Sirolimus
drugOn this page
Also known as (-)-rapamycinAY-22989FyarroHyftorL04AA10Npc-12gNSC-226080RapalimusRapamuneRapamycinSIROLIMUS COMPONENT OF SM-88WY-090217RAPAMYCIN (SIROLIMUS)AY 22989NSC-22608049,10,12,13,14,21,22,23,2423R*,26S*,27S*,34AR*]]-RAPAMYCINRAPAMYCIN (AY-22989)Sirolimus analogue
Summary
Sirolimus (CHEMBL413) is an approved small-molecule immunosuppressive agent (ATC S01XA23) targeting FKBP1A; indicated across 158 conditions including neoplasm and eye disorder; with CIViC clinical evidence for 24 variant-indication associations (e.g. STK11 Loss in lung non-small cell carcinoma).
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: S01XA23 (+2 more)
- Targets: 1 (FKBP1A)
- Indications: 158 conditions
- Clinical trials: 571
- Precision-oncology evidence (CIViC): 24 variant–indication associations
- Chemistry: C51H79NO13
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL413 |
| Name | Sirolimus |
| Type | Small molecule |
| Max phase | 4 |
| ChEBI | CHEBI:9168 |
| ATC | S01XA23, L04AH01, L01EG04 |
| Molecular formula | C51H79NO13 |
| InChIKey | QFJCIRLUMZQUOT-HPLJOQBZSA-N |
SMILES: [H][C@@]1(C[C@@H](C)[C@]2([H])CC(=O)[C@H](C)/C=C(\C)[C@@H](O)[C@@H](OC)C(=O)[C@H](C)C[C@H](C)/C=C/C=C/C=C(\C)[C@@H](OC)C[C@]3([H])CC[C@@H](C)[C@@](O)(O3)C(=O)C(=O)N3CCCC[C@@]3([H])C(=O)O2)CC[C@@H](O)[C@H](OC)C1
ChEBI definition: A macrolide lactam isolated from Streptomyces hygroscopicus consisting of a 29-membered ring containing 4 trans double bonds, three of which are conjugated. It is an antibiotic, immunosupressive and antineoplastic agent.
Pharmacological roles (ChEBI): immunosuppressive agent, antineoplastic agent, antibacterial drug, mTOR inhibitor, anticoronaviral agent, geroprotector.
Other ChEBI roles (chemical / environmental): bacterial metabolite.
Also known as: (-)-rapamycin, AY-22989, Fyarro, Hyftor, L04AA10, Npc-12g, NSC-226080, Rapalimus, Rapamune, Rapamycin, Sirolimus, SIROLIMUS COMPONENT OF SM-88
Patent coverage: 46,327 distinct patent families (172,798 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 |
|---|---|---|---|---|---|
| FKBP1A | FKBP prolyl isomerase 1A | Inhibition | 9.7 | 21.5% | P62942 |
Broader ChEMBL bioactivity targets: 34 (assay-derived). Sample: Pyruvate kinase PKM, Serine/threonine-protein kinase mTOR, Nuclear receptor ROR-gamma, Solute carrier organic anion transporter family member 1B1, Programmed cell death protein 4, Peptidyl-prolyl cis-trans isomerase FKBP1A, Amine oxidase [flavin-containing] A, Equilibrative nucleoside transporter 1, Peptidyl-prolyl cis-trans isomerase FKBP5, Muscarinic acetylcholine receptor M1.
