IC50 | 530 | nM | CHEMBL_ACT_25683586 |
| ABCB11 | 5.7 | IC50 | 2000 | nM | CHEMBL_ACT_18129022 |
Target pathways
Aggregated over 1 target gene(s): MTOR.
Top Reactome pathways
41 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| PIP3 activates AKT signaling | 1 | MTOR |
| Adaptive Immune System | 1 | MTOR |
| Signal Transduction | 1 | MTOR |
| Macroautophagy | 1 | MTOR |
| Disease | 1 | MTOR |
| MTOR signalling | 1 | MTOR |
| mTORC1-mediated signalling | 1 | MTOR |
| Immune System | 1 | MTOR |
| Signaling by VEGF | 1 | MTOR |
| Generic Transcription Pathway | 1 | MTOR |
| PI3K/AKT Signaling in Cancer | 1 | MTOR |
| Cellular responses to stress | 1 | MTOR |
| Cellular response to heat stress | 1 | MTOR |
| HSF1-dependent transactivation | 1 | MTOR |
| Transcriptional Regulation by TP53 | 1 | MTOR |
| Energy dependent regulation of mTOR by LKB1-AMPK | 1 | MTOR |
| Regulation of T cell activation by CD28 family | 1 | MTOR |
| Co-stimulation by CD28 | 1 | MTOR |
| CD28 dependent PI3K/Akt signaling | 1 | MTOR |
| VEGFA-VEGFR2 Pathway | 1 | MTOR |
| VEGFR2 mediated vascular permeability | 1 | MTOR |
| TP53 Regulates Metabolic Genes | 1 | MTOR |
| Regulation of TP53 Activity | 1 | MTOR |
| Diseases of signal transduction by growth factor receptors and second messengers | 1 | MTOR |
| Constitutive Signaling by AKT1 E17K in Cancer | 1 | MTOR |
| Regulation of TP53 Degradation | 1 | MTOR |
| Regulation of TP53 Expression and Degradation | 1 | MTOR |
| PTEN Regulation | 1 | MTOR |
| RNA Polymerase II Transcription | 1 | MTOR |
| Gene expression (Transcription) | 1 | MTOR |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| regulation of cell growth | 1 |
| T-helper 1 cell lineage commitment | 1 |
| heart morphogenesis | 1 |
| heart valve morphogenesis | 1 |
| energy reserve metabolic process | 1 |
| ‘de novo’ pyrimidine nucleobase biosynthetic process | 1 |
| inflammatory response | 1 |
| DNA damage response | 1 |
| cytoskeleton organization | 1 |
| germ cell development | 1 |
| regulation of cell size | 1 |
| cellular response to starvation | 1 |
| response to heat | 1 |
| response to virus | 1 |
| post-embryonic development | 1 |
Indications & clinical
Indications
147 indications (21 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 |
|---|---|---|---|
| breast carcinoma | 4 | MONDO:0004989 | EFO:0000305 |
| renal cell carcinoma | 4 | MONDO:0005086 | EFO:0000681 |
| neoplasm | 4 | MONDO:0005070 | EFO:0000616 |
| exocrine pancreatic carcinoma | 4 | MONDO:0005192 | EFO:0002618 |
| clear cell renal carcinoma | 4 | MONDO:0005005 | EFO:0000349 |
| lung neuroendocrine neoplasm | 4 | MONDO:0005454 | EFO:0005220 |
| breast neoplasm | 4 | MONDO:0021100 | EFO:0003869 |
| astrocytoma (excluding glioblastoma) | 4 | MONDO:0019781 | EFO:0000272 |
| anaplastic astrocytoma | 4 | MONDO:0016684 | EFO:0002499 |
| pancreatic neoplasm | 4 | MONDO:0021040 | EFO:0003860 |
| pancreatic neuroendocrine tumor | 4 | MONDO:0019954 | EFO:1000045 |
| neuroendocrine neoplasm | 4 | MONDO:0019496 | EFO:1001901 |
| tuberous sclerosis | 4 | MONDO:0001734 | HP:0009720 |
| angiomyolipoma | 4 | MONDO:0002603 | MONDO:0002603 |
| hamartoma | 4 | MONDO:0006499 | EFO:1000634 |
