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 ingredientDrug nameGene(s) / Molecular Target(s)FDA-approved indication(s)
Ado-Trastuzumab EmtansineKadcylaHER2 (ERBB2/neu)Breast cancer (HER2+)
AfatinibGilotrifEGFR (HER1/ERBB1), HER2 (ERBB2/neu)Non-small cell lung cancer (with EGFR exon 19 deletions or exon 21 substitution (L858R) mutations)
AldesleukinProleukin Renal cell carcinoma
AlectinibAlecensaALKNon-small cell lung cancer (with ALK fusion)
AlemtuzumabCampathCD52B-cell chronic lymphocytic leukemia
AxitinibInlytaKIT, PDGFRβ, VEGFR1/2/3Renal cell carcinoma
BelimumabBenlystaBAFFLupus erythematosus
BelinostatBeleodaqHDACPeripheral T-cell lymphoma
BevacizumabAvastinVEGF ligandCervical cancer
Colorectal cancer
Fallopian tube cancer
Non-small cell lung cancer
Ovarian cancer
Peritoneal cancer
Renal cell carcinoma
BortezomibVelcadeProteasomeMultiple myeloma
Mantle cell lymphoma
BosutinibBosulifABLChronic myelogenous leukemia (Philadelphia chromosome positive)
Brentuximab vedotinAdcetrisCD30Hodgkin lymphoma
Anaplastic large cell lymphoma
CabozantinibCabometyxFLT3, KIT, MET, RET, VEGFR2Medullary thyroid cancer
Renal cell carcinoma
CanakinumabIlarisIL-1βJuvenile idiopathic arthritis
Cryopyrin-associated periodic syndromes
CarfilzomibKyprolisProteasomeMultiple myeloma
CeritinibZykadiaALKNon-small cell lung cancer (with ALK fusion)
CetuximabErbituxEGFR (HER1/ERBB1)Colorectal cancer (KRAS wild type)
Squamous cell cancer of the head and neck
CobimetinibCotellicMEKMelanoma (with BRAF V600E or V600K mutation)
CrizotinibXalkoriALK, MET, ROS1Non-small cell lung cancer (with ALK fusion or ROS1 gene alteration)
DabrafenibTafinlarBRAFMelanoma (with BRAF V600 mutation)
DaratumumabDarzalexCD38Multiple myeloma
DasatinibSprycelABLChronic myelogenous leukemia (Philadelphia chromosome positive)
Acute lymphoblastic leukemia (Philadelphia chromosome positive)
DenosumabXgevaRANKLGiant cell tumor of the bone
DinutuximabUnituxinB4GALNT1 (GD2)Pediatric neuroblastoma
ElotuzumabEmplicitiSLAMF7 (CS1/CD319/CRACC)Multiple myeloma
ErlotinibTarcevaEGFR (HER1/ERBB1)Non-small cell lung cancer
Pancreatic cancer
EverolimusAfinitormTORPancreatic, gastrointestinal, or lung origin neuroendocrine tumor
Renal cell carcinoma
Nonresectable subependymal giant cell astrocytoma associated with tuberous sclerosis
Breast cancer (HR+, HER2-)
GefitinibIressaEGFR (HER1/ERBB1)Non-small cell lung cancer (with EGFR exon 19 deletions or exon 21 substitution (L858R) mutations)
Ibritumomab TiuxetanZevalinCD20Non-Hodgkin's lymphoma
IbrutinibImbruvicaBTKMantle cell lymphoma
Chronic lymphocytic leukemia
Waldenstrom's macroglobulinemia
IdelalisibZydeligPI3KδChronic lymphocytic leukemia
Follicular B-cell non-Hodgkin lymphoma
Small lymphocytic lymphoma
ImatinibGleevecKIT, PDGFR, ABLGI stromal tumor (KIT+)
Dermatofibrosarcoma protuberans
Multiple hematologic malignancies including Philadelphia chromosome-positive ALL and CML
IxazomibNinlaroProteasomeMultiple Myeloma
LapatinibTykerbHER2 (ERBB2/neu), EGFR (HER1/ERBB1)Breast cancer (HER2+)
LenvatinibLenvimaVEGFR2Thyroid cancer
NecitumumabPortrazzaEGFR (HER1/ERBB1)Squamous non-small cell lung cancer
NilotinibTasignaABLChronic myelogenous leukemia (Philadelphia chromosome positive)
Non-small cell lung cancer
Renal cell carcinoma
ObinutuzumabGazyvaCD20Chronic lymphocytic leukemia
Follicular lymphoma
OfatumumabArzerraCD20Chronic lymphocytic leukemia
OlaparibLynparzaPARPOvarian cancer (with BRCA mutation)
OsimertinibTagrissoEGFRNon-small cell lung cancer (with EGFR T790M mutation)
PalbociclibIbranceCDK4, CDK6Breast cancer (ER+, HER2-)
PanitumumabVectibixEGFR (HER1/ERBB1)Colorectal cancer (KRAS wild type)
PanobinostatFarydakHDACMultiple myeloma
PazopanibVotrientVEGFR, PDGFR, KITRenal cell carcinoma
Non-small cell lung cancer (PD-L1+)
PertuzumabPerjetaHER2 (ERBB2/neu)Breast cancer (HER2+)
PonatinibIclusigABL, FGFR1-3, FLT3, VEGFR2Chronic myelogenous leukemia
Acute lymphoblastic leukemia (Philadelphia chromosome positive)
RamucirumabCyramzaVEGFR2Colorectal cancer
Gastric cancer or Gastroesophageal junction (GEJ) adenocarcinoma
Non-small cell lung cancer
RegorafenibStivargaKIT, PDGFRβ, RAF, RET, VEGFR1/2/3Colorectal cancer
Gastrointestinal stromal tumors
RituximabRituxanCD20Non-Hodgkin’s lymphoma
Chronic lymphocytic leukemia
Rheumatoid arthritis
Granulomatosis with polyangiitis
RomidepsinIstodaxHDACCutaneous T-cell lymphoma
Peripheral T-cell lymphoma
SiltuximabSylvantIL-6Multicentric Castleman's disease
Sipuleucel-TProvenge Prostate cancer
SonidegibOdomzoSmoothenedBasal cell carcinoma
SorafenibNexavarVEGFR, PDGFR, KIT, RAFHepatocellular carcinoma
Renal cell carcinoma
Thyroid carcinoma
TemsirolimusToriselmTORRenal cell carcinoma
TocilizumabActemraIL-6RRheumatoid arthritis
Juvenile idiopathic arthritis
TofacitinibXeljanzJAK3Rheumatoid arthritis
TositumomabBexxar**CD20Non-Hodgkin's lymphoma
TrametinibMekinistMEKMelanoma (with BRAF V600 mutation)
TrastuzumabHerceptinHER2 (ERBB2/neu)Breast cancer (HER2+)
Gastric cancer (HER2+)
VandetanibCaprelsaEGFR (HER1/ERBB1), RET, VEGFR2Medullary thyroid cancer
VemurafenibZelborafBRAFMelanoma (with BRAF V600 mutation)
VenetoclaxVenclextaBCL2Chronic lymphocytic leukemia (with 17p deletion)
VismodegibErivedgePTCH, SmoothenedBasal cell carcinoma
VorinostatZolinzaHDACCutaneous T-cell lymphoma
Ziv-AfliberceptZaltrapPIGF, VEGFA/BColorectal 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.