Bioactivity
ChEMBL activities: 63 potent at pChembl ≥ 5 of 74 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| Q9JLN9 | 10.3 | IC50 | 0.05 | nM | CHEMBL_ACT_19453018 |
| MTOR | 10.3 | IC50 | 0.05 | nM | CHEMBL_ACT_24962478 |
| MTOR | 10.3 | IC50 | 0.05 | nM | CHEMBL_ACT_25872951 |
| MTOR | 10.22 | IC50 | 0.06 | nM | CHEMBL_ACT_25442004 |
| MTOR | 10 | IC50 | 0.1 | nM | CHEMBL_ACT_20681615 |
| MTOR | 10 | IC50 | 0.1 | nM | CHEMBL_ACT_2200407 |
| MTOR | 10 | IC50 | 0.1 | nM | CHEMBL_ACT_25044933 |
| MTOR | 10 | IC50 | 0.1 | nM | CHEMBL_ACT_25937259 |
| MTOR | 10 | IC50 | 0.1 | nM | CHEMBL_ACT_29205856 |
| FKBP1A | 9.89 | Kd | 0.13 | nM | CHEMBL_ACT_26165916 |
| FKBP1B | 9.7 | Kd | 0.2 | nM | CHEMBL_ACT_15049565 |
| FKBP1A | 9.7 | Kd | 0.2 | nM | CHEMBL_ACT_25848532 |
| FKBP1A | 9.7 | Ki | 0.2 | nM | CHEMBL_ACT_992867 |
| FKBP1A | 9.35 | IC50 | 0.45 | nM | CHEMBL_ACT_1605332 |
| MTOR | 9.35 | IC50 | 0.45 | nM | CHEMBL_ACT_1615020 |
| EIF4E | 9.3 | Kd | 0.5 | nM | CHEMBL_ACT_5113953 |
| FKBP1A | 9.22 | Ki | 0.6 | nM | CHEMBL_ACT_18721920 |
| MTOR | 9.22 | Kd | 0.6 | nM | CHEMBL_ACT_18813393 |
| FKBP1B | 9.22 | Kd | 0.6 | nM | CHEMBL_ACT_26165919 |
| FKBP1A | 9.22 | IC50 | 0.6 | nM | CHEMBL_ACT_64601 |
| MTOR | 9.1 | IC50 | 0.8 | nM | CHEMBL_ACT_23207009 |
| P26883 | 9 | IC50 | 1 | nM | CHEMBL_ACT_18721939 |
| FKBP1A | 8.96 | IC50 | 1.1 | nM | CHEMBL_ACT_719989 |
| MTOR | 8.8 | IC50 | 1.6 | nM | CHEMBL_ACT_1659907 |
| FKBP1A | 8.8 | IC50 | 1.6 | nM | CHEMBL_ACT_18721917 |
| FKBP1A | 8.8 | IC50 | 1.6 | nM | CHEMBL_ACT_937964 |
| FKBP5 | 8.52 | Ki | 3 | nM | CHEMBL_ACT_12726904 |
| MTOR | 8.46 | IC50 | 3.47 | nM | CHEMBL_ACT_881846 |
| FKBP5 | 8.43 | Kd | 3.7 | nM | CHEMBL_ACT_25072928 |
| FKBP4 | 8.38 | Kd | 4.2 | nM | CHEMBL_ACT_25072934 |
Target pathways
Aggregated over 1 target gene(s): FKBP1A.
