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Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Therapy Description
131I-MIBG Azedra (iobenguane I 131) is a norepinephrine-like moleule labeled with radioactive I 131, which can be used to image or eliminate tumor cells overexpressing norepinephrine transporters (PMID: 25874239). Azedra (iobenguane I 131) is FDA approved for use in adult and pediatric patients 12 years and older with iobenguane scan positive pheochromocytoma or paraganglioma (FDA.gov).
Afatinib Gilotrif (afatinib) is a second-generation pan-Egfr inhibitor with activity against EGFR T790M and EGFR exon 19 and 21 deletions, and also inhibits ERBB2 (HER2), ERBB3 (HER3), ERBB4 (HER4) (PMID: 24435321, PMID: 25505694). Gilotrif (afatinib) is FDA approved for use in non-small cell lung cancer patients harboring non-resistant EGFR mutations, including exon 19 deletions, L858R, S768I, G719X, and L861Q, and for patients with metastatic squamous NSCLC (FDA.gov).
Alclometasone Alclometasone is a topical, synthetic corticosteroid used as a anti-inflammatory and antipruritic agent (FDA.gov).
Apalutamide Erleada (apalutamide) is a second-generation antiandgrogen, that binds to the androgen receptor (AR) and inhibits downstream signaling, potentially resulting in decreased growth of AR-expressing tumors (PMID: 22266222, PMID: 23337756). Erleada (apalutamide) is FDA approved for use in patients with non-metastatic castration-resistant prostate cancer (FDA.gov).
Arsenic trioxide Trisenox (arsenic trioxide) has multiple mechanisms of action, including inhibition of Gli1 and Gli2 transcription factors (PMID: 26891329). Trisenox (arsenic trioxide) is FDA approved for patients with acute promyelocytic leukemia with PML-RARA translocations (FDA.gov).
Atezolizumab Tecentriq (atezolizumab) is a monoclonal antibody that binds to PD-L1 (CD274), preventing activation of its receptor, potentially enhancing T-cell-mediated immune response to neoplasms and inhibiting T-cell inactivation (PMID: 29449897). Tecentriq (atezolizumab) is FDA approved for use in patients with PD-L1-expressing advanced urothelial carcinoma ineligible for cisplatin-containing chemotherapy, advanced urothelial carcinoma ineligible for or progressed on platinum-containing therapy regardless of PD-L1 expression, metastatic non-small cell lung cancer (NSCLC) progressed on platinum-containing therapy, and in combination with bevacizumab, paclitaxel, and carboplatin for non-squamous NSCLC with no EGFR or ALK aberrations (FDA.gov).
Atezolizumab + Bevacizumab + Rucaparib Tecentriq (atezolizumab) is a monoclonal antibody that binds to PD-L1 (CD274), preventing activation of its receptor, potentially enhancing T-cell-mediated immune response to neoplasms and inhibiting T-cell inactivation (PMID: 29449897). Tecentriq (atezolizumab) is FDA approved for use in patients with PD-L1-expressing advanced urothelial carcinoma ineligible for cisplatin-containing chemotherapy, advanced urothelial carcinoma ineligible for or progressed on platinum-containing therapy regardless of PD-L1 expression, and metastatic non-small cell lung cancer progressed on platinum-containing therapy (FDA.gov).
Atezolizumab + Ramucirumab Cyramza (ramucirumab) is a monoclonal antibody, which binds and inhibits VEGFR2 (KDR) resulting in decreased angiogenesis (NCI Drug Dictionary). Cyramza (ramucirumab) is approved for gastric and gastroesophageal junction adenocarcinoma, non-small cell lung cancer, and colorectal cancer (FDA.gov).
Axitinib Inlyta (axitinib) inhibits the VEGFRs, PDGFR, and KIT, potentially resulting in decreased angiogenesis and reduced tumor growth (PMID: 16027439, PMID: 25709499). Inlyta (axitinib) is approved for renal cell carcinoma (FDA.gov).
Azacitidine + GSK3326595 Vidaza (azacitidine) is a cytidine analog that incorporates into DNA and RNA and binds to DNA methyltransferases (DNMTs), resulting in DNMT degradation and decreased DNA methylation, and leading to increased tumor cell death (PMID: 28159832, PMID: 28067760). Vidaza (azacitidine) is FDA-approved for use in patients with some subtypes of myelodysplastic syndrome (FDA.gov).
Belinostat Beleodaq (belinostat) is an HDAC inhibitor, which promotes tumor cell apoptosis, potentially leading to decreased tumor growth (PMID: 12939461, PMID: 16928830, PMID: 17935615). Beleodaq (belinostat) is FDA approved for patients with relapsed or refractory peripheral T-cell lymphoma (FDA.gov).
Bendamustine Treanda (bendamustine) is an alkylating agent with unique mechanisms, resulting in increased DNA damage and base-excision DNA repair pathway induction, and potentially leading to decreased growth of tumors, including those resistant to other alkylating agents (PMID: 18172283, PMID: 19117340, PMID: 19224851). Treanda (bendamustine) is FDA approved for use in chronic lymphocytic leukemia and indolent non-Hodgkin lymphoma (FDA.gov).
Bevacizumab Avastin (bevacizumab) is a monoclonal antibody that binds VEGF and inhibits binding to VEGFR, potentially resulting in decreased tumor growth (PMID: 15136787). Avastin (bevacizumab) is FDA approved for use in colorectal cancer, non-small cell lung cancer, glioblastoma, renal cell carcinoma, cervical carcinoma, and ovarian cancer, and in combination with carboplatin and paclitaxel in epithelial ovarian, fallopian tube, or primary peritoneal cancer (FDA.gov).
Bicalutamide Casodex (bicalutamide) is an androgen-receptor antagonist that binds androgen receptors and inhibits androgen binding, therefore inhibits AR-dependent growth of tumor cells (PMID: 9428389). Casodex (bicalutamide) is approved, in combination with a luteinizing hormone-releasing hormone (LHRH) analog, for metastatic carcinoma of the prostate (FDA.gov).
Blinatumomab + Inotuzumab ozogamicin Blincyto (blinatumomab) is a bispecific antibody that binds both CD19 on B-cells and the CD3 complex on T-cells, therefore facilitates T-cell mediated killing of CD19-expressing tumor cells (PMID: 26337639). Blincyto (blinatumomab) is FDA approved for the treatment of B-cell precursor acute lymphoblastic leukemia in adult and children negative for BCR-ABL1 (FDA.gov).
Bortezomib + Ibrutinib Velcade (bortezomib) inhibits cellular proteasome activity, thereby altering multiple signaling pathways and induces cytotoxicity and apoptosis. Velcade (bortezomib) also inactivates the NF-kB pathway in various cancers (PMID: 20530581). Velcade (bortezomib) is FDA approved for the treatment of relapsed and refractory mantle cell lymphoma and refractory multiple myeloma (FDA.gov).
Bosutinib Bosulif (bosutinib) inhibits SRC and ABL kinases, and has additional activity against other kinases including AXL, TNK2, BTK, resulting in decreased pathway activation and inhibition of tumor cell proliferation (PMID: 12543790, PMID: 19039322, PMID: 26555154). Bosulif (bosutinib) is FDA approved for use in patients with Ph+ (BCR-ABL) chronic myelogenous leukemia (FDA.gov).
Busulfan Busulfex (busulfan) inhibits DNA replication and RNA transcription, likely through induction of DNA alkylation (PMID: 27481448). Busulfex (busulfan) in combination with cyclophosphamide is FDA approved as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for patients with chronic myelogenous leukemia (FDA.gov).
Cabozantinib Cometriq (Cabometyx, cabozantinib) inhibits several receptor tyrosine kinases, including VEGFR2, FLT3, AXL, MET, RET, ROS1 fusions, and c-KIT (PMID: 27370605, PMID: 21926191). Cometriq (cabozantinib) is FDA approved for medullary thyroid cancer and Cabometyx (cabozantinib) is FDA approved for advanced renal cell carcinoma and in sorafenib previously treated hepatocellular carcinoma (FDA.gov).
Celecoxib + Interferon alpha-2b + Rintatolimod Celebra (celecoxib) is a COX-2 inhibitor FDA approved as an anti-inflammatory agent and for colorectal polyp reduction (FDA.gov). Celebra (celecoxib) also promotes apoptosis of tumor cells (PMID: 17909047).
Ceritinib Zykadia (ceritinib) inhibits ALK, including ALK mutations and fusions, and has additional activity against ROS1 fusions, potentially resulting in decreased tumor cell growth (PMID: 25322323, PMID: 26372962). Zykadia (ceritinib) is FDA approved for ALK-positive metastatic non-small cell lung cancer (FDA.gov).
Cetuximab Erbitux (cetuximab) is a monoclonal antibody directed against EGFR, which inhibits signal transduction and cell proliferation (PMID: 28695301). Erbitux (cetuximab) is FDA approved for use in head and neck squamous cell carcinoma and KRAS wild-type, EGFR positive colorectal cancer (FDA.gov).
Cobimetinib Cotellic (cobimetinib) is an allosteric inhibitor of MEK1/2, resulting in inhibition of extracellular signal-related kinase 2 (ERK2) phosphorylation and activation and decreased tumor cell proliferation (PMID: 22084396, PMID: 27956260). Cotellic (cobimetinib) is FDA approved for melanoma patients with BRAF V600E or V600K, in combination with Zelboraf (vemurafenib) (FDA.gov).
Crizotinib Xalkori (crizotinib), inhibits ALK kinase, ALK fusion proteins, c-Met, ROS1 fusion proteins, and MST1R (RON), resulting in growth inhibition of tumor cells (PMID: 26951079, PMID: 22617245). Xalkori (crizotinib) is FDA approved for use in patients with ALK or ROS1 positive non-small cell lung cancer (FDA.gov).
Cyclophosphamide + Doxorubicin + Paclitaxel + Pembrolizumab Keytruda (pembrolizumab) is an antibody that binds to and inhibits PD-1, resulting in activation of T-cell mediated immune response against tumor cells (PMID: 25977344). Keytruda (pembrolizumab) is approved for use in patients with melanoma, head and neck squamous cell carcinoma, classical Hodgkin Lymphoma, primary mediastinal large B-cell lymphoma, hepatocellular carcinoma, Merkel cell carcinoma, MSI-H or dMMR solid tumors, PD-L1 expressing NSCLC, gastric cancer, GEJ adenocarcinoma, urothelial carcinoma, and cervical cancer, in combination with pemetrexed and platinum in non-squamous NSCLC with no EGFR or ALK mutations, and in combination with carboplatin and paclitaxel/nab-paclitaxel in squamous NSCLC (FDA.gov).
Cyclophosphamide + Nivolumab Cytoxan (cyclophosphamide) is an alkylating agent, which inhibits DNA replication (NCI Drug Dictionary). Cytoxan (cyclophosphamide) is FDA approved in multiple hematological malignancies, breast cancer, neuroblastoma, ovarian cancer, and retinoblastoma (NCI Drug Dictionary).
Dabrafenib Tafinlar (dabrafenib) inhibits the activity of BRAF, including V600E, which results in inhibition of tumor cell proliferation (NCI Drug Dictionary). Tafinlar (dabrafenib) is FDA approved for BRAF V600E positive unresectable or metastatic melanoma, and in combination with Mekinist (trametinib) for BRAF V600E/K-mutant melanoma, BRAF V600E-mutant non-small cell lung cancer, and BRAF V600E-mutant anaplastic thyroid cancer (FDA.gov).
Dacarbazine Deticene (dacarbazine) alkylates and cross-links DNA, which disrupts the cell-cycle (NCI Drug Dictionary). Dacarbazine is FDA approved for melanoma (FDA.gov).
Daratumumab Darzalex (Daratumumab) is an antibody against CD38 that is FDA approved for multiple myeloma patients as a monotherapy and in combination with either Revlimid (lenalidomide) and Adexone (dexamethasone) or Velcade (bortezomib) and Adexone (dexamethasone) (FDA.gov).
Dasatinib Sprycel (dasatinib) is an inhibitor of the SRC-family of protein kinases, BCR-ABL, and ABL, and has additional activity against other kinases including KIT, DDR1/2, PDGFRA/B, and EPHA2, which prevents cell growth (PMID: 25709401, PMID: 25284748, PMID: 18797457). Sprycel (dasatinib) is FDA approved for both Philadelphia chromosome positive adult and pediatric chronic myelogenous leukemia and adult acute lymphoblastic leukemia (FDA.gov).
Daunorubicin Cerubidine (daunorubicin) is an anthracycline, which inhibits DNA replication and repair (NCI Drug Dictionary). Cerubidine (daunorubicin) is FDA approved for AML and MLL (FDA.gov).
