theragenomics: targeted therapies
thasso provides here an ever growing table of targeted therapies, meaning a gene, an allelic variant there of, or a mutation is the at the base of the decision if a therapy may successfully be applied to a patient or a group of patients who carry the according genetic outfit. At present, this concept is mostly, but not exclusively, applied in cancer treatment. For more information, particularly in oncology you may also consult The National Cancer Institute Fact Sheet.
| Pharmacologically active ingredient | Drug name | Gene(s) / Molecular Target(s) | FDA-approved indication(s) |
|---|---|---|---|
| Ado-Trastuzumab Emtansine | Kadcyla | HER2 (ERBB2/neu) | Breast cancer (HER2+) |
| Afatinib | Gilotrif | EGFR (HER1/ERBB1), HER2 (ERBB2/neu) | Non-small cell lung cancer (with EGFR exon 19 deletions or exon 21 substitution (L858R) mutations) |
| Aldesleukin | Proleukin | Renal cell carcinoma Melanoma |
|
| Alectinib | Alecensa | ALK | Non-small cell lung cancer (with ALK fusion) |
| Alemtuzumab | Campath | CD52 | B-cell chronic lymphocytic leukemia |
| Axitinib | Inlyta | KIT, PDGFRβ, VEGFR1/2/3 | Renal cell carcinoma |
| Belimumab | Benlysta | BAFF | Lupus erythematosus |
| Belinostat | Beleodaq | HDAC | Peripheral T-cell lymphoma |
| Bevacizumab | Avastin | VEGF ligand | Cervical cancer Colorectal cancer Fallopian tube cancer Glioblastoma Non-small cell lung cancer Ovarian cancer Peritoneal cancer Renal cell carcinoma |
| Bortezomib | Velcade | Proteasome | Multiple myeloma Mantle cell lymphoma |
| Bosutinib | Bosulif | ABL | Chronic myelogenous leukemia (Philadelphia chromosome positive) |
| Brentuximab vedotin | Adcetris | CD30 | Hodgkin lymphoma Anaplastic large cell lymphoma |
| Cabozantinib | Cabometyx | FLT3, KIT, MET, RET, VEGFR2 | Medullary thyroid cancer Renal cell carcinoma |
| Canakinumab | Ilaris | IL-1β | Juvenile idiopathic arthritis Cryopyrin-associated periodic syndromes |
| Carfilzomib | Kyprolis | Proteasome | Multiple myeloma |
| Ceritinib | Zykadia | ALK | Non-small cell lung cancer (with ALK fusion) |
| Cetuximab | Erbitux | EGFR (HER1/ERBB1) | Colorectal cancer (KRAS wild type) Squamous cell cancer of the head and neck |
| Cobimetinib | Cotellic | MEK | Melanoma (with BRAF V600E or V600K mutation) |
| Crizotinib | Xalkori | ALK, MET, ROS1 | Non-small cell lung cancer (with ALK fusion or ROS1 gene alteration) |
| Dabrafenib | Tafinlar | BRAF | Melanoma (with BRAF V600 mutation) |
| Daratumumab | Darzalex | CD38 | Multiple myeloma |
| Dasatinib | Sprycel | ABL | Chronic myelogenous leukemia (Philadelphia chromosome positive) Acute lymphoblastic leukemia (Philadelphia chromosome positive) |
| Denosumab | Xgeva | RANKL | Giant cell tumor of the bone |
| Dinutuximab | Unituxin | B4GALNT1 (GD2) | Pediatric neuroblastoma |
| Elotuzumab | Empliciti | SLAMF7 (CS1/CD319/CRACC) | Multiple myeloma |
| Erlotinib | Tarceva | EGFR (HER1/ERBB1) | Non-small cell lung cancer Pancreatic cancer |
| Everolimus | Afinitor | mTOR | Pancreatic, gastrointestinal, or lung origin neuroendocrine tumor Renal cell carcinoma Nonresectable subependymal giant cell astrocytoma associated with tuberous sclerosis Breast cancer (HR+, HER2-) |
| Gefitinib | Iressa | EGFR (HER1/ERBB1) | Non-small cell lung cancer (with EGFR exon 19 deletions or exon 21 substitution (L858R) mutations) |
| Ibritumomab Tiuxetan | Zevalin | CD20 | Non-Hodgkin's lymphoma |
| Ibrutinib | Imbruvica | BTK | Mantle cell lymphoma Chronic lymphocytic leukemia Waldenstrom's macroglobulinemia |