| renal cell adenocarcinoma | 4 | MONDO:0005549 | EFO:0005708 |
| carcinoma | 3 | MONDO:0004993 | EFO:0000313 |
| graft versus host disease | 3 | MONDO:0013730 | EFO:0004599 |
| diffuse large B-cell lymphoma | 3 | MONDO:0018905 | EFO:0000403 |
| gastric neoplasm | 3 | MONDO:0021085 | EFO:0003897 |
| chronic kidney disease | 3 | MONDO:0005300 | EFO:0003884 |
| cytomegalovirus infection | 3 | MONDO:0005132 | EFO:0001062 |
| coronary restenosis | 3 | MONDO:0005355 | EFO:0004224 |
| carcinoid syndrome | 3 | MONDO:0100347 | EFO:1000852 |
| autosomal dominant polycystic kidney disease | 3 | MONDO:0004691 | EFO:1001496 |
| kidney failure | 3 | MONDO:0001106 | EFO:1002048 |
| kidney cancer | 3 | MONDO:0002367 | MONDO:0002367 |
| skin neoplasm | 3 | MONDO:0002531 | MONDO:0002898 |
| carcinoid tumor | 3 | MONDO:0005369 | MONDO:0024503 |
| coronary artery disorder | 3 | MONDO:0005010 | EFO:0001645 |
| kidney neoplasm | 3 | MONDO:0021163 | EFO:0003865 |
| adenoid cystic carcinoma | 2 | MONDO:0004971 | EFO:0000231 |
| colorectal adenocarcinoma | 2 | MONDO:0005008 | EFO:0000365 |
| mesothelioma | 2 | MONDO:0005065 | EFO:0000588 |
| non-small cell lung carcinoma | 2 | MONDO:0005233 | EFO:0003060 |
| sarcoma | 2 | MONDO:0005089 | EFO:0000691 |
| small cell lung carcinoma | 2 | MONDO:0008433 | EFO:0000702 |
| cholangiocarcinoma | 2 | MONDO:0019087 | EFO:0005221 |
| glioblastoma | 2 | MONDO:0018177 | EFO:0000519 |
| hepatocellular carcinoma | 2 | MONDO:0007256 | EFO:0000182 |
| kidney disorder | 2 | MONDO:0005240 | EFO:0003086 |
| lymphoma | 2 | MONDO:0005062 | EFO:0000574 |
| malignant pleural mesothelioma | 2 | MONDO:0005112 | EFO:0000770 |
| primary cutaneous T-cell non-Hodgkin lymphoma | 2 | MONDO:0000607 | EFO:0002913 |
| germ cell tumor | 2 | MONDO:0005040 | EFO:0000514 |
| lung carcinoma | 2 | MONDO:0005138 | EFO:0001071 |
| central nervous system cancer | 2 | MONDO:0002714 | EFO:0000326 |
| chondrosarcoma | 2 | MONDO:0008977 | EFO:0000333 |
| Hodgkins lymphoma | 2 | MONDO:0004952 | EFO:0000183 |
| osteosarcoma | 2 | MONDO:0009807 | EFO:0000637 |
| hepatitis C virus infection | 2 | MONDO:0005231 | EFO:0003047 |
| acute myeloid leukemia | 2 | MONDO:0018874 | EFO:0000222 |
| brain neoplasm | 2 | MONDO:0021211 | EFO:0003833 |
| Kaposi’s sarcoma | 2 | MONDO:0005055 | EFO:0000558 |
| mantle cell lymphoma | 2 | MONDO:0018876 | EFO:1001469 |
| myelodysplastic syndrome | 2 | MONDO:0018881 | EFO:0000198 |
| age-related macular degeneration | 2 | MONDO:0005150 | EFO:0001365 |
| uveitis | 2 | MONDO:0020283 | EFO:1001231 |
| head and neck squamous cell carcinoma | 2 | MONDO:0010150 | EFO:0000181 |
| cutaneous melanoma | 2 | MONDO:0005012 | EFO:0000389 |
| epilepsy | 2 | MONDO:0005027 | EFO:0000474 |
| oligoastrocytoma | 2 | MONDO:0016702 | EFO:0000630 |
| oligodendroglioma | 2 | MONDO:0016695 | EFO:0000632 |
| plasma cell myeloma | 2 | MONDO:0009693 | EFO:0001378 |
| metastatic melanoma | 2 | MONDO:0005191 | EFO:0002617 |
| thymus neoplasm | 2 | MONDO:0005197 | EFO:0002626 |
| head and neck cancer | 2 | MONDO:0005627 | EFO:0006859 |
| pancreatic neuroendocrine neoplasm | 2 | MONDO:0005815 | EFO:0007331 |
| transitional cell carcinoma | 2 | MONDO:0006474 | EFO:1000601 |