Daunoxome Daunoxome is a liposomal formulation of the anthracyline, daunorubicin (NCI Drug Dictionary) and is FDA approved for HIV-associated Kaposi’s sarcoma (FDA.gov).
Decitabine + Quizartinib Dacogen (decitabine) is cytidine analog that incorporates into DNA and forms covalent bonds with DNA methyltransferases (DNMTs), resulting in decreased DNMT activity and hypomethylation, and potentially leading to reduced tumor growth (PMID: 28159832, PMID: 25130173). Dacogen (decitabine) is FDA approved for use in patients with myelodysplastic syndromes (FDA.gov).
Doxil Doxil (pegylated liposomal doxorubicin) is doxorubicin encapsulated within a liposome to allow better penetration into tumors, decrease toxicity, and increase the period of effectiveness. Doxorubicin intercalates into DNA, disrupting DNA replication (NCI Drug Dictionary). Doxil is FDA approved for ovarian cancer, AIDS-related Kaposi's sarcoma and multiple myeloma (FDA.gov).
Doxorubicin Adriamycin (doxorubicin) is an anthracycline chemotherapeutic, in a non-liposomal formulation, which intercalates into DNA and inhibits topoisomerase II (PMID: 24367159). Doxorubicin is FDA approved for multiple cancer types (FDA.gov).
Durvalumab + IPH2201 Imfinzi (durvalumab) is a monoclonal antibody that binds to and inhibits PD-L1 (CD274), potentially resulting in increased immune response to tumors (PMID: 25943534, 28214651). Imfinzi (durvalumab) is FDA approved for use in patients with urothelial carcinoma and unresectable, stage III non-small cell lung cancer (FDA.gov).
epirubicin Ellence (epirubicin) is an anthracycline, which intercalates into DNA and inhibits topoisomerase II (PMID: 24367159). Ellence (epirubicin) is FDA approved as a component of adjuvant therapy in patients with evidence of axillary node tumor involvement for primary breast cancer (FDA.gov).
Erlotinib Tarceva (erlotinib) is a first-generation EGFR inhibitor, which is FDA approved for non-small cell lung carcinoma patients with EGFR exon 19 deletions and/or EGFR L858R and in combination with gemcitabine for patients with advanced pancreatic cancer (FDA.gov; PMID: 25302162).
Etoposide + Gemtuzumab ozogamicin + Mitoxantrone Mylotarg (gemtuzumab ozogamicin) is an antibody-drug conjugate comprising an anti-CD33 antibody linked to the cytotoxic agent calicheamicin, which results in DNA damage in CD33-expressing tumor cells (PMID: 28607471). Mylotarg (gemtuzumab ozogamicin) is FDA approved for CD33-positive acute myeloid leukemia (FDA.gov).
Everolimus Afinitor (everolimus) binds to FKBP-12 and allosterically inhibits mTOR, leading to decreased mTORC1 signaling and potentially resulting in decreased tumor cell growth (PMID: 17766661, PMID: 28400999). Afinitor (everolimus) is FDA approved for use in neuroendocrine tumors of pancreatic, lung or gastrointestinal tract origin, advanced renal cell carcinoma, and in combination with Aromasin (exemestane) in hormone receptor-positive, HER2-negative breast cancer (FDA.gov).
Fingolimod Gilenya (fingolimod) is a sphingosine 1-phosphate receptor modulator that activates PP2A, which may result in restoration of tumor suppressor activity thereby leading to apoptosis and inhibition of cell proliferation (PMID: 24448818). Gilenya (fingolimod) is FDA approved for multiple sclerosis (FDA.gov).
GDC-0084 + Trastuzumab Herceptin (trastuzumab) is a monoclonal antibody, which binds ERBB2 (HER2) to induce tumor cellular cytotoxicity (PMID: 17611206). Herceptin (trastuzumab) is FDA approved for HER2-overexpressing breast cancer, gastric adenocarcinoma, and gastroesophageal junction adenocarcinoma (FDA.gov).
Gefitinib Iressa (gefitinib) is a first-generation EGFR inhibitor that binds EGFR and inhibits downstream signaling, potentially resulting in decreased growth of tumors with EGFR activation (PMID: 26980062, PMID: 25302162, PMID: 15284455). Iressa (gefitinib) is FDA approved for use in patients with non-small cell lung cancer harboring an EGFR exon 19 deletion or EGFR L858R (FDA.gov).
Gilteritinib + Venetoclax Venclexta (venetoclax) is a BH3-mimetic that binds to and inhibits BCL2, resulting in increased tumor cell apoptosis (PMID: 26589495, PMID: 25048785). Venclexta (venetoclax) is FDA approved for use in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, and in combination with chemotherapy in patients 75 years old or older with newly-diagnosed acute myeloid leukemia (FDA.gov).
Hydroxychloroquine + Trametinib Mekinist (trametinib) inhibits MEK 1 and 2, which potentially leads to reduced tumor cell proliferation (PMID: 27956260). Mekinist (trametinib) is FDA approved for melanoma patients harboring BRAF V600E or BRAF V600K mutations, and in combination with Tafinlar (dabrafinib) for BRAF V600E/K-mutant melanoma, BRAF V600E- mutant non-small cell lung cancer, and BRAF V600E-mutant anaplastic thyroid cancer (FDA.gov).
Idarubicin Idarubicin is an anthracycline, which inhibits DNA replication thereby preventing RNA and protein synthesis (NCI Drug Dictionary). Idarubicin, in combination with other approved drugs, is FDA approved for acute myelogenous leukemia (FDA.gov).
Imatinib Gleevec (imatinib mesylate) is a multi-target inhibitor of c-Kit, Pdgfr, and Bcr-Abl (NCI Drug Dictionary). Gleevec (imatinib mesylate) is FDA approved for c-KIT positive GIST and dermatofibrosarcoma protuberans, Philadelphia chromosome positive chronic myeloid leukemia, and FIP1L1-PDGFRA positive chronic eosinophilic leukemia (FDA.gov).
Lapatinib Tykerb (lapatinib) reversibly inhibits ERBB2 (HER2) and EGFR, resulting in decreased downstream signaling and potentially leading to reduced tumor growth (PMID: 22477724). Tykerb (lapatinib) is FDA approved for ERBB2 (HER2)-positive breast cancer (FDA.gov).
Letrozole Femara (letrozole) is an aromatase inhibitor, which inhibits estrogen synthesis (NCI Drug Dictionary). Femara (letrozole) is approved for postmenopausal women with hormone receptor positive breast cancer (FDA.gov).
lutetium Lu 177 dotatate Lutathera (lutetium Lu 177 dotatate) is a radiolabeled somatostatin analog which may selectively kill somatostatin receptor-positive tumor cells (PMID: 28076709). Lutathera (lutetium Lu 177 dotatate) is FDA approved for use in patients with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (FDA.gov).
Melphalan Alkeran (melphalan) is an antineoplastic alkylating agent, which cross-links DNA and induces cell toxicity (NCI Drug Dictionary). Alkeran (melphalan) is FDA approved for multiple myeloma and epithelial ovarian carcinoma (FDA.gov).
Moxetumomab pasudotox-tdfk Lumoxiti (moxetumomab pasudotox-tdfk) is an immunotoxin targeting CD22-positive tumor cells (PMID: 30030507). Lumoxiti (moxetumomab pasudotox-tdfk) is FDA approved for use in patients with relapsed or refractory hairy cell leukemia (FDA.gov).
Nilotinib Tasigna (nilotinib) inhibits several tyrosine kinases including BCR-ABL, PDGFR, KIT, DDR and CSF-1R, potentially resulting in reduced tumor cell proliferation (PMID: 21419934, PMID: 25284748). Tasigna (nilotinib) is FDA approved for use in patients with Ph+ chronic myeloid leukemia (FDA.gov).
Obinutuzumab + Vemurafenib Gazyva (obinutuzumab) is a glycoengineered anti-CD20 antibody that binds to CD20 on B-cells, resulting in increased antibody-dependent cellular cytotoxicity and induction of apoptosis, and potentially leading to decreased growth of B-cell tumors (PMID: 20194898, PMID: 21220500). Gazyva (obinutuzumab) is FDA approved in combination with Ambochlorin (chlorambucil) for chronic lymphocytic leukemia and in combination with Treanda (bendamustine) or chemotherapy for follicular lymphoma (FDA.gov).
Ofatumumab Arzerra (ofatumumab) is a monoclonal antibody that binds to CD20 and induces cytotoxic immune response against CD20 over-expressing cells (PMID: 26855591). Arzerra (ofatumumab) is FDA approved for chronic lymphocytic leukemia (FDA.gov).
Palbociclib Ibrance (palbociclib) is a selective inhibitor of cyclin-dependent kinase 4 (CDK4) and 6 (CDK6) (PMID: 19874578). Ibrance (palbociclib) is approved in combination with an aromatase inhibitor in postmenopausal patients with ER-positive, ERBB2 (HER2)-negative metastatic breast cancer, and in combination with Faslodex (fulvestrant) in patients with ER-positive, ERBB2 (HER2)-negative metastatic breast cancer (FDA.gov).
Panitumumab Vectibix (panitumumab) is a monoclonal antibody directed against EGFR, which inhibits cell proliferation and induces apoptosis (PMID: 18998757). Vectibix (panitumumab) is FDA approved for metastatic colorectal cancer patients with wild-type KRAS and NRAS (FDA.gov).
Panobinostat Farydak (panobinostat) is an HDAC inhibitor, which induces cell-cycle arrest and decreases growth of tumor cells (PMID: 18349321, PMID: 19671764, PMID: 27802904). Farydak (panobinostat) is FDA approved, in combination with Velcade (Bortezomib) and dexamethasone, for multiple myeloma (FDA.gov).
Pazopanib Votrient (pazopanib) inhibits VEGFR-1, -2 and -3, KIT, and PDGFR, and FGFR1, 2, and 3, leading to decreased tumor angiogenesis and growth (PMID: 17620431, PMID: 23786770). Votrient (pazopanib) is approved for renal cell carcinoma and soft tissue sarcoma (FDA.gov).
Pembrolizumab Keytruda (pembrolizumab) is an antibody that binds to and inhibits PD-1, resulting in activation of T-cell mediated immune response against tumor cells (PMID: 25977344). Keytruda (pembrolizumab) is approved for use in patients with melanoma, head and neck squamous cell carcinoma, classical Hodgkin Lymphoma, primary mediastinal large B-cell lymphoma, urothelial carcinoma, hepatocellular carcinoma, Merkel cell carcinoma, MSI-H or dMMR solid tumors, PD-L1 expressing NSCLC, gastric cancer, GEJ adenocarcinoma, and cervical cancer, in combination with pemetrexed and platinum in non-squamous NSCLC with no EGFR or ALK mutations, and in combination with carboplatin and paclitaxel/nab-paclitaxel in squamous NSCLC (FDA.gov).
Pertuzumab Perjeta (pertuzumab) is a monoclonal antibody that binds ERBB2 (HER2) to prevent dimerization and subsequent HER signaling, thereby resulting in apoptosis and tumor growth inhibition (PMID: 15699478). Perjeta (pertuzumab) is FDA approved in combination with Herceptin (trastuzumab) and Taxotere (docetaxel) for patients with ERBB2 (HER2)-positive breast cancer and in combination with Herceptin (trastuzumab) and chemotherapy as neoadjuvant treatment for patients with ERBB2 (HER2)-positive breast cancer (FDA.gov).
Ponatinib Iclusig (ponatinib) inhibits the Bcr-Abl fusion, and other tyrosine kinases, such as RET, DDR, VEGFR, FGFR, KIT, and FLT3 (PMID: 23526464, PMID: 25284748, PMID: 19878872). Iclusig (ponatinib) is FDA approved for chronic myeloid leukemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukemia (Ph positive ALL) as well as ABL1 T315I CML and ABL1 T315I Ph positive ALL (FDA.gov).
Ramucirumab Cyramza (ramucirumab) is a monoclonal antibody, which binds and inhibits VEGFR2 (KDR) resulting in decreased angiogenesis (NCI Drug Dictionary). Cyramza (ramucirumab) is approved for gastric and gastroesophageal junction adenocarcinoma, non-small cell lung cancer, and colorectal cancer (FDA.gov).
Ramucirumab + Paclitaxel Cyramza (ramucirumab) in combination with paclitaxel is FDA approved for the treatment of patients with advanced gastric or gastroesophageal junction (GEJ) adenocarcinoma (FDA.gov).