Top Reactome pathways
7 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| mTORC1-mediated signalling | 1 | FKBP1A |
| Calcineurin activates NFAT | 1 | FKBP1A |
| TGF-beta receptor signaling activates SMADs | 1 | FKBP1A |
| TGF-beta receptor signaling in EMT (epithelial to mesenchymal transition) | 1 | FKBP1A |
| TGFBR1 LBD Mutants in Cancer | 1 | FKBP1A |
| Potential therapeutics for SARS | 1 | FKBP1A |
| SARS-CoV-1 activates/modulates innate immune responses | 1 | FKBP1A |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| heart morphogenesis | 1 |
| protein folding | 1 |
| ‘de novo’ protein folding | 1 |
| regulation of cardiac muscle contraction by regulation of the release of sequestered calcium ion | 1 |
| regulation of skeletal muscle contraction by regulation of release of sequestered calcium ion | 1 |
| negative regulation of transforming growth factor beta receptor signaling pathway | 1 |
| regulation of protein localization | 1 |
| negative regulation of activin receptor signaling pathway | 1 |
| protein refolding | 1 |
| T cell activation | 1 |
| positive regulation of canonical NF-kappaB signal transduction | 1 |
| regulation of immune response | 1 |
| protein maturation | 1 |
| ventricular cardiac muscle tissue morphogenesis | 1 |
| heart trabecula formation | 1 |
Indications & clinical
Indications
158 indications (8 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 |
|---|---|---|---|
| neoplasm | 4 | MONDO:0005070 | EFO:0000616 |
| eye disorder | 4 | MONDO:0005328 | EFO:0003966 |
| tuberous sclerosis | 4 | MONDO:0001734 | MONDO:0001734 |
| graft versus host disease | 3 | MONDO:0013730 | EFO:0004599 |
| hepatocellular carcinoma | 3 | MONDO:0007256 | EFO:0000182 |
| fibroma | 3 | MONDO:0005167 | EFO:0002424 |
| chronic kidney disease | 3 | MONDO:0005300 | EFO:0003884 |
| Hodgkins lymphoma | 3 | MONDO:0004952 | EFO:0000183 |
| non-Hodgkin lymphoma | 3 | MONDO:0018908 | EFO:0005952 |
| inclusion body myositis | 3 | MONDO:0007827 | EFO:0007323 |
| autosomal dominant polycystic kidney disease | 3 | MONDO:0004691 | EFO:1001496 |
| lichen planus, oral | 3 | MONDO:0043923 | EFO:0008517 |
| skin carcinoma | 3 | MONDO:0002656 | EFO:0009259 |
| Birt-Hogg-Dube syndrome 1 | 3 | MONDO:0800445 | Orphanet:122 |
| skin neoplasm | 3 | MONDO:0002531 | MONDO:0002898 |
| breast neoplasm | 3 | MONDO:0021100 | MONDO:0007254 |
| diabetic kidney disease | 3 | MONDO:0005016 | EFO:0000401 |
| neurofibromatosis | 3 | MONDO:0021061 | EFO:0008514 |
| type 1 diabetes mellitus | 3 | MONDO:0005147 | MONDO:0005147 |
| lymphangioleiomyomatosis | 3 | MONDO:0011705 | MONDO:0011705 |
| pachyonychia congenita | 3 | MONDO:0016471 | MONDO:0016471 |
| lymphedema | 3 | MONDO:0019297 | MONDO:0019313 |
| influenza | 3 | MONDO:0005812 | EFO:0007328 |
| acute lymphoblastic leukemia | 2 | MONDO:0004967 | EFO:0000220 |
| sarcoma | 2 | MONDO:0005089 | EFO:0000691 |
| hypertensive disorder | 2 | MONDO:0005044 | EFO:0000537 |
| plasma cell myeloma | 2 | MONDO:0009693 | EFO:0001378 |
| age-related macular degeneration | 2 | MONDO:0005150 | EFO:0001365 |