| marginal zone lymphoma | 2 | MONDO:0017604 | EFO:1000630 |
| soft tissue sarcoma | 2 | MONDO:0018078 | EFO:1001968 |
| triple-negative breast carcinoma | 2 | MONDO:0005494 | EFO:0005537 |
| Waldenstrom macroglobulinemia | 2 | MONDO:0100280 | EFO:0009441 |
| malignant peripheral nerve sheath tumor | 2 | MONDO:0017827 | EFO:0000760 |
| polycystic kidney disease | 2 | MONDO:0020642 | EFO:0008620 |
| diffuse intrinsic pontine glioma | 2 | MONDO:0006033 | EFO:1000026 |
| hyperaldosteronism | 2 | MONDO:0003009 | MONDO:0001422 |
| neuroendocrine carcinoma | 2 | MONDO:0002120 | MONDO:0002120 |
| uterine cancer | 2 | MONDO:0002715 | MONDO:0002715 |
| glioma | 2 | MONDO:0021042 | MONDO:0003268 |
| ependymoma | 2 | MONDO:0016698 | MONDO:0003478 |
| gallbladder neoplasm | 2 | MONDO:0021253 | MONDO:0005411 |
| ovarian cancer | 2 | MONDO:0008170 | MONDO:0008170 |
| lung neoplasm | 2 | MONDO:0021117 | MONDO:0008903 |
| endometrium neoplasm | 2 | MONDO:0021251 | MONDO:0011962 |
| colonic neoplasm | 2 | MONDO:0005401 | MONDO:0021063 |
| Peutz-Jeghers syndrome | 2 | MONDO:0008280 | MONDO:0008280 |
| lymphangioleiomyomatosis | 2 | MONDO:0011705 | MONDO:0011705 |
| meningioma | 2 | MONDO:0016642 | MONDO:0016642 |
| tuberculosis | 2 | MONDO:0018076 | MONDO:0018076 |
| neurofibromatosis type 1 | 2 | MONDO:0018975 | MONDO:0018975 |
| thyroid gland carcinoma | 2 | MONDO:0015075 | EFO:0002892 |
| bile duct carcinoma | 2 | MONDO:0005496 | EFO:0005540 |
| paraganglioma | 2 | MONDO:0000448 | EFO:1000453 |
| uveal melanoma | 2 | MONDO:0006486 | EFO:1000616 |
| colorectal neoplasm | 2 | MONDO:0005335 | MONDO:0005575 |
| Sturge-Weber syndrome | 2 | MONDO:0008501 | MONDO:0008501 |
| acute coronary syndrome | 1 | MONDO:0005542 | EFO:0005672 |
| carcinoma of esophagus | 1 | MONDO:0019086 | EFO:0002916 |
| acute lymphoblastic leukemia | 1 | MONDO:0004967 | EFO:0000220 |
| adenocarcinoma | 1 | MONDO:0004970 | EFO:0000228 |
| chronic myeloid leukemia | 1 | MONDO:0011996 | EFO:0000339 |
| leukemia | 1 | MONDO:0005059 | EFO:0000565 |
| synovial sarcoma | 1 | MONDO:0010434 | EFO:0001376 |
| nasopharyngeal neoplasm | 1 | MONDO:0005375 | EFO:0004252 |
| lymphoid leukemia | 1 | MONDO:0005402 | EFO:0004289 |
| non-Hodgkin lymphoma | 1 | MONDO:0018908 | EFO:0005952 |
| male breast carcinoma | 1 | MONDO:0005628 | EFO:0006861 |
| hematologic disorder | 1 | MONDO:0005570 | HP:0001871 |
| central nervous system neoplasm | 1 | MONDO:0006130 | EFO:1000158 |
| peritoneal neoplasm | 1 | MONDO:0006901 | MONDO:0002087 |
| fallopian tube neoplasm | 1 | MONDO:0021092 | MONDO:0002158 |
| progeroid syndrome | 1 | MONDO:0015333 | MONDO:0020732 |
| gastrointestinal stromal tumor | 1 | MONDO:0011719 | MONDO:0011719 |
| Cowden disease | 1 | MONDO:0016063 | MONDO:0017623 |
| plasma cell neoplasm | 1 | MONDO:0004959 | EFO:0000200 |
31 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 712.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 265 |
| PHASE1 | 129 |
| PHASE1/PHASE2 | 93 |
| PHASE3 | 91 |
| PHASE4 | 81 |
| Not specified | 32 |
| PHASE2/PHASE3 | 12 |
| EARLY_PHASE1 | 9 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT02081755 | PHASE4 | ENROLLING_BY_INVITATION | Safety and Efficacy of Everolimus Treatment in Liver Transplantation for Liver Cancer |