Regorafenib Stivarga (regorafenib) is an inhibitor of several kinases including VEGFR, RET, KIT, PDGFR and RAF, thereby inhibiting tumor angiogenesis and tumor cell proliferation (PMID: 24756792). Stivarga (regorafenib) is FDA approved for colorectal cancer, hepatocellular carcinoma, and GIST (FDA.gov).
romidepsin Istodax (romidepsin) inhibits Class I HDACs, resulting in cell cycle arrest, apoptosis, and decreased tumor cell growth (PMID: 12208741, PMID: 12234611, PMID: 21258344). Istodax (romidepsin) is FDA approved for patients with cutaneous T-cell lymphoma (FDA.gov).
Ruxolitinib Jakafi (ruxolitinib) is an inhibitor of protein tyrosine kinases JAK1 and JAK2, thus resulting in reduced inflammation and reduced proliferation (NCI Drug Dictionary). Jakafi (ruxolitinib) is FDA approved to treat bone marrow cancer, specifically intermediate or high-risk myelofibrosis (FDA.gov)
siltuximab Sylvant (siltuxmab) is an anti-IL-6 chimeric monoclonal antibody, which inhibits IL6 signaling and may lead to decreased tumor cell growth (PMID: 26170629, PMID: 26744529). Sylvant (siltuximab) is FDA approved for patients with multicentric Castleman's disease who do not have HIV or HHV-8 (FDA.gov).
Sorafenib Nexavar (sorafenib) is a multikinase inhibitor with activity against several kinases, including RAF kinases, VEGFR2, VEGFR3, PDGFR-beta, KIT, FLT3, RET, and CSF1R, potentially resulting in decreased tumor growth (PMID: 18445656, PMID: 15466206, PMID: 21517818). Nexavar (sorafenib) is approved for metastatic differentiated thyroid carcinoma, hepatocellular carcinoma, and renal cell carcinoma (FDA.gov).
Sulindac Clinoril (sulindac) is a non-selective COX1-2 inhibitor, FDA approved as an anti-inflammatory agent (FDA.gov). Clinoril (sulindac) also reduces intestinal tumor burden (PMID: 19755659).
Sunitinib Sutent (sunitinib) inhibits KDR (VEGFR2), PDGFR, c-KIT, FLT3, RET, and CSF1R, thereby inhibiting angiogenesis and cell proliferation (NCI Drug Dictionary; PMID: 25085632). Sutent (sunitinib) is approved for neuroendocrine tumors of the pancreas, advanced renal cell carcinoma, and GIST (FDA.gov).
Tagraxofusp-erzs Elzonris (tagraxofusp-erzs) is a fusion protein consists of human interleukin 3 (IL3) and the first 388 amino acids of diphtheria toxin [DT(388)], which binds to IL3 receptors and subsequently inhibits protein synthesis, leading to cell death (PMID: 28479592). Elzonris (tagraxofusp-erzs) is FDA approved for use in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (FDA.gov).
Temsirolimus Torisel (temsirolimus) binds to FKBP-12 and allosterically inhibits mTOR (PMID: 16883305). Torisel (temsirolimus) is FDA approved for advanced renal cell carcinoma (FDA.gov).
Tofacitinib Xeljanz (tofacitinib) is a pan-JAK inhibitor, which decreases downstream signaling and potentially leads to reduced tumor cell growth (PMID: 26300391, PMID: 21106989). Xeljanz (tofacitinib) is FDA approved for rheumatoid arthritis (FDA.gov).
trastuzumab emtansine Kadcyla (trastuzumab emtansine) is an antibody drug conjugate combined of the ERBB2 (HER2) monoclonal antibody, trastuzumab, and an anti-tubulin agent, DM1, which results in tumor cell cytotoxicity (PMID: 24887180). Kadcyla (trastuzumab emtansine) is FDA approved for metastatic ERBB2 (HER2)-positive (overexpression or gene amplification) breast cancer patients who have previously received trastuzumab and/or taxane (FDA.gov).
Tretinoin Tretinoin is a form of retinoic acid, which binds to retinoic acid receptors and activates downstream signaling, potentially inducing cell differentiation and subsequently, apoptosis (PMID: 11704842). Tretinoin is FDA approved for patients with acute promyelocytic leukemia harboring PML-RARA (FDA.gov).
Valproic acid Valproic acid inhibits HDAC activity, potentially resulting in decreased angiogenesis, increased tumor cell differentiation, and reduced tumor growth (PMID: 11742974, PMID: 14978230). Valproic acid is FDA-approved for use in treating seizures (FDA.gov).
Valstar Valstar (valrubicin) is an anthracycline that is metabolized into compounds that inhibit topoisomerase II, resulting in inhibition of DNA synthesis and potentially leading to decreased tumor growth (PMID: 19145271, PMID: 20554745). Valstar (valrubicin) is FDA-approved for treatment of BCG-refractory urinary bladder carcinoma in situ (FDA.gov).
Vandetanib Caprelsa (vandetanib) is a multi-tyrosine kinase inhibitor with activity against VEGFR, EGFR, RET, SRC, and ABL1 kinases, potentially resulting in decreased tumor growth (PMID: 25031937, PMID: 25490448). Caprelsa (vandetanib) is FDA approved for use in medullary thyroid cancer (FDA.gov).
Vemurafenib Zelboraf (vemurafenib) inhibits BRAF V600E, wild-type BRAF, ARAF, and CRAF (PMID: 20179705), which may result in an inhibition of the MAPK signaling pathway resulting in a reduction of tumor cell proliferation (PMID: 20823850). Zelboraf (vemurafenib) is FDA approved for BRAF V600E-mutant melanoma and for BRAF V600-positive Erdheim-Chester disease (FDA.gov).
Vinorelbine Navelbine (vinorelbine) is a tubulin binding alkaloid that inhibits tubulin polymerization, thus inhibiting spindle formation, leading to apoptosis (NCI Drug Dictionary). Navelbine (vinorelbine) is approved for use in NSCLC (FDA.gov).
Vismodegib Erivedge (vismodegib) binds to and inhibits Smoothened (SMO), resulting in inhibition of downstream signaling and decreased tumor growth (PMID: 24259609, PMID: 19716296). Erivedge (vismodegib) is FDA approved for use in patients with basal cell carcinoma (FDA.gov).
Vorinostat Zolinza (vorinostat) inhibits HDAC activity, resulting in cell-cycle arrest, decreased proliferation, and increased differentiation of tumor cells (PMID: 9501205, PMID: 11731433, PMID: 11016644). Zolinza (vorinostat) is FDA approved for use in cutaneous T-cell lymphoma (FDA.gov).
Drug Name Trade Name Synonyms Drug Classes Drug Description
131I-MIBG Azedra I131 Metaiodobenzylguanidine iodine|iobenguane I 131 Azedra (iobenguane I 131) is a norepinephrine-like moleule labeled with radioactive I 131, which can be used to image or eliminate tumor cells overexpressing norepinephrine transporters (PMID: 25874239). Azedra (iobenguane I 131) is FDA approved for use in adult and pediatric patients 12 years and older with iobenguane scan positive pheochromocytoma or paraganglioma (FDA.gov).
Abemaciclib Verzenio LY2835219 CDK4/6 Inhibitor 8 Verzenio (abemaciclib) is a dual CDK4/6 inhibitor, which inhibits Rb1 protein phosphorylation and may induce cell cycle arrest and prevent growth in cancer cells (PMID: 24919854). Verzenio (abemaciclib) is FDA approved as a monotherapy and in combination with Faslodex (fulvestrant) in patients with hormone receptor-positive (ESR, PGR) and ERBB2 (HER2) receptor-negative advanced or metastatic breast cancer (FDA.gov).
Ado-trastuzumab emtansine Kadcyla T-DM1|trastuzumab emtansine HER2 (ERBB2) Antibody 25 Kadcyla (trastuzumab emtansine) is an antibody drug conjugate combined of the ERBB2 (HER2) monoclonal antibody, trastuzumab, and an anti-tubulin agent, DM1, which results in tumor cell cytotoxicity (PMID: 24887180). Kadcyla (trastuzumab emtansine) is FDA approved for metastatic ERBB2 (HER2)-positive (overexpression or gene amplification) breast cancer patients who have previously received trastuzumab and/or taxane (FDA.gov).
Afatinib Gilotrif BIBW 2992 EGFR Inhibitor (Pan) 38 EGFR Inhibitor 2nd gen 3 HER inhibitor (Pan) 6 HER3 Inhibitor 2 Gilotrif (afatinib) is a second-generation pan-Egfr inhibitor with activity against EGFR T790M and EGFR exon 19 and 21 deletions, and also inhibits ERBB2 (HER2), ERBB3 (HER3), ERBB4 (HER4) (PMID: 24435321, PMID: 25505694). Gilotrif (afatinib) is FDA approved for use in non-small cell lung cancer patients harboring non-resistant EGFR mutations, including exon 19 deletions, L858R, S768I, G719X, and L861Q, and for patients with metastatic squamous NSCLC (FDA.gov).
Aflibercept Zaltrap AVE 0005|Eylea VEGF Antibody 10 VEGFR Inhibitor (Pan) 29 Zaltrap (aflibercept) is comprised of human IgG fused to the extracellular domains of VEGFR1 and VEGFR2, which binds VEGF to inhibit tumor angiogenesis and metastasis (NCI Drug Dictionary). Zaltrap (aflibercept) is FDA approved in patients with metastatic colorectal cancer (FDA.gov).
Alectinib Alecensa CH5424802|RO5424802 ALK Inhibitor 19 RET Inhibitor 36 Alecensa (alectinib) is an inhibitor of RET and ALK, including ALK fusions and the gatekeeper mutation, L1196M (PMID: 21575866, PMID: 25349307). Alecensa (alectinib) is FDA-approved for use in patients with ALK-positive non-small cell lung cancer (FDA.gov).
Apalutamide Erleada ARN-509|JNJ-56021927 Hormone - Anti-androgens 22 Erleada (apalutamide) is a second-generation antiandgrogen, that binds to the androgen receptor (AR) and inhibits downstream signaling, potentially resulting in decreased growth of AR-expressing tumors (PMID: 22266222, PMID: 23337756). Erleada (apalutamide) is FDA approved for use in patients with non-metastatic castration-resistant prostate cancer (FDA.gov).
Arsenic trioxide Trisenox ATO GLI1/2 inhibitor 6 Trisenox (arsenic trioxide) has multiple mechanisms of action, including inhibition of Gli1 and Gli2 transcription factors (PMID: 26891329). Trisenox (arsenic trioxide) is FDA approved for patients with acute promyelocytic leukemia with PML-RARA translocations (FDA.gov).
Asparaginase Erwinia chrysanthemi Erwinaze crisantaspase Erwinaze (asparaginase Erwinia chrysanthemi) is an bacterial derived enzyme that converts L-asparagine to L-aspartate and ammonia, which decreases L-asparagine levels, potentially leading to reduced leukemic cell growth (PMID: 25098829, PMID: 26698391). Erwinaze (asparaginase Erwinia chrysanthemi) is FDA-approved for use in a chemotherapeutic regimen for acute lymphoblastic leukemia (FDA.gov).
Atezolizumab Tecentriq RG7446|MPDL3280A Immune Checkpoint Inhibitor 99 PD-L1/PD-1 antibody 48 Tecentriq (atezolizumab) is a monoclonal antibody that binds to PD-L1 (CD274), preventing activation of its receptor, potentially enhancing T-cell-mediated immune response to neoplasms and inhibiting T-cell inactivation (PMID: 29449897). Tecentriq (atezolizumab) is FDA approved for use in patients with PD-L1-expressing advanced urothelial carcinoma ineligible for cisplatin-containing chemotherapy, advanced urothelial carcinoma ineligible for or progressed on platinum-containing therapy regardless of PD-L1 expression, metastatic non-small cell lung cancer (NSCLC) progressed on platinum-containing therapy, and in combination with bevacizumab, paclitaxel, and carboplatin for non-squamous NSCLC with no EGFR or ALK aberrations (FDA.gov).
Avelumab Bavencio MSB0010718C Immune Checkpoint Inhibitor 99 PD-L1/PD-1 antibody 48 Bavencio (avelumab) is a monoclonal antibody binds to human immunosuppressive ligand programmed death-ligand 1 (PD-L1, CD274) and blocks protein signaling, resulting in immune regulation and antitumor immunity (PMID: 26014098). Bavencio (avelumab) is FDA-approved for use in patients with metastatic Merkel cell carcinoma and urothelial carcinoma (FDA.gov).