| primary cutaneous T-cell non-Hodgkin lymphoma | 2 | MONDO:0000607 | EFO:0002913 |
| dry eye syndrome | 2 | MONDO:0006733 | EFO:1000906 |
| systemic sclerosis | 2 | MONDO:0005100 | EFO:0000717 |
| exocrine pancreatic carcinoma | 2 | MONDO:0005192 | EFO:0002618 |
| B-cell chronic lymphocytic leukemia | 2 | MONDO:0004948 | EFO:0000095 |
| myelodysplastic syndrome | 2 | MONDO:0018881 | EFO:0000198 |
| acute myeloid leukemia | 2 | MONDO:0018874 | EFO:0000222 |
| diffuse large B-cell lymphoma | 2 | MONDO:0018905 | EFO:0000403 |
| germ cell tumor | 2 | MONDO:0005040 | EFO:0000514 |
| glioblastoma | 2 | MONDO:0018177 | EFO:0000519 |
| leukemia | 2 | MONDO:0005059 | EFO:0000565 |
| lymphoma | 2 | MONDO:0005062 | EFO:0000574 |
| neuroblastoma | 2 | MONDO:0005072 | EFO:0000621 |
| osteosarcoma | 2 | MONDO:0009807 | EFO:0000637 |
| renal cell carcinoma | 2 | MONDO:0005086 | EFO:0000681 |
| IgA glomerulonephritis | 2 | MONDO:0005342 | EFO:0004194 |
| focal segmental glomerulosclerosis | 2 | MONDO:0100313 | EFO:0004236 |
| vascular malformation | 2 | MONDO:0024291 | EFO:0006888 |
| aplastic anemia | 2 | MONDO:0015909 | EFO:0006927 |
| pineoblastoma | 2 | MONDO:0016722 | EFO:1000475 |
| intermediate uveitis | 2 | MONDO:0006806 | EFO:1000986 |
| posterior uveitis | 2 | MONDO:0006918 | EFO:1001119 |
| gliosarcoma | 2 | MONDO:0016681 | EFO:1001465 |
| mantle cell lymphoma | 2 | MONDO:0018876 | EFO:1001469 |
| hematologic disorder | 2 | MONDO:0005570 | HP:0001871 |
| arteriovenous malformations of the brain | 2 | MONDO:0007154 | Orphanet:46724 |
| ovarian carcinoma | 2 | MONDO:0005140 | EFO:0001075 |
| bronchitis | 2 | MONDO:0003781 | EFO:0009661 |
| soft tissue sarcoma | 2 | MONDO:0018078 | EFO:1001968 |
| kidney failure | 2 | MONDO:0001106 | EFO:1002048 |
| brain aneurysm | 2 | MONDO:0005291 | EFO:0003870 |
| polycystic kidney disease | 2 | MONDO:0020642 | EFO:0008620 |
| anemia | 2 | MONDO:0002280 | MONDO:0002280 |
| Castleman disease | 2 | MONDO:0015564 | MONDO:0015564 |
| beta thalassemia | 2 | MONDO:0019402 | Orphanet:848 |
| depressive disorder | 2 | MONDO:0002050 | MONDO:0002050 |
| severe acute respiratory syndrome | 2 | MONDO:0005091 | MONDO:0100096 |
| lupus nephritis | 2 | MONDO:0005556 | EFO:0005761 |
| IgG4-related retroperitoneal fibrosis | 2 | MONDO:0018848 | MONDO:0018848 |
| vitiligo | 2 | MONDO:0008661 | EFO:0004208 |
| multiple system atrophy | 2 | MONDO:0007803 | EFO:1001050 |
| inborn error of immunity | 2 | MONDO:0003778 | HP:0002721 |
| amyotrophic lateral sclerosis | 2 | MONDO:0004976 | MONDO:0004976 |
| Sturge-Weber syndrome | 2 | MONDO:0008501 | MONDO:0008501 |
| sickle cell disease | 2 | MONDO:0011382 | MONDO:0011382 |
| severe combined immunodeficiency | 2 | MONDO:0015974 | MONDO:0015974 |
| neurofibromatosis type 1 | 2 | MONDO:0018975 | MONDO:0018975 |
| sarcoidosis | 2 | MONDO:0019338 | MONDO:0019338 |
| Fanconi anemia | 2 | MONDO:0019391 | MONDO:0019391 |
| hemangioendothelioma | 2 | MONDO:0021121 | MONDO:0021121 |
| liver disorder | 2 | MONDO:0005154 | EFO:0001421 |
| neoplasm with perivascular epithelioid cell differentiation | 2 | MONDO:0006359 | EFO:1000464 |