| NCT05252585 | PHASE4 | ACTIVE_NOT_RECRUITING | A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML) |
| NCT06584773 | PHASE4 | RECRUITING | Efficacy of a Quadruple Immunosuppressor Regimen With mTOR Inhibitors in Sensitized Kidney Transplant Patients |
| NCT06942156 | PHASE4 | NOT_YET_RECRUITING | Optimize Immunosuppressive Therapy Using Everolimus and Low-dose Calcineurin Inhibitors in Heart Transplant Patients in Korea |
| NCT07426484 | PHASE4 | NOT_YET_RECRUITING | Cosibelimab for CSCC in Patients With Kidney Transplant or Hematologic Malignancy |
| NCT00154297 | PHASE4 | COMPLETED | Efficacy and Safety of Early Versus Delayed Administration of Everolimus in de Novo Renal Transplant Patients |
| NCT00154310 | PHASE4 | COMPLETED | Efficacy and Safety of Everolimus With Enteric-Coated Mycophenolate Sodium (EC-MPS) in a Cyclosporine Microemulsion-free Regimen Compared to Standard Therapy in de Novo Renal Transplant Patients |
| NCT00170820 | PHASE4 | COMPLETED | Efficacy and Safety of the Switch From Sirolimus to Everolimus in Stable Maintenance Renal Transplant Patients Receiving a Calcineurin Inhibitor Free Regimen |
| NCT00170846 | PHASE4 | COMPLETED | ASCERTAIN: Assessment of Everolimus in Addition to Calcineurin Inhibitor Reduction in the Maintenance of Renal Transplant Recipients |
| NCT00170859 | PHASE4 | COMPLETED | Everolimus Versus Mycophenolate Mofetil in Combination With Reduced Dose Cyclosporine Microemulsion in Maintenance Heart Transplant Recipients |
| NCT00170885 | PHASE4 | COMPLETED | Everolimus in Combination With Cyclosporine Microemulsion in de Novo Renal Transplant Recipients |
| NCT00332839 | PHASE4 | TERMINATED | Comparison of CNI-based Regimen Versus CNI-free Regimen in Kidney Transplant Recipients. |
| NCT00369161 | PHASE4 | COMPLETED | A Twelve-month, Multicenter, Open-label, Randomized Study of the Safety, Tolerability and Efficacy of Everolimus With Basiliximab, Corticosteroids and Two Different Exposure Levels of Tacrolimus in de Novo Renal Transplant Recipients |
| NCT00371826 | PHASE4 | COMPLETED | SOCRATES: Steroid or Cyclosporine Removal After Transplantation Using Everolimus |
| NCT00377962 | PHASE4 | COMPLETED | Nordic Everolimus (Certican) Trial in Heart and Lung Transplantation |
| NCT00414440 | PHASE4 | COMPLETED | Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease in Patients With Autosomal Dominant Polycystic Kidney Disease |
| NCT00420537 | PHASE4 | TERMINATED | Shift to Everolimus (RAD) Kidney Sparing Study |
| NCT00443937 | PHASE4 | COMPLETED | Pharmacokinetics of Everolimus and Enteric-Coated Mycophenolatesodium Before and After Withdrawal of Cyclosporine in Renal Transplant Patients |
| NCT00505102 | PHASE4 | UNKNOWN | Safe Renal Function In Long Term Heart Transplanted Patients |
| NCT00596557 | PHASE4 | COMPLETED | Everolimus and Low Dose CNI Compared With MMF and Full CNI Dose in Heart Transplanted Patients: One Year Follow up |
| NCT00634920 | PHASE4 | COMPLETED | Evaluation of Early Conversion to Everolimus From Cyclosporine in de Novo Renal Transplant Recipients |