Axitinib Inlyta AG-013736 KIT Inhibitor 48 PDGFR Inhibitor (Pan) 27 VEGFR Inhibitor (Pan) 29 Inlyta (axitinib) inhibits the VEGFRs, PDGFR, and KIT, potentially resulting in decreased angiogenesis and reduced tumor growth (PMID: 16027439, PMID: 25709499). Inlyta (axitinib) is approved for renal cell carcinoma (FDA.gov).
Azacitidine Vidaza azacytidine|CC-486|5-azacytidine|5-AC|U-18496 DNMT inhibitor (Pan) 5 Vidaza (azacitidine) is a cytidine analog that incorporates into DNA and RNA and binds to DNA methyltransferases (DNMTs), resulting in DNMT degradation and decreased DNA methylation, and leading to increased tumor cell death (PMID: 28159832, PMID: 28067760). Vidaza (azacitidine) is FDA-approved for use in patients with some subtypes of myelodysplastic syndrome (FDA.gov).
BCG solution TICE BCG solution Bacillus Calmette-Guerin solution TICE BCG solution (BCG solution) is an attenuated live culture of Bacillus Calmette Guerin that induces anti-tumor immunity in bladder cancer (PMID: 26000263). TICE BCG solution (BCG solution) is FDA approved for carcinoma in situ of the bladder (FDA.gov).
Balsalazide Carbadox Balsalazida [Spanish] Carbadox (balsalazide) is a 5-aminosalicylate (5-ASA) type drug that is FDA approved for the treatment of inflammation resulting from ulcerative colitis (FDA.gov), however, has also been demonstrated to induce apoptosis and decrease cell proliferation in cancer cells (PMID: 28108625).
Belinostat Beleodaq PDX101|NSC726630|PX-105684 HDAC Inhibitor 34 Beleodaq (belinostat) is an HDAC inhibitor, which promotes tumor cell apoptosis, potentially leading to decreased tumor growth (PMID: 12939461, PMID: 16928830, PMID: 17935615). Beleodaq (belinostat) is FDA approved for patients with relapsed or refractory peripheral T-cell lymphoma (FDA.gov).
Bendamustine Treanda Ribomustin|SyB L-0501|SDX-105 Chemotherapy - Alkylating 14 Treanda (bendamustine) is an alkylating agent with unique mechanisms, resulting in increased DNA damage and base-excision DNA repair pathway induction, and potentially leading to decreased growth of tumors, including those resistant to other alkylating agents (PMID: 18172283, PMID: 19117340, PMID: 19224851). Treanda (bendamustine) is FDA approved for use in chronic lymphocytic leukemia and indolent non-Hodgkin lymphoma (FDA.gov).
Bevacizumab Avastin VEGF Antibody 10 VEGFR Inhibitor (Pan) 29 Avastin (bevacizumab) is a monoclonal antibody that binds VEGF and inhibits binding to VEGFR, potentially resulting in decreased tumor growth (PMID: 15136787). Avastin (bevacizumab) is FDA approved for use in colorectal cancer, non-small cell lung cancer, glioblastoma, renal cell carcinoma, cervical carcinoma, and ovarian cancer, and in combination with carboplatin and paclitaxel in epithelial ovarian, fallopian tube, or primary peritoneal cancer (FDA.gov).
Bevacizumab-awwb Mvasi ABP 215 VEGF Antibody 10 Mvasi (bevacizumab-awwb) is a biosimilar to the anti-VEGF antibody Avastin (bevacizumab), which binds to and inhibits VEGF, resulting in decreased tumor growth and angiogenesis (Ann Oncol (2014) 25 (suppl 4): iv163; Abstract 489P). Mvasi (bevacizumab-awwb) is FDA approved for patients with colorectal cancer, non-small cell lung carcinoma, glioblastoma, renal cell carcinoma, and cervical cancer (FDA.gov).
Bicalutamide Casodex Hormone - Anti-androgens 22 Casodex (bicalutamide) is an androgen-receptor antagonist that binds androgen receptors and inhibits androgen binding, therefore inhibits AR-dependent growth of tumor cells (PMID: 9428389). Casodex (bicalutamide) is approved, in combination with a luteinizing hormone-releasing hormone (LHRH) analog, for metastatic carcinoma of the prostate (FDA.gov).
Binimetinib Mektovi ARRY-162|ARRY-438162|MEK162 MEK inhibitor (Pan) 20 MEK1 Inhibitor 20 MEK2 Inhibitor 18 Mektovi (binimetinib) inhibits MEK1 and MEK2 resulting in inhibition of growth factor-mediated signaling and decreased tumor cell proliferation (PMID: 23587417). Mektovi (binimetinib) in combination with Braftovi (encorafenib) is FDA approved for use in patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation (FDA.gov).
Blinatumomab Blincyto anti-CD19/anti-CD3 recombinant bispecific|monoclonal antibody MT103|MEDI-538 CD19 Antibody 7 Blincyto (blinatumomab) is a bispecific antibody that binds both CD19 on B-cells and the CD3 complex on T-cells, therefore facilitates T-cell mediated killing of CD19-expressing tumor cells (PMID: 26337639). Blincyto (blinatumomab) is FDA approved for the treatment of B-cell precursor acute lymphoblastic leukemia in adult and children negative for BCR-ABL1 (FDA.gov).
Bortezomib Velcade Velcade (bortezomib) inhibits cellular proteasome activity, thereby altering multiple signaling pathways and induces cytotoxicity and apoptosis. Velcade (bortezomib) also inactivates the NF-kB pathway in various cancers (PMID: 20530581). Velcade (bortezomib) is FDA approved for the treatment of relapsed and refractory mantle cell lymphoma and refractory multiple myeloma (FDA.gov).
Bosutinib Bosulif SKI-606 ABL Inhibitor (pan) 8 AXL Inhibitor 24 BTK inhibitor 20 SRC Inhibitor 26 TNK2 Inhibitor 6 Bosulif (bosutinib) inhibits SRC and ABL kinases, and has additional activity against other kinases including AXL, TNK2, BTK, resulting in decreased pathway activation and inhibition of tumor cell proliferation (PMID: 12543790, PMID: 19039322, PMID: 26555154). Bosulif (bosutinib) is FDA approved for use in patients with Ph+ (BCR-ABL) chronic myelogenous leukemia (FDA.gov).
Brentuximab vedotin Adcetris SGN-35 Adcetris (brentuximab vedotin) is an antibody-drug conjugate comprised of a monoclonal antibody targeting CD30 linked to the tubulin-binding drug MMAE, which binds CD30-expressing cells and disrupts microtubule polymerization, potentially resulting in decreased tumor growth (PMID: 22684302). Adcetris (brentuximab vedotin) is FDA approved for Hodgkin lymphoma, anaplastic large cell lymphoma, CD30-expressing mycosis fungoides, and in combination with chemotherapy in other CD30-expressing peripheral T-cell lymphomas (FDA.gov).
Brigatinib Alunbrig AP26113 ALK Inhibitor 19 EGFR Inhibitor (Pan) 38 FLT3 Inhibitor 48 IGF-1R Inhibitor 17 ROS1 Inhibitor 14 Alunbrig (brigatinib) is a multi-kinase inhibitor with selected activity against ALK, ROS1 fusions, FLT3, IGF1R and mutant EGFR, potentially resulting in decreased tumor growth (PMID: 27780853). Alunbrig (brigatinib) is FDA approved for use in patients with ALK-positive non-small cell lung cancer who have progressed on or are intolerant to crizotinib (FDA.gov).
Busulfan Busulfex Busulphan Busulfex (busulfan) inhibits DNA replication and RNA transcription, likely through induction of DNA alkylation (PMID: 27481448). Busulfex (busulfan) in combination with cyclophosphamide is FDA approved as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for patients with chronic myelogenous leukemia (FDA.gov).
CPX-351 Vyxeos Vyxeos (CPX-351) is a liposomal formulation of cytarabine and daunorubicin with anti-tumor activities (PMID: 25223583). Vyxeos (CPX-351) is FDA approved for use in patients with newly-diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes (FDA.gov).
Cabozantinib Cometriq Cabometyx|Cabozantinib-s-malate|XL184 AXL Inhibitor 24 FLT3 Inhibitor 48 KIT Inhibitor 48 MET Inhibitor 50 RET Inhibitor 36 ROS1 Inhibitor 14 VEGFR2 Inhibitor 34 Cometriq (Cabometyx, cabozantinib) inhibits several receptor tyrosine kinases, including VEGFR2, FLT3, AXL, MET, RET, ROS1 fusions, and c-KIT (PMID: 27370605, PMID: 21926191). Cometriq (cabozantinib) is FDA approved for medullary thyroid cancer and Cabometyx (cabozantinib) is FDA approved for advanced renal cell carcinoma and in sorafenib previously treated hepatocellular carcinoma (FDA.gov).
Carfilzomib Kyprolis PR-171 Kyprolis (carfilzomib) binds to the 20S proteasome and inhibits its protein degradation activity, thereby leading to the accumulation of ubiquinated proteins, which may result in apoptosis and growth arrest in tumor cells (NCI Drug Dictionary). Kyprolis (carfilzomib), in combination with lenalidomide and dexamethasone, is FDA approved for multiple myeloma (FDA.gov).
Celecoxib Celebra Celebra (celecoxib) is a COX-2 inhibitor FDA approved as an anti-inflammatory agent and for colorectal polyp reduction (FDA.gov). Celebra (celecoxib) also promotes apoptosis of tumor cells (PMID: 17909047).
Cemiplimab Libtayo REGN2810|SAR439684 Immune Checkpoint Inhibitor 99 PD-L1/PD-1 antibody 48 Libtayo (cemiplimab) is a human antibody that targets PD-1 (PDCD1) and prevents interaction with PD-L1 (CD274) and PD-L2 (PDCD1LG2), potentially resulting in increased anti-tumor immune response and decreased tumor growth (PMID: 28265006). Libtayo (cemiplimab) is FDA approved for use in patients with cutaneous squamous cell carcinoma, who are not not eligible for curative surgery or radiation (FDA.gov).
Ceritinib Zykadia LDK378 ALK Inhibitor 19 ROS1 Inhibitor 14 Zykadia (ceritinib) inhibits ALK, including ALK mutations and fusions, and has additional activity against ROS1 fusions, potentially resulting in decreased tumor cell growth (PMID: 25322323, PMID: 26372962). Zykadia (ceritinib) is FDA approved for ALK-positive metastatic non-small cell lung cancer (FDA.gov).
Cetuximab Erbitux EGFR Antibody 27 Erbitux (cetuximab) is a monoclonal antibody directed against EGFR, which inhibits signal transduction and cell proliferation (PMID: 28695301). Erbitux (cetuximab) is FDA approved for use in head and neck squamous cell carcinoma and KRAS wild-type, EGFR positive colorectal cancer (FDA.gov).
Cobimetinib Cotellic GDC-0973 MEK inhibitor (Pan) 20 MEK1 Inhibitor 20 MEK2 Inhibitor 18 Cotellic (cobimetinib) is an allosteric inhibitor of MEK1/2, resulting in inhibition of extracellular signal-related kinase 2 (ERK2) phosphorylation and activation and decreased tumor cell proliferation (PMID: 22084396, PMID: 27956260). Cotellic (cobimetinib) is FDA approved for melanoma patients with BRAF V600E or V600K, in combination with Zelboraf (vemurafenib) (FDA.gov).
Copanlisib Aliqopa BAY80-6946 PIK3CA inhibitor 14 PIK3CD inhibitor 24 Aliqopa (copanlisib) is a selective PIK3CA and PIK3CD inhibitor that prevents activation of Akt, resulting in inhibition of cell cycle progression and increased apoptosis (PMID: 24013662, PMID: 25528022). Aliqopa (copanlisib) is FDA approved for adult patients with follicular lymphoma (FDA.gov).
Crizotinib Xalkori PF-02341066 ALK Inhibitor 19 MET Inhibitor 50 RON Inhibitor 10 ROS1 Inhibitor 14 Xalkori (crizotinib), inhibits ALK kinase, ALK fusion proteins, c-Met, ROS1 fusion proteins, and MST1R (RON), resulting in growth inhibition of tumor cells (PMID: 26951079, PMID: 22617245). Xalkori (crizotinib) is FDA approved for use in patients with ALK or ROS1 positive non-small cell lung cancer (FDA.gov).
Cyclophosphamide Cytoxan CPM Chemotherapy - Alkylating 14 Cytoxan (cyclophosphamide) is an alkylating agent, which inhibits DNA replication (NCI Drug Dictionary). Cytoxan (cyclophosphamide) is FDA approved in multiple hematological malignancies, breast cancer, neuroblastoma, ovarian cancer, and retinoblastoma (NCI Drug Dictionary).