| autoimmune hemolytic anemia | 2 | MONDO:0020108 | EFO:1001264 |
| systemic lupus erythematosus | 2 | MONDO:0007915 | MONDO:0007915 |
| paraganglioma | 2 | MONDO:0000448 | EFO:1000453 |
| blood platelet disease | 2 | MONDO:0002245 | MONDO:0002245 |
| ovarian cancer | 2 | MONDO:0008170 | MONDO:0008170 |
| choroidal neovascularization | 2 | MONDO:0810000 | MONDO:0810000 |
| kidney disorder | 1 | MONDO:0005240 | EFO:0003086 |
| diabetic retinopathy | 1 | MONDO:0005266 | EFO:0003770 |
| HIV infectious disease | 1 | MONDO:0005109 | EFO:0000180 |
| relapsing-remitting multiple sclerosis | 1 | MONDO:0005314 | EFO:0003929 |
| rectal cancer | 1 | MONDO:0006519 | EFO:1000657 |
| chronic myeloid leukemia | 1 | MONDO:0011996 | EFO:0000339 |
| small cell lung carcinoma | 1 | MONDO:0008433 | EFO:0000702 |
| squamous cell lung carcinoma | 1 | MONDO:0005097 | EFO:0000708 |
| non-small cell lung carcinoma | 1 | MONDO:0005233 | EFO:0003060 |
| anterior uveitis | 1 | MONDO:0006651 | EFO:1000811 |
| metastatic prostate carcinoma | 1 | MONDO:0004956 | EFO:0000196 |
| lung adenocarcinoma | 1 | MONDO:0005061 | EFO:0000571 |
| plasma cell leukemia | 1 | MONDO:0018689 | EFO:0006475 |
| fallopian tube carcinoma | 1 | MONDO:0006206 | EFO:1000251 |
| malignant peripheral nerve sheath tumor | 1 | MONDO:0017827 | EFO:0000760 |
| central nervous system neoplasm | 1 | MONDO:0006130 | EFO:1000158 |
| scleritis | 1 | MONDO:0001718 | MONDO:0001718 |
| hyperinsulinism | 1 | MONDO:0002177 | MONDO:0002177 |
| pulmonary hypertension | 1 | MONDO:0005149 | MONDO:0005149 |
| lung neoplasm | 1 | MONDO:0021117 | MONDO:0008903 |
| hematopoietic and lymphoid system neoplasm | 1 | MONDO:0002334 | MONDO:0044881 |
| acute lung injury | 1 | MONDO:0015796 | EFO:0004610 |
| acute respiratory distress syndrome | 1 | MONDO:0006502 | EFO:1000637 |
| Gaucher disease | 1 | MONDO:0018150 | Orphanet:77260 |
| desmoid tumor | 1 | MONDO:0007608 | EFO:0009907 |
| heart failure | 1 | MONDO:0005252 | EFO:0003144 |
| thalassemia | 1 | MONDO:0000984 | EFO:1001996 |
| Parkinson disease | 1 | MONDO:0005180 | MONDO:0005180 |
| alcohol abuse | 1 | MONDO:0002046 | MONDO:0007079 |
| frontotemporal dementia | 1 | MONDO:0017276 | MONDO:0017276 |
| chronic granulomatous disease | 1 | MONDO:0018305 | MONDO:0018305 |
| Sjogren syndrome | 1 | MONDO:0010030 | EFO:0000699 |
| plasma cell neoplasm | 1 | MONDO:0004959 | EFO:0000200 |
| leiomyosarcoma | 1 | MONDO:0005058 | EFO:0000564 |
| prostate carcinoma | 1 | MONDO:0005159 | EFO:0001663 |
| eosinophilic esophagitis | 1 | MONDO:0005361 | EFO:0004232 |
| respiratory failure | 1 | MONDO:0021113 | EFO:0009686 |
| panuveitis | 1 | MONDO:0017255 | EFO:1001082 |
| fallopian tube neoplasm | 1 | MONDO:0021092 | MONDO:0002158 |
| Alzheimer disease | 1 | MONDO:0004975 | MONDO:0004975 |
| glioma | 1 | MONDO:0021042 | MONDO:0021637 |
| pemphigus | 0 | MONDO:0006594 | EFO:1000749 |
| lymphoid leukemia | 0 | MONDO:0005402 | EFO:0004289 |
29 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 571.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 185 |
| PHASE1 | 100 |
| PHASE1/PHASE2 | 83 |
| PHASE4 | 76 |
| PHASE3 | 50 |
| Not specified | 37 |