| NCT00695344 | PHASE4 | UNKNOWN | Study to Evaluate Effect of Everolimus in Progression of Graft Vascular Illness on Patients With Heart Transplant |
| NCT00716573 | PHASE4 | COMPLETED | Efficacy Study of Everolimus on Renal Function in Heart Transplant Recipients With Established Chronic Renal Failure |
| NCT00862979 | PHASE4 | COMPLETED | A Study Investigating the Renal Tolerability, Efficacy, and Safety of a CNI-free Versus a Standard Regimen in de Novo Heart Transplant (HTx) Recipients |
| NCT00888758 | PHASE4 | UNKNOWN | Comparison of Biolimus A9 and Everolimus Drug-Eluting Stents in Patients With ST Segment Elevation Myocardial Infarction |
| NCT00903188 | PHASE4 | UNKNOWN | Calcineurin Inhibitor (CNI) Versus Steroid Cessation in Renal Transplantation |
| NCT00956293 | PHASE4 | TERMINATED | Study to Evaluate the Efficacy, Safety and Tolerability of Everolimus in de Novo Renal Transplant Recipients Participating in the Eurotransplant Senior Program |
| NCT00965094 | PHASE4 | COMPLETED | Efficacy and Safety of Everolimus+EC-MPS After Early CNI Elimination vs EC-MPS +Tacrolimus in Renal Transplant Recipients |
| NCT01017029 | PHASE4 | COMPLETED | Everolimus in de Novo Heart Transplant Recipients |
| NCT01046045 | PHASE4 | COMPLETED | Everolimus Rescue Immunosuppression in the Treatment of Chronic Allograft Dysfunction in Renal Transplant Recipients |
| NCT01169701 | PHASE4 | COMPLETED | 24 Months Follow-up, Two Arm Study to Compare the Cardiovascular Profile in a Regimen With Everolimus + Mycophenolic Acid (MPA) Versus (vs.) a Regimen of CNI+MPA in Maintenance Renal Transplant Recipients |
| NCT01183247 | PHASE4 | COMPLETED | An Open, Single Centre, Randomised, Parallel Group Study to Investigate Three Different Immunosuppressive Regimens |
| NCT01206764 | PHASE4 | COMPLETED | A Trial of Everolimis in Patients With Advanced Renal Cell Carcinoma. |
| NCT01239472 | PHASE4 | COMPLETED | Cytokines Evaluation in Early Calcineurin Inhibitors Withdrawn on Renal Transplant |
| NCT01266148 | PHASE4 | COMPLETED | SCHEDULE - Scandinavian Heart Transplant Everolimus de Novo Study With Early Calcineurin Inhibitor (CNI) Avoidance |
| NCT01266837 | PHASE4 | COMPLETED | Open Label, Single Arm Trial to Characterize Patients With Metastatic RCC Treated With Everolimus After Failure of the First VEGF-targeted Therapy (MARC-2) |
| NCT01269684 | PHASE4 | WITHDRAWN | Immediate Conversion From Tacrolimus to Everolimus in Stable Maintenance Renal Transplant Recipients |
| NCT01276834 | PHASE4 | TERMINATED | Comparison of Immunosuppression on Progression of Arteriosclerosis in Renal Transplantation |
| NCT01312064 | PHASE4 | TERMINATED | De Novo Everolimus-based Therapy for Renal Transplantation Using Rituximab Induction |
| NCT01317615 | PHASE4 | COMPLETED | RAD001 With Paclitaxel and Carboplatin in First Line Treatment of Patients With Advanced Large Cell Lung Cancer With Neuroendocrine Differentiation |
Clinical evidence (CIViC)
Variant × indication × effect (42 predictive associations from 42 curated evidence items):
| Variant | Indication | Effect | Therapy | Level | CIViC |
|---|---|---|---|---|---|
| TSC1 Loss-of-function | Tuberous Sclerosis | Sensitivity/Response | Everolimus | CIViC A | EID7280 |