Dabrafenib Tafinlar GSK2118436 BRAF Inhibitor 18 Tafinlar (dabrafenib) inhibits the activity of BRAF, including V600E, which results in inhibition of tumor cell proliferation (NCI Drug Dictionary). Tafinlar (dabrafenib) is FDA approved for BRAF V600E positive unresectable or metastatic melanoma, and in combination with Mekinist (trametinib) for BRAF V600E/K-mutant melanoma, BRAF V600E-mutant non-small cell lung cancer, and BRAF V600E-mutant anaplastic thyroid cancer (FDA.gov).
Dacarbazine Deticene Biocarbazine R Chemotherapy - Alkylating 14 Deticene (dacarbazine) alkylates and cross-links DNA, which disrupts the cell-cycle (NCI Drug Dictionary). Dacarbazine is FDA approved for melanoma (FDA.gov).
Dacomitinib Vizimpro PF-00299804|PF299804 EGFR Inhibitor 2nd gen 3 HER inhibitor (Pan) 6 Vizimpro (dacomitinib) is a second-generation EGFR inhibitor and pan-HER inhibitor that inhibits HER2 and multiple EGFR subtypes including EGFR T790M, resulting in decreased proliferation and increased apoptosis in EGFR-expressing and HER2-expressing tumor cells (PMID: 22761403, PMID: 18606718). Vizimpro (dacomitinib) is FDA approved for use in patients with metastatic non-small cell lung cancer with EGFR exon 19 deletion or L858R mutation as first-line treatment (FDA.gov).
Daratumumab Darzalex Darzalex (Daratumumab) is an antibody against CD38 that is FDA approved for multiple myeloma patients as a monotherapy and in combination with either Revlimid (lenalidomide) and Adexone (dexamethasone) or Velcade (bortezomib) and Adexone (dexamethasone) (FDA.gov).
Dasatinib Sprycel BMS-354825 ABL Inhibitor (pan) 8 BCR-ABL Inhibitor 23 BTK inhibitor 20 CSF1R Inhibitor 22 DDR1 Inhibitor 8 DDR2 inhibitor 7 KIT Inhibitor 48 PDGFR Inhibitor (Pan) 27 SRC Inhibitor 26 TNK2 Inhibitor 6 Sprycel (dasatinib) is an inhibitor of the SRC-family of protein kinases, BCR-ABL, and ABL, and has additional activity against other kinases including KIT, DDR1/2, PDGFRA/B, and EPHA2, which prevents cell growth (PMID: 25709401, PMID: 25284748, PMID: 18797457). Sprycel (dasatinib) is FDA approved for both Philadelphia chromosome positive adult and pediatric chronic myelogenous leukemia and adult acute lymphoblastic leukemia (FDA.gov).
Daunorubicin Cerubidine Rubidomycin|daunomycin hydrochloride|rubomycin C|Acetyladriamycin Chemotherapy - Anthracycline 11 Cerubidine (daunorubicin) is an anthracycline, which inhibits DNA replication and repair (NCI Drug Dictionary). Cerubidine (daunorubicin) is FDA approved for AML and MLL (FDA.gov).
Daunoxome liposomal daunorubicin citrate Chemotherapy - Anthracycline 11 Daunoxome is a liposomal formulation of the anthracyline, daunorubicin (NCI Drug Dictionary) and is FDA approved for HIV-associated Kaposi’s sarcoma (FDA.gov).
Decitabine Dacogen 5-aza-2-deoxycytidine DNMT inhibitor (Pan) 5 Dacogen (decitabine) is cytidine analog that incorporates into DNA and forms covalent bonds with DNA methyltransferases (DNMTs), resulting in decreased DNMT activity and hypomethylation, and potentially leading to reduced tumor growth (PMID: 28159832, PMID: 25130173). Dacogen (decitabine) is FDA approved for use in patients with myelodysplastic syndromes (FDA.gov).
Denosumab Xgeva AMG162|AMG-162|AMG 162|Prolia Xgeva (denosumab) is an antibody that binds to RANKL protein, inhibiting osteoclast activity (NCI Drug Dictionary). Xgeva (denosumab) is FDA approved for use in patients with giant cell tumor of the bone (FDA.gov).
Dinutuximab Unituxin MOAB Ch14.18 Unituxin (dinutuximab) is a monoclonal antibody that targets B4GALNT1 (GD2) and increases antibody-dependent and complement-dependent cytotoxicity against GD2-expressing tumor cells (NCI Drug Dictionary). Unituxin (dinutuximab) is FDA approved for pediatric high-risk neuroblastoma (FDA.gov).
Doxorubicin Adriamycin Adria|ADR Chemotherapy - Anthracycline 11 TOPO2 inhibitor 4 Adriamycin (doxorubicin) is an anthracycline chemotherapeutic, in a non-liposomal formulation, which intercalates into DNA and inhibits topoisomerase II (PMID: 24367159). Doxorubicin is FDA approved for multiple cancer types (FDA.gov).
Durvalumab Imfinzi MEDI4736 Immune Checkpoint Inhibitor 99 PD-L1/PD-1 antibody 48 Imfinzi (durvalumab) is a monoclonal antibody that binds to and inhibits PD-L1 (CD274), potentially resulting in increased immune response to tumors (PMID: 25943534, 28214651). Imfinzi (durvalumab) is FDA approved for use in patients with urothelial carcinoma and unresectable, stage III non-small cell lung cancer (FDA.gov).
Duvelisib Copiktra IPI-145|INK1197 PIK3CD inhibitor 24 PIK3CG inhibitor 8 Copiktra (duvelisib) prevents the activation of the PI3K delta/gamma-mediated signaling pathways, resulting in tumor cell death (PMID: 25258342). Copiktra (duvelisib) is FDA approved for use in adult patients with relapsed or refractory chronic lymphocytic leukemia, small lymphocytic lymphoma, and follicular lymphoma (FDA.gov)
Elotuzumab Empliciti Empliciti (elotuzumab) is a humanized monoclonal antibody against human CS1, which induces antibody-dependent cellular cytotoxicity in CS1-positive cells (PMID: 26005365). Empliciti (elotuzumab) is approved in combination with Revlimid (lenalidomide) and dexamethasone for use in patients with multiple myeloma (FDA.gov).
Enasidenib Idhifa AG-221 IDH2 Inhibitor 2 Idhifa (enasidenib) inhibits IDH2 to prevent 2HG formation, which may promote differentiation and inhibit proliferation of cancer cells (PMID: 28280273). Idhifa (enasidenib) is FDA-approved for use in patients with relapsed or refractory acute myeloid leukemia harboring an IDH2 mutation (FDA.gov).
Encorafenib Braftovi LGX818 BRAF Inhibitor 18 Braftovi (encorafenib) is an inhibitor of Raf kinase with selective activity against BRAF V600E, resulting in growth inhibition (PMID: 24864047). Braftovi (encorafenib) in combination with Mektovi (binimetinib) is FDA approved for use in patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation (FDA.gov).
Enzalutamide Xtandi MDV3100 Hormone - Anti-androgens 22 Xtandi (enzalutamide) is a second-generation small molecule androgen receptor (AR) inhibitor that inhibits AR signaling, thereby resulting in decreased tumor growth (PMID: 24009414, PMID: 25945058). Xtandi (enzalutamide) is FDA approved for use in patients with castration-resistant prostate cancer (FDA.gov).
Epirubicin Ellence Chemotherapy - Anthracycline 11 Ellence (epirubicin) is an anthracycline, which intercalates into DNA and inhibits topoisomerase II (PMID: 24367159). Ellence (epirubicin) is FDA approved as a component of adjuvant therapy in patients with evidence of axillary node tumor involvement for primary breast cancer (FDA.gov).
Erlotinib Tarceva CP358774 EGFR Inhibitor (Pan) 38 EGFR Inhibitor 1st gen 3 Tarceva (erlotinib) is a first-generation EGFR inhibitor, which is FDA approved for non-small cell lung carcinoma patients with EGFR exon 19 deletions and/or EGFR L858R and in combination with gemcitabine for patients with advanced pancreatic cancer (FDA.gov; PMID: 25302162).
Everolimus Afinitor RAD001|Zortress mTORC1 Inhibitor 8 Afinitor (everolimus) binds to FKBP-12 and allosterically inhibits mTOR, leading to decreased mTORC1 signaling and potentially resulting in decreased tumor cell growth (PMID: 17766661, PMID: 28400999). Afinitor (everolimus) is FDA approved for use in neuroendocrine tumors of pancreatic, lung or gastrointestinal tract origin, advanced renal cell carcinoma, and in combination with Aromasin (exemestane) in hormone receptor-positive, HER2-negative breast cancer (FDA.gov).
Fingolimod Gilenya FTY720 PP2A Activator 7 Gilenya (fingolimod) is a sphingosine 1-phosphate receptor modulator that activates PP2A, which may result in restoration of tumor suppressor activity thereby leading to apoptosis and inhibition of cell proliferation (PMID: 24448818). Gilenya (fingolimod) is FDA approved for multiple sclerosis (FDA.gov).
Fluorouracil Adrucil 5-FU Chemotherapy - Antimetabolite 10 Adrucil (fluorouracil) is an antimetabolite chemotherapeutic agent, which interferes with DNA and RNA synthesis thereby preventing cancer cell growth (NCI Drug Dictionary). Fluorouracil is FDA approved for colorectal, breast, stomach, and pancreatic cancer (FDA.gov).
Fulvestrant Faslodex ICI 182,780 Hormone - Anti-estrogens 15 Faslodex (fulvestrant) is a competitive inhibitor of estrogen that binds and deforms estrogen receptors, and causes inhibition of growth in cultured estrogen-sensitive breast cancer cells (NCI Drug Dictionary). Faslodex (fulvestrant) is FDA approved for use in patients with hormone receptor-positive breast cancer (FDA.gov).
Gefitinib Iressa ZD1839 EGFR Inhibitor (Pan) 38 EGFR Inhibitor 1st gen 3 Iressa (gefitinib) is a first-generation EGFR inhibitor that binds EGFR and inhibits downstream signaling, potentially resulting in decreased growth of tumors with EGFR activation (PMID: 26980062, PMID: 25302162, PMID: 15284455). Iressa (gefitinib) is FDA approved for use in patients with non-small cell lung cancer harboring an EGFR exon 19 deletion or EGFR L858R (FDA.gov).
Gemtuzumab ozogamicin Mylotarg gemtuzumab|CDP-771|CMA-676|WAY-CMA-676 Mylotarg (gemtuzumab ozogamicin) is an antibody-drug conjugate comprising an anti-CD33 antibody linked to the cytotoxic agent calicheamicin, which results in DNA damage in CD33-expressing tumor cells (PMID: 28607471). Mylotarg (gemtuzumab ozogamicin) is FDA approved for CD33-positive acute myeloid leukemia (FDA.gov).
Gilteritinib Xospata ASP2215 AXL Inhibitor 24 FLT3 Inhibitor 48 Xospata (gilteritinib) is a small molecule inhibitor of FLT3 and AXL that has activity against FLT3-ITD, FLT3 F691L, and FLT3 D835 mutations, potentially resulting in decreased tumor growth (J Clin Oncol 32:5s, 2014 (suppl; abstr 7070), PMID: 25891481). Xospata (gilteritinib) is FDA approved for use in patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation (FDA.gov).
Glasdegib Daurismo PF-04449913 SMO Inhibitor 16 Daurismo (glasdegib) inhibits Smoothened (SMO), resulting in decreased Hedgehog (Hh) pathway signalling, and potentially inhibiting Hh-mediated tumor growth (PMID: 24900436, PMID: 25388167). Daurismo (glasdegib) is FDA approved for use in combination with low-dose cytarabine in patients 75 years old or older with newly-diagnosed acute myeloid leukemia (FDA.gov).
Goserelin Zoladex Goserelin acetate Zoladex (goserelin) is a synthetic analog of lutenizing hormone-releasing hormone, which causes decreased testosterone production in males and decreased estradiol production in females (NCI Drug Dictionary). Zoladex (goserelin) is FDA approved for use in prostate carcinoma (FDA.gov).
Idarubicin Idamycin Idarubicin Hcl Chemotherapy - Anthracycline 11 Idarubicin is an anthracycline, which inhibits DNA replication thereby preventing RNA and protein synthesis (NCI Drug Dictionary). Idarubicin, in combination with other approved drugs, is FDA approved for acute myelogenous leukemia (FDA.gov).
Idelalisib Zydelig CAL-101|GS-1101 PIK3CD inhibitor 24 Zydelig (idelalisib) inhibits PI3K-delta, preventing the activation of the PI3K signaling pathway and inhibiting tumor cell proliferation, motility, and survival (NCI Drug Dictionary). Zydelig (idelalisib) is FDA approved for follicular B-cell non-Hodgkin lymphoma, relapsed small lymphocytic lymphoma, and chronic lymphocytic leukemia (FDA.gov).