| Idelalisib | Zydelig | PI3Kδ | Chronic lymphocytic leukemia Follicular B-cell non-Hodgkin lymphoma Small lymphocytic lymphoma |
| Imatinib | Gleevec | KIT, PDGFR, ABL | GI stromal tumor (KIT+) Dermatofibrosarcoma protuberans Multiple hematologic malignancies including Philadelphia chromosome-positive ALL and CML |
| Ipilimumab | Yervoy | CTLA-4 | Melanoma |
| Ixazomib | Ninlaro | Proteasome | Multiple Myeloma |
| Lapatinib | Tykerb | HER2 (ERBB2/neu), EGFR (HER1/ERBB1) | Breast cancer (HER2+) |
| Lenvatinib | Lenvima | VEGFR2 | Thyroid cancer |
| Necitumumab | Portrazza | EGFR (HER1/ERBB1) | Squamous non-small cell lung cancer |
| Nilotinib | Tasigna | ABL | Chronic myelogenous leukemia (Philadelphia chromosome positive) |
| Nivolumab | Opdivo | PD-1 | Melanoma Non-small cell lung cancer Renal cell carcinoma |
| Obinutuzumab | Gazyva | CD20 | Chronic lymphocytic leukemia Follicular lymphoma |
| Ofatumumab | Arzerra | CD20 | Chronic lymphocytic leukemia |
| Olaparib | Lynparza | PARP | Ovarian cancer (with BRCA mutation) |
| Osimertinib | Tagrisso | EGFR | Non-small cell lung cancer (with EGFR T790M mutation) |
| Palbociclib | Ibrance | CDK4, CDK6 | Breast cancer (ER+, HER2-) |
| Panitumumab | Vectibix | EGFR (HER1/ERBB1) | Colorectal cancer (KRAS wild type) |
| Panobinostat | Farydak | HDAC | Multiple myeloma |
| Pazopanib | Votrient | VEGFR, PDGFR, KIT | Renal cell carcinoma |
| Pembrolizumab | Keytruda | PD-1 | Melanoma Non-small cell lung cancer (PD-L1+) |
| Pertuzumab | Perjeta | HER2 (ERBB2/neu) | Breast cancer (HER2+) |
| Ponatinib | Iclusig | ABL, FGFR1-3, FLT3, VEGFR2 | Chronic myelogenous leukemia Acute lymphoblastic leukemia (Philadelphia chromosome positive) |
| Ramucirumab | Cyramza | VEGFR2 | Colorectal cancer Gastric cancer or Gastroesophageal junction (GEJ) adenocarcinoma Non-small cell lung cancer |
| Regorafenib | Stivarga | KIT, PDGFRβ, RAF, RET, VEGFR1/2/3 | Colorectal cancer Gastrointestinal stromal tumors |
| Rituximab | Rituxan | CD20 | Non-Hodgkin’s lymphoma Chronic lymphocytic leukemia Rheumatoid arthritis Granulomatosis with polyangiitis |
| Romidepsin | Istodax | HDAC | Cutaneous T-cell lymphoma Peripheral T-cell lymphoma |
| Ruxolitinib | Jakafi | JAK1/2 | Myelofibrosis |
| Siltuximab | Sylvant | IL-6 | Multicentric Castleman's disease |
| Sipuleucel-T | Provenge | Prostate cancer | |
| Sonidegib | Odomzo | Smoothened | Basal cell carcinoma |
| Sorafenib | Nexavar | VEGFR, PDGFR, KIT, RAF | Hepatocellular carcinoma Renal cell carcinoma Thyroid carcinoma |
| Temsirolimus | Torisel | mTOR | Renal cell carcinoma |
| Tocilizumab | Actemra | IL-6R | Rheumatoid arthritis Juvenile idiopathic arthritis |
| Tofacitinib | Xeljanz | JAK3 | Rheumatoid arthritis |
| Tositumomab | Bexxar** | CD20 | Non-Hodgkin's lymphoma |
| Trametinib | Mekinist | MEK | Melanoma (with BRAF V600 mutation) |
| Trastuzumab | Herceptin | HER2 (ERBB2/neu) | Breast cancer (HER2+) Gastric cancer (HER2+) |
| Vandetanib | Caprelsa | EGFR (HER1/ERBB1), RET, VEGFR2 | Medullary thyroid cancer |
| Vemurafenib | Zelboraf | BRAF | Melanoma (with BRAF V600 mutation) |
| Venetoclax | Venclexta | BCL2 | Chronic lymphocytic leukemia (with 17p deletion) |
| Vismodegib | Erivedge | PTCH, Smoothened | Basal cell carcinoma |
| Vorinostat | Zolinza | HDAC | Cutaneous T-cell lymphoma |
| Ziv-Aflibercept | Zaltrap | PIGF, VEGFA/B | Colorectal cancer |
(* ) More than one drug label may be available with a given active ingredient or commercial drug name
(**) Bexxar is no longer on the market
Table updated November 10, 2016.