| TSC1 mutation OR TSC2 mutation | Tuberous Sclerosis | Sensitivity/Response | Everolimus | CIViC A | EID11269 |
| TSC2 Loss-of-function | Tuberous Sclerosis | Sensitivity/Response | Everolimus | CIViC A | EID7281 |
| FGFR1 Amplification | Breast Cancer | Sensitivity/Response | Endocrine Drug Therapy + Everolimus | CIViC B | EID12498 |
| KDM5C Mutation | Renal Cell Carcinoma | Sensitivity/Response | Sunitinib + Everolimus | CIViC B | EID5997 |
| NF1 Loss | Malignant Peripheral Nerve Sheath Tumor | Sensitivity/Response | Everolimus + Bevacizumab | CIViC B | EID7727 |
| PBRM1 Mutation | Renal Cell Carcinoma | Sensitivity/Response | Sunitinib + Everolimus | CIViC B | EID5996 |
| PIK3CA Mutation | Her2-receptor Positive Breast Cancer | Sensitivity/Response | Everolimus | CIViC B | EID1296 |
| PTEN Deletion | Prostate Cancer | Sensitivity/Response | Everolimus | CIViC B | EID1231 |
| PTEN Loss | Her2-receptor Positive Breast Cancer | Sensitivity/Response | Everolimus | CIViC B | EID1297 |
| PTEN Loss | Cancer | Sensitivity/Response | Everolimus | CIViC B | EID5840 |
| PTEN Mutation | Endometrial Carcinoma | Sensitivity/Response | Everolimus | CIViC B | EID1232 |
| STK11 F354L | Breast Cancer | Sensitivity/Response | Everolimus + Exemestane | CIViC B | EID5568 |
| TSC1 Frameshift Truncation | Invasive Bladder Transitional Cell Carcinoma | Sensitivity/Response | Everolimus | CIViC B | EID320 |
| TSC1 Loss-of-function | Bladder Carcinoma | Sensitivity/Response | Everolimus | CIViC B | EID155 |
| VHL Mutation | Renal Cell Carcinoma | Sensitivity/Response | Everolimus | CIViC B | EID5323 |
| BAP1 Mutation | Renal Cell Carcinoma | Resistance | Everolimus + Sunitinib | CIViC B | EID5339 |
| PTEN Loss | Bladder Carcinoma | Resistance | Everolimus | CIViC B | EID644 |
| AKT2 Amplification | Lung Adenocarcinoma | Sensitivity/Response | Vandetanib + Everolimus | CIViC C | EID1621 |
| FLCN Mutation | Papillary Renal Cell Carcinoma | Sensitivity/Response | Everolimus | CIViC C | EID5990 |
| FLCN c.1285dupC | Renal Cell Carcinoma | Sensitivity/Response | Everolimus | CIViC C | EID10137 |
| KIF5B::RET Fusion | Lung Adenocarcinoma | Sensitivity/Response | Everolimus + Vandetanib | CIViC C | EID1622 |
| MTOR E2014K AND MTOR E2419K | Transitional Cell Carcinoma | Sensitivity/Response | Pazopanib + Everolimus | CIViC C | EID1438 |
| MTOR Mutation | Bladder Carcinoma | Sensitivity/Response | Everolimus + Pazopanib | CIViC C | EID705 |
| PIK3CA H1047R | Her2-receptor Positive Breast Cancer | Sensitivity/Response | Everolimus + Fulvestrant | CIViC C | EID1623 |
| STK11 D194E | Pancreatic Cancer | Sensitivity/Response | Everolimus | CIViC C | EID1617 |
| TSC2 Q1178* | Thyroid Gland Carcinoma | Sensitivity/Response | Everolimus | CIViC C | EID1108 |
| MTOR F2108L | Thyroid Gland Carcinoma | Resistance | Everolimus | CIViC C | EID1110 |
| PTEN Expression | Her2-receptor Positive Breast Cancer | Resistance | Everolimus + Fulvestrant | CIViC C | EID1624 |
| EIF4EBP1 PHOSPHORYLATION | Gastric Adenocarcinoma | Sensitivity/Response | Everolimus | CIViC D | EID918 |
+12 more predictive associations (showing top 30 by level).