Imatinib mesylate Gleevec CGP-57148B|STI571 ABL Inhibitor (pan) 8 BCR-ABL Inhibitor 23 CSF1R Inhibitor 22 KIT Inhibitor 48 PDGFR Inhibitor (Pan) 27 Gleevec (imatinib mesylate) is a multi-target inhibitor of c-Kit, Pdgfr, and Bcr-Abl (NCI Drug Dictionary). Gleevec (imatinib mesylate) is FDA approved for c-KIT positive GIST and dermatofibrosarcoma protuberans, Philadelphia chromosome positive chronic myeloid leukemia, and FIP1L1-PDGFRA positive chronic eosinophilic leukemia (FDA.gov).
Ipilimumab Yervoy BMS-734016 CTLA4 Antibody 8 Immune Checkpoint Inhibitor 99 Yervoy (ipilimumab) is an antibody that binds to cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4), causing increased T-cell activation (NCI Drug Dictionary). Yervoy (ipilimumab) is FDA approved for use in patients with metastatic melanoma, including patients 12 years or older, and in combination with Opdivo (nivolumab) for intermediate or poor-risk renal cell carcinoma and microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (FDA.gov).
Ivosidenib Tibsovo AG-120 IDH1 Inhibitor 7 Tibsovo (ivosidenib) inhibits mutant IDH1, leading to increased differentiation and decreased proliferation in IDH1 positive tumors (PMID: 27245312). Tibsovo (ivosidenib) is FDA approved for adult patients with relapsed or refractory IDH1-mutant acute myeloid leukemia (FDA.gov).
KTE-C19 Yescarta KTE-C19 CAR|axicabtagene ciloleucel Yescarta (KTE-C19) is an immunotherapy consisted of peripheral T-lymphocytes engineered to express a chimeric antigen receptor targeting CD19, CD28 and CD3zeta, resulting in immunostimulating and antineoplastic activities (Blood 2015 126:3991). Yescarta (KTE-C19) is FDA approved for treatment of relapsed or refractory large B-cell lymphoma (FDA.gov).
Lapatinib Tykerb FMM EGFR Inhibitor (Pan) 38 HER2 (ERBB2) Antibody 25 Tykerb (lapatinib) reversibly inhibits ERBB2 (HER2) and EGFR, resulting in decreased downstream signaling and potentially leading to reduced tumor growth (PMID: 22477724). Tykerb (lapatinib) is FDA approved for ERBB2 (HER2)-positive breast cancer (FDA.gov).
Larotrectinib Vitrakvi LOXO-101|ARRY-470 Trk Receptor Inhibitor (Pan) 21 Vitrakvi (larotrectinib) binds to and prevents activation of TRK receptors (NTRK1, -2, -3), resulting in decreased growth and increased apoptosis in Trk-expressing tumor cells (PMID: 29532798). Vitrakvi (larotrectinib) is FDA approved for use in adult and pediatric patients with solid tumors that have progressed, are metastatic, or are not surgically resectable and have an NTRK fusion without acquired resistance mutations (FDA.gov).
Lenvatinib Lenvima E7080 FGFR Inhibitor (Pan) 17 KIT Inhibitor 48 PDGFR Inhibitor (Pan) 27 RET Inhibitor 36 VEGFR Inhibitor (Pan) 29 Lenvima (lenvatinib) inhibits VEGFR, FGFR, PDGFR, KIT, and RET, and suppresses cell proliferation and angiogenesis (PMID: 21781317, PMID: 25295214, PMID: 17943726). Lenvima (lenvatinib) is FDA approved for use in patients with radioactive iodine-refractory differentiated thyroid cancer, unresectable hepatocellular carcinoma, and in combination with Afinitor (everolimus) for renal cell carcinoma (FDA.gov).
Letrozole Femara Letrozol Aromatase Inhibitor 3 Femara (letrozole) is an aromatase inhibitor, which inhibits estrogen synthesis (NCI Drug Dictionary). Femara (letrozole) is approved for postmenopausal women with hormone receptor positive breast cancer (FDA.gov).
Lorlatinib Lorbrena PF-06463922 ALK Inhibitor 19 ROS1 Inhibitor 14 Lorbrena (lorlatinib) is a small molecule inhibitor of Alk and Ros1 fusion proteins, which may result in suppression of Alk and Ros1-mediated signaling thereby inhibiting cell growth and causing tumor regression (PMID: 26144315). Lorbrena (lorlatinib) is FDA approved for use in patients with ALK-positive non-small cell lung cancer (FDA.gov).
Melphalan Alkeran Chemotherapy - Alkylating 14 Alkeran (melphalan) is an antineoplastic alkylating agent, which cross-links DNA and induces cell toxicity (NCI Drug Dictionary). Alkeran (melphalan) is FDA approved for multiple myeloma and epithelial ovarian carcinoma (FDA.gov).
Metformin Glucophage Apo-Metformin Glucophage (metformin) inhibits part of the mitochondrial respiratory chain and may exert antineoplastic effects through inhibition of mTOR (NCI Drug Dictionary). Glucophage (metformin) is FDA approved for type 2 diabetes (FDA.gov).
Methotrexate Abitrexate Amethopterin Chemotherapy - Antimetabolite 10 Methotrexate is an antimetabolite, which inhibits DHFR resulting in decreased immune function and antineoplastic activity (NCI Drug Dictionary). Methotrexate is FDA approved for psoriasis, RA, and several cancers including choriocarcinoma, AML, lung, head and neck and epidermoid (FDA.gov).
Midostaurin Rydapt PKC412|CGP 41251 CSF1R Inhibitor 22 FLT3 Inhibitor 48 KIT Inhibitor 48 PKC alpha Inhibitor 6 PKC beta Inhibitor 6 PKC Inhibitor (Pan) 9 VEGFR2 Inhibitor 34 Rydapt (midostaurin) is a multi-kinase inhibitor with activity against FLT3, KIT, PDGFRB, KDR (VEGFR2) and PKC, with higher selectivity for conventional PKC isoforms, which induces cell cycle arrest and apoptosis (PMID: 12124173, PMID: 23127174, PMID: 15914319). Rydapt (midostaurin) is FDA approved for FLT3-mutant AML in combination with chemotherapy, and is approved for use in aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, or mast cell leukemia as a single therapy (FDA.gov).
Mogamulizumab Poteligeo KW-0761|Mogamulizumab-kpkc Poteligeo (mogamulizumab-kpkc) is a monoclonal antibody that binds to and inhibits C-C chemokine receptor 4 (CCR4) signaling, which leads to inhibition of Tregs and enhancement of host antitumor immunity (PMID: 25496334). Poteligeo (mogamulizumab-kpkc) is FDA approved for adult patients with relapsed or refractory mycosis fungoides or Sézary syndrome (FDA.gov).
Moxetumomab pasudotox-tdfk Lumoxiti Lumoxiti (moxetumomab pasudotox-tdfk) is an immunotoxin targeting CD22-positive tumor cells (PMID: 30030507). Lumoxiti (moxetumomab pasudotox-tdfk) is FDA approved for use in patients with relapsed or refractory hairy cell leukemia (FDA.gov).
Neratinib Nerlynx HKI-272|PB272 HER2 Inhibitor 20 Nerlynx (neratinib) inhibits the ERBB2 (HER2) receptor as well as EGFR, resulting in decreased proliferation of HER2 and EGFR expressing tumor cells (PMID: 15173008). Nerlynx (neratinib) is FDA approved for breast cancer patients with ERBB2 (HER2) overexpression and/or amplification (FDA.gov).
Nilotinib Tasigna AMN-107 ABL Inhibitor (pan) 8 BCR-ABL Inhibitor 23 CSF1R Inhibitor 22 DDR1 Inhibitor 8 DDR2 inhibitor 7 KIT Inhibitor 48 PDGFR Inhibitor (Pan) 27 Tasigna (nilotinib) inhibits several tyrosine kinases including BCR-ABL, PDGFR, KIT, DDR and CSF-1R, potentially resulting in reduced tumor cell proliferation (PMID: 21419934, PMID: 25284748). Tasigna (nilotinib) is FDA approved for use in patients with Ph+ chronic myeloid leukemia (FDA.gov).
Nintedanib Ofev BIBF 1120|Vargatef FGFR Inhibitor (Pan) 17 PDGFR Inhibitor (Pan) 27 RET Inhibitor 36 VEGFR Inhibitor (Pan) 29 Ofev (nintedanib) is a triple angiokinase inhibitor that acts by blocking VEGFR, FGFR, RET, and PDGFR, thereby preventing tumorigenesis (PMID: 24947258, PMID: 24561444). Ofev (nintedanib) is FDA approved for use in patients with idiopathic pulmonary fibrosis (FDA.gov).
Niraparib Zejula MK4827 PARP Inhibitor (Pan) 17 Zejula (niraparib) binds and inhibits PARP, which may result in accumulation of DNA damage and apoptosis of tumor cells (PMID: 23810788). Zejula (niraparib) is FDA-approved as maintenance therapy in patients with epithelial ovarian, fallopian tube, or primary peritoneal cancer in complete or partial response to platinum-based chemotherapy (FDA.gov).
Nivolumab Opdivo MDX-1106|BMS-936558 Immune Checkpoint Inhibitor 99 PD-L1/PD-1 antibody 48 Opdivo (nivolumab) is an antibody that targets PD-1 (PDCD1), which results in increased T-cell activation and enhanced anti-tumor immune response (NCI Drug Dictionary). Opdivo (nivolumab) is FDA approved for the treatment of metastatic melanoma, squamous NSCLC, metastatic small cell lung cancer, renal cell carcinoma, head and neck squamous cell carcinoma, urothelial carcinoma, Hodgkin's lymphoma, hepatocellular carcinoma, and for dMMR or MSI-H colorectal cancer as a single agent or in combination with Yervoy (ipilimumab) (FDA.gov).
Obinutuzumab Gazyva R7159|afutuzumab|GA101|GA-101|RO 5072759 CD20 Antibody 3 Gazyva (obinutuzumab) is a glycoengineered anti-CD20 antibody that binds to CD20 on B-cells, resulting in increased antibody-dependent cellular cytotoxicity and induction of apoptosis, and potentially leading to decreased growth of B-cell tumors (PMID: 20194898, PMID: 21220500). Gazyva (obinutuzumab) is FDA approved in combination with Ambochlorin (chlorambucil) for chronic lymphocytic leukemia and in combination with Treanda (bendamustine) or chemotherapy for follicular lymphoma (FDA.gov).
Ofatumumab Arzerra HuMax-CD20 Arzerra (ofatumumab) is a monoclonal antibody that binds to CD20 and induces cytotoxic immune response against CD20 over-expressing cells (PMID: 26855591). Arzerra (ofatumumab) is FDA approved for chronic lymphocytic leukemia (FDA.gov).
Olaparib Lynparza AZD2281|KU-0059436 PARP Inhibitor (Pan) 17 Lynparza (olaparib) binds to and inhibits PARP, resulting in inhibition of DNA repair and lethality in homologous-recombination deficient cells, and may be a sensitizing agent for chemotherapy and radiotherapy (PMID: 25028150, PMID: 24225019). Lynparza (olaparib) is FDA approved for use in patients with germline BRCA-mutated advanced ovarian cancer, germline BRCA-mutated and ERBB2 (HER2)-negative breast cancer, and advanced epithelial ovarian, fallopian tube or primary peritoneal cancer with deleterious or suspected deleterious germline or somatic BRCA mutations (FDA.gov).
Olaratumab Lartruvo IMC-3G3 Lartruvo (olaratumab) is a monoclonal antibody that targets PDGFR-alpha and prevents ligand binding, potentially leading to decreased angiogenesis and reduced proliferation of tumor cells (NCI Drug Dictionary). Lartruvo (olaratumab) is FDA approved for patients with soft tissue sarcoma (FDA.gov).
Osimertinib Tagrisso AZD9291 EGFR Inhibitor 3rd gen 15 Tagrisso (osimertinib) is a third-generation EGFR inhibitor that selectively binds mutant forms of the EGF receptor, inhibiting downstream signaling and potentially reducing cell proliferation and growth in EGFR overexpressing tumors (NCI Drug Dictionary). Tagrisso (osimertinib) is FDA-approved for use in patients with non-small cell lung cancer harboring EGFR exon 19 deletions, L858R, or T790M (FDA.gov).