| PHASE2/PHASE3 | 22 |
| EARLY_PHASE1 | 18 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT05193565 | PHASE4 | ACTIVE_NOT_RECRUITING | Study to Compare the Efficacy and Safety After Conversion to RaparoBell® or My-Rept® in Kidney Transplant Patients |
| NCT06236022 | PHASE4 | RECRUITING | The Effects of Sirolimus in Patients With Dilated Cardiomyopathy Infected With Kaposi Sarcoma-associated Virus |
| NCT06722586 | PHASE4 | RECRUITING | Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia |
| NCT07426484 | PHASE4 | NOT_YET_RECRUITING | Cosibelimab for CSCC in Patients With Kidney Transplant or Hematologic Malignancy |
| NCT07542314 | PHASE4 | NOT_YET_RECRUITING | Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting |
| NCT00044720 | PHASE4 | COMPLETED | Study Evaluating Sirolimus in End Stage Renal Disease in High Risk Kidney Transplant Recipients |
| NCT00118742 | PHASE4 | COMPLETED | Liver Spare the Nephron (STN) Study - A Study of CellCept (Mycophenolate Mofetil) and Sirolimus in Recipients of a Liver Transplant |
| NCT00121810 | PHASE4 | COMPLETED | Kidney Spare the Nephron (STN) Study - A Study of CellCept (Mycophenolate Mofetil) and Rapamune (Sirolimus) in Kidney Transplant Recipients |
| NCT00123331 | PHASE4 | COMPLETED | Rapamycin Use in Calcineurin Inhibitor (CNI)-Free Immunosuppression for Stabilization/Improvement of Renal Function After Heart Transplantation |
| NCT00129961 | PHASE4 | COMPLETED | Study Evaluating the Effect of Sirolimus on Non-Melanoma Skin Cancer in Kidney Transplant Recipients |
| NCT00166712 | PHASE4 | TERMINATED | A Trial of Two Steroid-Free Approaches Toward Mycophenolate Mofetil-Based Monotherapy Immunosuppression |
| NCT00166816 | PHASE4 | UNKNOWN | The Pharmacokinetics of Sirolimus When Combined With Cyclosporine or Tacrolimus in Renal Transplant Patients |
| NCT00166829 | PHASE4 | UNKNOWN | The Effect of Sirolimus on the Pharmacokinetics of Tacrolimus |
| NCT00166842 | PHASE4 | UNKNOWN | Sirolimus Blood Concentrations on Conversion From Oral Solution to Tablets |
| NCT00188955 | PHASE4 | COMPLETED | Pilot Study on the Use of Sirolimus to Treat Chronic Allograft Nephropathy in Children After Kidney Transplant |
| NCT00195429 | PHASE4 | COMPLETED | A Study Comparing the Withdrawal of Steroids or Tacrolimus in Kidney Transplant Recipients |
| NCT00195481 | PHASE4 | COMPLETED | Study Evaluating Sirolimus in Kidney Transplant Recipients in India |
| NCT00223028 | PHASE4 | COMPLETED | Investigation of the Steady State Pharmacokinetics of Sirolimus, Mycophenolat Mofetil and Fluvastatin After Renal Transplantation |
| NCT00223678 | PHASE4 | COMPLETED | Mycophenolate Mofetil and Rapamycin as Secondary Intervention vs. Continuation of Calcineurin Inhibitors in Patients at Risk for Chronic Renal Allograft Failure |
| NCT00252655 | PHASE4 | UNKNOWN | Use of Sirolimus vs. Tacrolimus For African-American Renal Transplant Recipients |
| NCT00254709 | PHASE4 | COMPLETED | Study Evaluating Two Different Sirolimus-based Immunosuppressive Regimens in Elderly Kidney Transplant Recipients |