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline, but PharmGKB curates 8 clinical and 31 variant annotation(s) for this drug (gene-keyed; see PharmGKB).
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.
158 molecules share ≥1 primary target. Top 60 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| ALPELISIB | ChEMBL | Phase 4 (approved) | MTOR |
| AMIODARONE | ChEMBL | Phase 4 (approved) | MTOR |
| AMITRIPTYLINE | ChEMBL | Phase 4 (approved) | MTOR |
| AMOXAPINE | ChEMBL | Phase 4 (approved) | MTOR |
| AMSACRINE | ChEMBL | Phase 4 (approved) | MTOR |
| AZACITIDINE | ChEMBL | Phase 4 (approved) | MTOR |
| BROMOCRIPTINE | ChEMBL | Phase 4 (approved) | MTOR |
| CARVEDILOL | ChEMBL | Phase 4 (approved) | MTOR |
| CHLOROQUINE | ChEMBL | Phase 4 (approved) | MTOR |
| CHLORPROMAZINE | ChEMBL | Phase 4 (approved) | MTOR |
| CLEMASTINE | ChEMBL | Phase 4 (approved) | MTOR |
| CLOMIPRAMINE | ChEMBL | Phase 4 (approved) | MTOR |
| COPANLISIB | ChEMBL | Phase 4 (approved) | MTOR |
| CYCLOBENZAPRINE | ChEMBL | Phase 4 (approved) | MTOR |
| CYCLOSPORINE | ChEMBL | Phase 4 (approved) | MTOR |
| CYPROHEPTADINE | ChEMBL | Phase 4 (approved) | MTOR |
| CYTARABINE | ChEMBL | Phase 4 (approved) | MTOR |
| DASATINIB | ChEMBL | Phase 4 (approved) | MTOR |
| DEQUALINIUM | ChEMBL | Phase 4 (approved) | MTOR |
| DESIPRAMINE | ChEMBL | Phase 4 (approved) | MTOR |
| DISULFIRAM | ChEMBL | Phase 4 (approved) | MTOR |
| DOMPERIDONE | ChEMBL | Phase 4 (approved) | MTOR |
| DOPAMINE | ChEMBL | Phase 4 (approved) | MTOR |
| DOXAZOSIN | ChEMBL | Phase 4 (approved) | MTOR |
| EBASTINE | ChEMBL | Phase 4 (approved) | MTOR |
| EMETINE | ChEMBL | Phase 4 (approved) | MTOR |
| EPINEPHRINE | ChEMBL | Phase 4 (approved) | MTOR |
| FELODIPINE | ChEMBL | Phase 4 (approved) | MTOR |
| FENOFIBRATE | ChEMBL | Phase 4 (approved) | MTOR |
| FLUOROURACIL | ChEMBL | Phase 4 (approved) | MTOR |
| FLUOXETINE | ChEMBL | Phase 4 (approved) | MTOR |
| FLUPHENAZINE | ChEMBL | Phase 4 (approved) | MTOR |
| FLUSPIRILENE | ChEMBL | Phase 4 (approved) | MTOR |
| GUANABENZ | ChEMBL | Phase 4 (approved) | MTOR |
| HALOPERIDOL | ChEMBL | Phase 4 (approved) | MTOR |
| HYDROQUINONE | ChEMBL | Phase 4 (approved) | MTOR |
| IDARUBICIN | ChEMBL | Phase 4 (approved) | MTOR |
| IMIPRAMINE | ChEMBL | Phase 4 (approved) | MTOR |
| INDOMETHACIN | ChEMBL | Phase 4 (approved) | MTOR |
| ISOPROTERENOL | ChEMBL | Phase 4 (approved) | MTOR |
| ISOTRETINOIN | ChEMBL | Phase 4 (approved) | MTOR |
| LOPERAMIDE | ChEMBL | Phase 4 (approved) | MTOR |
| LORATADINE | ChEMBL | Phase 4 (approved) | MTOR |
| MAPROTILINE | ChEMBL | Phase 4 (approved) | MTOR |
| MASOPROCOL | ChEMBL | Phase 4 (approved) | MTOR |