Oxaliplatin Eloxatin Diaminocyclohexane Oxalatoplatinum Chemotherapy - Platinum 6 Eloxatin (oxaliplatin) is comprised of a platinum complex, which causes DNA-platinum cross-links, inhibition of DNA replication and transcription, and cell toxicity (NCI Drug Dictionary). Eloxatin (oxaliplatin) is FDA approved for colorectal cancer (FDA.gov).
Palbociclib Ibrance PD0332991 CDK4/6 Inhibitor 8 Ibrance (palbociclib) is a selective inhibitor of cyclin-dependent kinase 4 (CDK4) and 6 (CDK6) (PMID: 19874578). Ibrance (palbociclib) is approved in combination with an aromatase inhibitor in postmenopausal patients with ER-positive, ERBB2 (HER2)-negative metastatic breast cancer, and in combination with Faslodex (fulvestrant) in patients with ER-positive, ERBB2 (HER2)-negative metastatic breast cancer (FDA.gov).
Panitumumab Vectibix ABX-EGF EGFR Antibody 27 Vectibix (panitumumab) is a monoclonal antibody directed against EGFR, which inhibits cell proliferation and induces apoptosis (PMID: 18998757). Vectibix (panitumumab) is FDA approved for metastatic colorectal cancer patients with wild-type KRAS and NRAS (FDA.gov).
Panobinostat Farydak LBH589 HDAC Inhibitor 34 Farydak (panobinostat) is an HDAC inhibitor, which induces cell-cycle arrest and decreases growth of tumor cells (PMID: 18349321, PMID: 19671764, PMID: 27802904). Farydak (panobinostat) is FDA approved, in combination with Velcade (Bortezomib) and dexamethasone, for multiple myeloma (FDA.gov).
Pazopanib Votrient GW-786034 FGFR1 Inhibitor 21 FGFR2 Inhibitor 15 FGFR3 Inhibitor 12 KIT Inhibitor 48 PDGFR Inhibitor (Pan) 27 VEGFR Inhibitor (Pan) 29 Votrient (pazopanib) inhibits VEGFR-1, -2 and -3, KIT, and PDGFR, and FGFR1, 2, and 3, leading to decreased tumor angiogenesis and growth (PMID: 17620431, PMID: 23786770). Votrient (pazopanib) is approved for renal cell carcinoma and soft tissue sarcoma (FDA.gov).
Pembrolizumab Keytruda MK-3475 Immune Checkpoint Inhibitor 99 PD-L1/PD-1 antibody 48 Keytruda (pembrolizumab) is an antibody that binds to and inhibits PD-1, resulting in activation of T-cell mediated immune response against tumor cells (PMID: 25977344). Keytruda (pembrolizumab) is approved for use in patients with melanoma, head and neck squamous cell carcinoma, classical Hodgkin Lymphoma, primary mediastinal large B-cell lymphoma, urothelial carcinoma, hepatocellular carcinoma, Merkel cell carcinoma, MSI-H or dMMR solid tumors, PD-L1 expressing NSCLC, gastric cancer, GEJ adenocarcinoma, and cervical cancer, in combination with pemetrexed and platinum in non-squamous NSCLC with no EGFR or ALK mutations, and in combination with carboplatin and paclitaxel/nab-paclitaxel in squamous NSCLC (FDA.gov).
Pertuzumab Perjeta 2C4 Antibody HER2 (ERBB2) Antibody 25 Perjeta (pertuzumab) is a monoclonal antibody that binds ERBB2 (HER2) to prevent dimerization and subsequent HER signaling, thereby resulting in apoptosis and tumor growth inhibition (PMID: 15699478). Perjeta (pertuzumab) is FDA approved in combination with Herceptin (trastuzumab) and Taxotere (docetaxel) for patients with ERBB2 (HER2)-positive breast cancer and in combination with Herceptin (trastuzumab) and chemotherapy as neoadjuvant treatment for patients with ERBB2 (HER2)-positive breast cancer (FDA.gov).
Ponatinib Iclusig AP24534 BCR-ABL Inhibitor 23 DDR1 Inhibitor 8 DDR2 inhibitor 7 FGFR Inhibitor (Pan) 17 FLT3 Inhibitor 48 KIT Inhibitor 48 PDGFR-alpha Inhibitor 7 RET Inhibitor 36 SRC Inhibitor 26 VEGFR Inhibitor (Pan) 29 Iclusig (ponatinib) inhibits the Bcr-Abl fusion, and other tyrosine kinases, such as RET, DDR, VEGFR, FGFR, KIT, and FLT3 (PMID: 23526464, PMID: 25284748, PMID: 19878872). Iclusig (ponatinib) is FDA approved for chronic myeloid leukemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukemia (Ph positive ALL) as well as ABL1 T315I CML and ABL1 T315I Ph positive ALL (FDA.gov).
Ramucirumab Cyramza LY3009806 VEGFR2 Antibody 4 Cyramza (ramucirumab) is a monoclonal antibody, which binds and inhibits VEGFR2 (KDR) resulting in decreased angiogenesis (NCI Drug Dictionary). Cyramza (ramucirumab) is approved for gastric and gastroesophageal junction adenocarcinoma, non-small cell lung cancer, and colorectal cancer (FDA.gov).
Regorafenib Stivarga BAY 73-4506 BRAF Inhibitor 18 CRAF Inhibitor 10 KIT Inhibitor 48 PDGFR Inhibitor (Pan) 27 RET Inhibitor 36 VEGFR Inhibitor (Pan) 29 VEGFR2 Inhibitor 34 Stivarga (regorafenib) is an inhibitor of several kinases including VEGFR, RET, KIT, PDGFR and RAF, thereby inhibiting tumor angiogenesis and tumor cell proliferation (PMID: 24756792). Stivarga (regorafenib) is FDA approved for colorectal cancer, hepatocellular carcinoma, and GIST (FDA.gov).
Ribociclib Kisqali LEE011 CDK4/6 Inhibitor 8 Kisqali (ribociclib) is a dual CDK4/6 inhibitor, which may induce cell cycle arrest and reduce proliferation in cancer cells (PMID: 24045179). Kisqali (ribociclib) is FDA-approved in combination with an aromatase inhibitor in women with hormone receptor-positive, ERBB2 (HER2)-negative breast cancer, and in combination with Faslodex (fulvestrant) in postmenopausal women with hormone receptor-positive, ERBB2 (HER2)-negative breast cancer (FDA.gov).
Rituximab Rituxan IDEC-C2B8 CD20 Antibody 3 Rituxan (rituximab) is a chimeric mononclonal CD20-targeted antibody that binds to CD20 on B-cells, resulting in induction of complement-dependent cytotoxicity and antibody-dependent cellular cytoxicity, and potentially leading to decreased B-cell tumor growth (PMID: 28983798). Rituxan (rituximab) is FDA approved for use in non-Hodgkin lymphoma and chronic lymphocytic leukemia (FDA.gov).
Romidepsin Istodax FK228|FR901228|NSC 630176|depsipeptide HDAC Inhibitor 34 Istodax (romidepsin) inhibits Class I HDACs, resulting in cell cycle arrest, apoptosis, and decreased tumor cell growth (PMID: 12208741, PMID: 12234611, PMID: 21258344). Istodax (romidepsin) is FDA approved for patients with cutaneous T-cell lymphoma (FDA.gov).
Rucaparib Rubraca AG014699|PF-01367338|CO-388|AG14447 PARP Inhibitor (Pan) 17 Rubraca (rucaparib) binds to and inhibits PARP, which may result in accumulation of DNA damage and chemosensitization of tumor cells (PMID: 17363489). Rubraca (rucaparib) is FDA approved for use in ovarian cancer patients with deleterious somatic and/or germline BRCA mutations (FDA.gov).
Ruxolitinib Jakafi INCB 18424|INCB018424|INCB18424 JAK1 Inhibitor - ATP competitive 4 JAK2 Inhibitor - ATP competitive 14 Jakafi (ruxolitinib) is an inhibitor of protein tyrosine kinases JAK1 and JAK2, thus resulting in reduced inflammation and reduced proliferation (NCI Drug Dictionary). Jakafi (ruxolitinib) is FDA approved to treat bone marrow cancer, specifically intermediate or high-risk myelofibrosis (FDA.gov)
Sirolimus Rapamune Rapamycin mTORC1 Inhibitor 8 Rapamune (sirolimus) binds to the FKBP-12 to generate an immunosuppressive complex that binds and allosterically inhibits mTOR (NCI Drug Dictionary, PMID: 25261369). Rapamune (sirolimus) is FDA approved for the prevention of renal transplant rejection and for patients with lymphangioleiomyomatosis (FDA.gov).
Sorafenib Nexavar BAY 43-9006 CSF1R Inhibitor 22 FLT3 Inhibitor 48 KIT Inhibitor 48 PDGFR-beta Inhibitor 13 RAF Inhibitor (Pan) 17 RET Inhibitor 36 VEGFR2 Inhibitor 34 Nexavar (sorafenib) is a multikinase inhibitor with activity against several kinases, including RAF kinases, VEGFR2, VEGFR3, PDGFR-beta, KIT, FLT3, RET, and CSF1R, potentially resulting in decreased tumor growth (PMID: 18445656, PMID: 15466206, PMID: 21517818). Nexavar (sorafenib) is approved for metastatic differentiated thyroid carcinoma, hepatocellular carcinoma, and renal cell carcinoma (FDA.gov).
Sulindac Clinoril Clinoril (sulindac) is a non-selective COX1-2 inhibitor, FDA approved as an anti-inflammatory agent (FDA.gov). Clinoril (sulindac) also reduces intestinal tumor burden (PMID: 19755659).
Sunitinib Sutent SU011248 CSF1R Inhibitor 22 FLT3 Inhibitor 48 KIT Inhibitor 48 PDGFR Inhibitor (Pan) 27 RET Inhibitor 36 VEGFR2 Inhibitor 34 Sutent (sunitinib) inhibits KDR (VEGFR2), PDGFR, c-KIT, FLT3, RET, and CSF1R, thereby inhibiting angiogenesis and cell proliferation (NCI Drug Dictionary; PMID: 25085632). Sutent (sunitinib) is approved for neuroendocrine tumors of the pancreas, advanced renal cell carcinoma, and GIST (FDA.gov).
Talazoparib Talzenna BMN673 PARP Inhibitor (Pan) 17 Talzenna (talazoparib) is an inhibitor of PARP1 and PARP2, which prevents the DNA repair of single strand DNA breaks, thus causing the accumulation of DNA strand breaks, genomic instability and apoptosis, and leads to lethality in homologous recombination repair deficient cells (PMID: 28242752). Talzenna (talazoparib) is FDA approved for use in patients with ERBB2 (HER2)-negative breast cancer harboring deleterious or suspected deleterious germline BRCA mutations (FDA.gov).
Temsirolimus Torisel CCI-779 mTORC1 Inhibitor 8 Torisel (temsirolimus) binds to FKBP-12 and allosterically inhibits mTOR (PMID: 16883305). Torisel (temsirolimus) is FDA approved for advanced renal cell carcinoma (FDA.gov).
Tisagenlecleucel Kymriah CTL019 Kymriah (tisagenlecleucel) is a preparation of autologous T-lymphocytes engineered to express chimeric antigen receptors targeting CD19-positive cells, resulting in anti-tumor immune response (J Clin Oncol 35, 2017 (suppl; abstr 10523)). Kymriah (tisagenlecleucel) is FDA approved for pediatric and young adult patients with B-cell precursor acute lymphoblastic leukemia, and adult patient with elapsed or refractory large B-cell lymphoma (FDA.gov).
Tocilizumab Actemra Atlizumab Actemra (tocilizumab) is a humamized monoclonal antibody that targets IL-6R, resulting in decreased downstream signaling and potentially resulted in decreased tumor growth and progression (PMID: 26751841, PMID: 25658637). Actemra (tocilizumab) is FDA approved for use in treating rheumatoid arthritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis (FDA.gov).
Tofacitinib Xeljanz Tasocitinib|CP-690550|CP-690,550 JAK Inhibitor (Pan) - ATP competitive 3 Xeljanz (tofacitinib) is a pan-JAK inhibitor, which decreases downstream signaling and potentially leads to reduced tumor cell growth (PMID: 26300391, PMID: 21106989). Xeljanz (tofacitinib) is FDA approved for rheumatoid arthritis (FDA.gov).
Trametinib Mekinist GSK1120212 MEK inhibitor (Pan) 20 MEK1 Inhibitor 20 MEK2 Inhibitor 18 Mekinist (trametinib) inhibits MEK 1 and 2, which potentially leads to reduced tumor cell proliferation (PMID: 27956260). Mekinist (trametinib) is FDA approved for melanoma patients harboring BRAF V600E or BRAF V600K mutations, and in combination with Tafinlar (dabrafinib) for BRAF V600E/K-mutant melanoma, BRAF V600E- mutant non-small cell lung cancer, and BRAF V600E-mutant anaplastic thyroid cancer (FDA.gov).