| NCT00261820 | PHASE4 | COMPLETED | Study Comparing Two Immunosuppressive Regimens in De Novo Renal Allograft Recipients |
| NCT00266123 | PHASE4 | COMPLETED | Study Comparing Two Sirolimus Regimens vs. Tacrolimus and Mycophenolate Mofetil Regimen in Kidney Transplant Recipients |
| NCT00275522 | PHASE4 | COMPLETED | The Comparison of Three Different Immunosuppressant Regimens in Kidney Transplant Recipients. |
| NCT00275535 | PHASE4 | COMPLETED | The Comparison of Tacrolimus and Sirolimus Immunosuppression Based Drug Regimens in Kidney Transplant Recipients |
| NCT00282217 | PHASE4 | COMPLETED | Study Evaluating Sirolimus in the Treatment of Kidney Transplant |
| NCT00290069 | PHASE4 | UNKNOWN | Renal Function Optimization With Mycophenolate Mofetil (MMF) Immunosuppressor Regimes (ALHAMBRA) |
| NCT00305396 | PHASE4 | COMPLETED | Calcineurin Inhibitor Avoidance With Thymoglobulin and Sirolimus in Kidney Transplantation |
| NCT00306397 | PHASE4 | COMPLETED | Pilot Study to Investigate a Steroid Free Immunosuppressive Regimen for Renal Transplant Recipients |
| NCT00321906 | PHASE4 | COMPLETED | Comparison of Sirolimus and Azathioprine in Lung Transplantation |
| NCT00352092 | PHASE4 | COMPLETED | Pilot Study for HLA Identical Living Donor Renal Transplant Recipients |
| NCT00369382 | PHASE4 | COMPLETED | Study Of The Safety And Efficacy Of Conversion From A CNI To Sirolimus In Renally-Impaired Heart Transplant Recipients |
| NCT00374647 | PHASE4 | COMPLETED | Study to Evaluate Safety and Efficacy of Early Calcineurin Inhibitor Withdrawal in Primary Renal Allografts |
| NCT00452361 | PHASE4 | TERMINATED | Study Evaluating of Calcineurin Inhibitors Versus Sirolimus in Renal Allograft Recipient |
| NCT00493194 | PHASE4 | UNKNOWN | Fibrosis in Renal Allografts |
| NCT00507793 | PHASE4 | COMPLETED | Study Evaluating the Efficacy and Safety of Cyclosporine Reduction in Kidney Transplant Recipients Receiving Sirolimus |
| NCT00552669 | PHASE4 | COMPLETED | Study of Oral Rapamycin Plus Bare Metal Stents vs Drug Eltuting Stents |
| NCT00556933 | PHASE4 | COMPLETED | Improved Induction and Maintenance Immunosuppression in Kidney Transplantation |
| NCT00598533 | PHASE4 | COMPLETED | Efficacy Study of Rapamycin- vs. Zotarolimus-Eluting Stents to Reduce Coronary Restenosis |
| NCT00681213 | PHASE4 | COMPLETED | Tacrolimus/Sirolimus Versus Tacrolimus/Mycophenolate Mofetil (MMF) Versus Neoral/Sirolimus in Adult, Primary Kidney Transplantation |
Clinical evidence (CIViC)
Variant × indication × effect (24 predictive associations from 28 curated evidence items):
| Variant | Indication | Effect | Therapy | Level | CIViC |
|---|---|---|---|---|---|
| STK11 Loss | Lung Non-small Cell Carcinoma | Sensitivity/Response | Sirolimus | CIViC B | EID2314 +3 |
| EGFR P753S | Skin Squamous Cell Carcinoma | Sensitivity/Response | Cetuximab + Sirolimus | CIViC C | EID1089 |
| STK11 Loss | Peutz-Jeghers Syndrome | Sensitivity/Response | Sirolimus | CIViC D | EID1618 +1 |
| CRLF2 Rearrangement | Acute Lymphoblastic Leukemia | Sensitivity/Response | Sirolimus | CIViC D | EID11097 |