| MEMANTINE | ChEMBL | Phase 4 (approved) | MTOR |
| METHYLDOPA | ChEMBL | Phase 4 (approved) | MTOR |
| MIBEFRADIL | ChEMBL | Phase 4 (approved) | MTOR |
| MIFEPRISTONE | ChEMBL | Phase 4 (approved) | MTOR |
| MITOXANTRONE | ChEMBL | Phase 4 (approved) | MTOR |
| NAFTOPIDIL | ChEMBL | Phase 4 (approved) | MTOR |
| NICARDIPINE | ChEMBL | Phase 4 (approved) | MTOR |
| NICLOSAMIDE | ChEMBL | Phase 4 (approved) | MTOR |
| NIFEDIPINE | ChEMBL | Phase 4 (approved) | MTOR |
| NORTRIPTYLINE | ChEMBL | Phase 4 (approved) | MTOR |
| PACLITAXEL | ChEMBL | Phase 4 (approved) | MTOR |
| PANCURONIUM | ChEMBL | Phase 4 (approved) | MTOR |
| PAROXETINE | ChEMBL | Phase 4 (approved) | MTOR |
| PENTAMIDINE ISETHIONATE | ChEMBL | Phase 4 (approved) | MTOR |
| PERPHENAZINE | ChEMBL | Phase 4 (approved) | MTOR |
Related Atlas pages
- Genes: MTOR
- Diseases: breast carcinoma, renal cell carcinoma, neoplasm, exocrine pancreatic carcinoma, clear cell renal carcinoma, lung neuroendocrine neoplasm, breast neoplasm, astrocytoma (excluding glioblastoma), anaplastic astrocytoma, pancreatic neoplasm, pancreatic neuroendocrine tumor, neuroendocrine neoplasm, tuberous sclerosis, hamartoma, renal cell adenocarcinoma, carcinoma, graft versus host disease, diffuse large B-cell lymphoma, gastric neoplasm, chronic kidney disease, cytomegalovirus infection, coronary restenosis, carcinoid syndrome, autosomal dominant polycystic kidney disease, kidney failure, kidney cancer, skin neoplasm, carcinoid tumor, coronary artery disorder, kidney neoplasm, malignant peripheral nerve sheath tumor, HER2 positive breast carcinoma, prostate carcinoma, cancer, endometrial carcinoma, infiltrating bladder urothelial carcinoma, urinary bladder carcinoma, lung adenocarcinoma, papillary renal cell carcinoma, transitional cell carcinoma, malignant pancreatic neoplasm, thyroid gland carcinoma, gastric adenocarcinoma
- Drugs: Alpelisib, Amiodarone, Amitriptyline, Amoxapine, Amsacrine, Azacitidine, Bromocriptine, Carvedilol, Chloroquine, Chlorpromazine, Clemastine, Clomipramine, Copanlisib, Cyclobenzaprine, Cyclosporine, Cyproheptadine, Cytarabine, Dasatinib, Dequalinium, Desipramine, Disulfiram, Domperidone, Dopamine, Doxazosin, Ebastine, Emetine, Epinephrine, Felodipine, Fenofibrate, Fluorouracil, Fluoxetine, Fluphenazine, Fluspirilene, Guanabenz, Haloperidol, Hydroquinone, Idarubicin, Imipramine, Indomethacin, Isoproterenol, Isotretinoin, Loperamide, Loratadine, Maprotiline, Masoprocol, Memantine, Methyldopa, Mibefradil, Mifepristone, Mitoxantrone, Naftopidil, Nicardipine, Niclosamide, Nifedipine, Nortriptyline, Paclitaxel, Pancuronium, Paroxetine, Perphenazine
- Biomarker genes: EIF4EBP1, FBXW7, FLCN, PTEN, RPS6, STK11, TSC1, TSC2, VHL