| ERBB2 Y772_A775DUP | Lung Non-small Cell Carcinoma | Sensitivity/Response | Sirolimus + Afatinib | CIViC D | EID960 |
| FBXW7 Loss-of-function | Cancer | Sensitivity/Response | Sirolimus | CIViC D | EID1632 |
| FLCN Loss | Renal Carcinoma | Sensitivity/Response | Sirolimus | CIViC D | EID5991 |
| IGH::CRLF2 Fusion AND JAK2 R683G | B-lymphoblastic Leukemia/lymphoma With BCR-ABL1 | Sensitivity/Response | Sirolimus | CIViC D | EID7641 |
| MTOR C1483F | Solid Tumor | Sensitivity/Response | Sirolimus | CIViC D | EID7324 |
| MTOR C1483Y | T-cell Acute Lymphoblastic Leukemia | Sensitivity/Response | Sirolimus | CIViC D | EID1320 |
| MTOR E1799K | Clear Cell Renal Cell Carcinoma | Sensitivity/Response | Sirolimus | CIViC D | EID1321 |
| MTOR F1888L | Solid Tumor | Sensitivity/Response | Sirolimus | CIViC D | EID7325 |
| MTOR L2230V | Solid Tumor | Sensitivity/Response | Sirolimus | CIViC D | EID7327 |
| MTOR S2215F | Solid Tumor | Sensitivity/Response | Sirolimus | CIViC D | EID7326 |
| MTOR S2215Y | Endometrial Adenocarcinoma | Sensitivity/Response | Sirolimus | CIViC D | EID1319 |
| MTOR T1977K | Solid Tumor | Sensitivity/Response | Sirolimus | CIViC D | EID7323 |
| NF1 Mutation | Skin Melanoma | Sensitivity/Response | Mirdametinib + Sirolimus | CIViC D | EID1469 |
| PIK3CA E542K | Breast Cancer | Sensitivity/Response | Sirolimus | CIViC D | EID311 |
| STK11 Loss | Lung Non-small Cell Carcinoma | Sensitivity/Response | Everolimus + Sirolimus | CIViC D | EID1620 |
| TSC1 Frameshift Truncation | Lung Non-small Cell Carcinoma | Sensitivity/Response | Sirolimus | CIViC D | EID154 |
| MTOR A2034V | Breast Cancer | Resistance | Sirolimus | CIViC D | EID1547 |
| MTOR F2108L | Breast Cancer | Resistance | Sirolimus | CIViC D | EID1543 |
| MTOR M2327I | Breast Cancer | Resistance | RapaLink-1 + Sirolimus | CIViC D | EID1545 |
| RHEB Y35N | Kidney Cancer | Resistance | Sirolimus | CIViC D | EID6340 |
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.
5 molecules share ≥1 primary target. Top 5 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| CYCLOSPORINE | ChEMBL | Phase 4 (approved) | FKBP1A |
| TACROLIMUS | ChEMBL | Phase 4 (approved) | FKBP1A |
| THIABENDAZOLE | ChEMBL | Phase 4 (approved) | FKBP1A |
| BIRICODAR | ChEMBL | Phase 2 | FKBP1A |
| CYCLOHEXIMIDE | ChEMBL | Phase 2 | FKBP1A |
Related Atlas pages
- Genes: FKBP1A
- Diseases: neoplasm, eye disorder, tuberous sclerosis, graft versus host disease, hepatocellular carcinoma, chronic kidney disease, Hodgkins lymphoma, non-Hodgkin lymphoma, inclusion body myositis, autosomal dominant polycystic kidney disease, lichen planus, oral, skin carcinoma, Birt-Hogg-Dube syndrome 1, skin neoplasm, breast neoplasm, diabetic kidney disease, neurofibromatosis, type 1 diabetes mellitus, lymphangioleiomyomatosis, pachyonychia congenita, lymphedema, influenza, skin squamous cell carcinoma, Peutz-Jeghers syndrome, acute lymphoblastic leukemia, cancer, renal carcinoma, T-cell acute lymphoblastic leukemia, nonpapillary renal cell carcinoma, endometrium adenocarcinoma, cutaneous melanoma, breast carcinoma
- Drugs: Cyclosporine, Tacrolimus, Thiabendazole
- Biomarker genes: FBXW7, MTOR, STK11, TSC1