Trastuzumab Herceptin Anti HER2 HER2 (ERBB2) Antibody 25 Herceptin (trastuzumab) is a monoclonal antibody, which binds ERBB2 (HER2) to induce tumor cellular cytotoxicity (PMID: 17611206). Herceptin (trastuzumab) is FDA approved for HER2-overexpressing breast cancer, gastric adenocarcinoma, and gastroesophageal junction adenocarcinoma (FDA.gov).
Tretinoin Aberel|Aknoten|Avita|Renova|Retin-A|all-trans retinoic acid|Vitamin A Acid|ATRA Tretinoin is a form of retinoic acid, which binds to retinoic acid receptors and activates downstream signaling, potentially inducing cell differentiation and subsequently, apoptosis (PMID: 11704842). Tretinoin is FDA approved for patients with acute promyelocytic leukemia harboring PML-RARA (FDA.gov).
Valproic acid Depakene Depacon HDAC Inhibitor 34 Valproic acid inhibits HDAC activity, potentially resulting in decreased angiogenesis, increased tumor cell differentiation, and reduced tumor growth (PMID: 11742974, PMID: 14978230). Valproic acid is FDA-approved for use in treating seizures (FDA.gov).
Valrubicin Valstar Antibiotic Ad 32|AD-32 Chemotherapy - Anthracycline 11 TOPO2 inhibitor 4 Valstar (valrubicin) is an anthracycline that is metabolized into compounds that inhibit topoisomerase II, resulting in inhibition of DNA synthesis and potentially leading to decreased tumor growth (PMID: 19145271, PMID: 20554745). Valstar (valrubicin) is FDA-approved for treatment of BCG-refractory urinary bladder carcinoma in situ (FDA.gov).
Vandetanib Caprelsa ZD6474 ABL1 Inhibitor 4 EGFR Inhibitor (Pan) 38 RET Inhibitor 36 SRC Inhibitor 26 VEGFR Inhibitor (Pan) 29 Caprelsa (vandetanib) is a multi-tyrosine kinase inhibitor with activity against VEGFR, EGFR, RET, SRC, and ABL1 kinases, potentially resulting in decreased tumor growth (PMID: 25031937, PMID: 25490448). Caprelsa (vandetanib) is FDA approved for use in medullary thyroid cancer (FDA.gov).
Vemurafenib Zelboraf RO5185426|PLX4032 RAF Inhibitor (Pan) 17 Zelboraf (vemurafenib) inhibits BRAF V600E, wild-type BRAF, ARAF, and CRAF (PMID: 20179705), which may result in an inhibition of the MAPK signaling pathway resulting in a reduction of tumor cell proliferation (PMID: 20823850). Zelboraf (vemurafenib) is FDA approved for BRAF V600E-mutant melanoma and for BRAF V600-positive Erdheim-Chester disease (FDA.gov).
Venetoclax Venclexta ABT-199|RG7601|GDC-0199 BCL2 inhibitor 15 Venclexta (venetoclax) is a BH3-mimetic that binds to and inhibits BCL2, resulting in increased tumor cell apoptosis (PMID: 26589495, PMID: 25048785). Venclexta (venetoclax) is FDA approved for use in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, and in combination with chemotherapy in patients 75 years old or older with newly-diagnosed acute myeloid leukemia (FDA.gov).
Vinorelbine Navelbine Vinorelbin Antimicrotubule Agent 11 Navelbine (vinorelbine) is a tubulin binding alkaloid that inhibits tubulin polymerization, thus inhibiting spindle formation, leading to apoptosis (NCI Drug Dictionary). Navelbine (vinorelbine) is approved for use in NSCLC (FDA.gov).
Vismodegib Erivedge GDC-0449 SMO Inhibitor 16 Erivedge (vismodegib) binds to and inhibits Smoothened (SMO), resulting in inhibition of downstream signaling and decreased tumor growth (PMID: 24259609, PMID: 19716296). Erivedge (vismodegib) is FDA approved for use in patients with basal cell carcinoma (FDA.gov).
Vorinostat Zolinza SAHA|L-001079038|MSK390 HDAC Inhibitor 34 Zolinza (vorinostat) inhibits HDAC activity, resulting in cell-cycle arrest, decreased proliferation, and increased differentiation of tumor cells (PMID: 9501205, PMID: 11731433, PMID: 11016644). Zolinza (vorinostat) is FDA approved for use in cutaneous T-cell lymphoma (FDA.gov).
inotuzumab ozogamicin Besponsa CMC-544|InO Besponsa (inotuzumab ozogamicin) consists of calecheamicin covalently linked to anti-CD22 antibody, which delivers calecheamicin into CD22-positive cells, leading to DNA damage and apoptosis (PMID: 24389139). Besponsa (inotuzumab ozogamicin) is FDA approved for relapsed or refractory B-cell precursor acute lymphoblastic leukemia (FDA.gov).
lutetium Lu 177 dotatate Lutathera Lutathera (lutetium Lu 177 dotatate) is a radiolabeled somatostatin analog which may selectively kill somatostatin receptor-positive tumor cells (PMID: 28076709). Lutathera (lutetium Lu 177 dotatate) is FDA approved for use in patients with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (FDA.gov).
pegylated liposomal-doxorubicin Doxil ATI-0918|CAELYX Chemotherapy - Anthracycline 11 Doxil (pegylated liposomal doxorubicin) is doxorubicin encapsulated within a liposome to allow better penetration into tumors, decrease toxicity, and increase the period of effectiveness. Doxorubicin intercalates into DNA, disrupting DNA replication (NCI Drug Dictionary). Doxil is FDA approved for ovarian cancer, AIDS-related Kaposi's sarcoma and multiple myeloma (FDA.gov).
siltuximab Sylvant CNTO 328|CNTO328 Sylvant (siltuxmab) is an anti-IL-6 chimeric monoclonal antibody, which inhibits IL6 signaling and may lead to decreased tumor cell growth (PMID: 26170629, PMID: 26744529). Sylvant (siltuximab) is FDA approved for patients with multicentric Castleman's disease who do not have HIV or HHV-8 (FDA.gov).
tagraxofusp-erzs Elzonris DT(388)IL3 fusion protein|IL3R-targeting fusion protein SL-401 Elzonris (tagraxofusp-erzs) is a fusion protein consists of human interleukin 3 (IL3) and the first 388 amino acids of diphtheria toxin [DT(388)], which binds to IL3 receptors and subsequently inhibits protein synthesis, leading to cell death (PMID: 28479592). Elzonris (tagraxofusp-erzs) is FDA approved for use in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (FDA.gov).
talimogene laherparepvec Imlygic OncoVEX GM-CSF Imlygic (talimogene laherparepvec) is a GM-CSF expressing herpes simplex type-1 virus (HSV-1) that selectively infects and lyses tumor cells, and potentially induces a cytotoxic immune response against tumor cells (PMID: 25777572). Imlygic (talimogene laherparepvec) is FDA approved for use in patients with recurrent melanoma (FDA.gov).
trifluridine/tipiracil hydrochloride Lonsurf TAS-102 Lonsurf (trifluridine/tipiracil hydrochloride) is an oral combination comprised of TFT and a thymidine phosphorylase inhibitor, which can result in DNA fragmentation thereby preventing angiogenesis and tumor growth (NCI Drug Dictionary). Lonsurf (TAS-102) is FDA approved for colorectal cancer (FDA.gov).
Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown Indication other than cancer not applicable Imatinib FDA approved Actionable Gleevec (imatinib) is FDA approved for patients with aggressive systemic mastocytosis in which c-KIT mutational status is unknown (FDA.gov). detail...
Unknown unknown thyroid cancer not applicable Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.8 months in metastatic differentiated thyroid cancer patients (PMID: 24768112). detail... 24768112
Unknown unknown stomach cancer not applicable Ramucirumab FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cyramza (ramucirumab) increased progression-free and overall survival in patients with advanced gastric cancer (PMID: 24094768). 24094768 detail...
Unknown unknown Indication other than cancer not applicable Valproic acid FDA approved Actionable Valproic acid is FDA approved for use in patients with seizures, migraines, and bipolar disorder (FDA.gov). detail...
Unknown unknown thyroid medullary carcinoma not applicable Cabozantinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cometriq (cabozantinib) resulted in improved progression free survival in patients with metastatic medullary thyroid cancer (PMID: 23319867). detail... 23319867
Unknown unknown colorectal cancer not applicable Aflibercept FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Zaltrap (aflibercept) improved median progression free survival to 6.9 months and overall survival to 13.5 months in patients with metastatic colorectal cancer (PMID: 23444216). detail... 23444216
Unknown unknown Indication other than cancer not applicable Metformin FDA approved Actionable Glucophage (metformin) is FDA approved for use in patients with type-2 diabetes (FDA.gov). detail...
Unknown unknown natural killer cell leukemia not applicable Tagraxofusp-erzs FDA approved Actionable In a Phase I/II trial that supported FDA approval, Elzonris (tagraxofusp-erzs) treatment resulted in complete response/clinical complete response in 53.8% (7/13) of patients with untreated blastic plasmacytoid dendritic cell neoplasm (BPDCN, also known as natural killer cell leukemia/lymphoma) with a median follow-up of 11.5 months, and 1 complete response and 1 clinical complete response in 15 patients with relapsed or refractory BPDCN (FDA.gov; NCT02113982). detail...
Unknown unknown prostate cancer sensitive Bicalutamide FDA approved Actionable In a clinical trial that supported FDA approval, combined with a luteinizing hormone-releasing hormone analogue (LHRH-A) therapy, Casodex (bicalutamide) demonstrated efficacy similar to Eulexin (flutamide), resulted in comparable median time to progression (97 vs 77 weeks, HR=0.93, p=0.41) and survival time (180 vs 148 weeks, HR=0.87, p=0.15) in patients with metastatic prostate cancer (PMID: 9301693). 9301693 detail...
Unknown unknown renal cell carcinoma not applicable Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) resulted in an improved median progression-free survival of 167 days in patients with renal cell carcinoma (PMID: 17189398). detail... 17189398
Unknown unknown stomach cancer not applicable Ramucirumab + Paclitaxel FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cyramza (ramucirumab) in combination with Taxol (paclitaxel) increased progression-free and overall survival and improved quality of life compared to treatment with Taxol (paclitaxel) alone in patients with advanced gastric cancer (PMID: 24706672). detail... 24706672
Unknown unknown Indication other than cancer not applicable Nintedanib FDA approved Actionable Ofev (nintedanib) is FDA approved for use in patients with idiopathic pulmonary fibrosis (FDA.gov). detail...
Unknown unknown Indication other than cancer not applicable Sirolimus FDA approved Actionable Rapamune (sirolimus) is FDA approved for use in patients with lymphangioleiomyomatosis and for prophylactic immunosuppression in renal transplant patients (FDA.gov). detail...
Unknown unknown basal cell carcinoma not applicable Vismodegib FDA approved Actionable In a Phase II trial that supported FDA approval, treatment with Erivedge (vismodegib) resulted in an objective response rate of 30% (10/33) in patients with metastatic basal cell carcinoma and 43% (27/63) in patients with locally advanced basal cell carcinoma, with 21% (13/63) patients with locally advanced basal cell carcinoma demonstrating complete response (PMID: 22679179, PMID: 22670903). detail... 22679179 22670903
Unknown unknown gastroesophageal junction adenocarcinoma not applicable Ramucirumab + Paclitaxel FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cyramza (ramucirumab) in combination with Taxol (paclitaxel) increased progression-free and overall survival and improved quality of life compared to treatment with Taxol (paclitaxel) alone in patients with gastroesophageal junction adenocarcinoma (PMID: 24706672). detail... 24706672
Unknown unknown sarcoma not applicable Pazopanib Clinical Study Actionable In a retrospective study, Votrient (pazopanib) treatment resulted in median progression free survival of 15.4 weeks and median survival of 11.2 months in patients with soft tissue sarcoma (PMID: 26970174). 26970174
Unknown unknown sarcoma not applicable Pazopanib FDA approved Actionable In a Phase III trial that supported FDA approval, Votrient (pazopanib) improved progression free survival in patients with advanced soft tissue sarcoma (PMID: 22595799). detail... 22595799
Unknown unknown pancreatic endocrine carcinoma not applicable Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, Sutent (sunitinib) demonstrated safety and improved progression free survival in patients with pancreatic neuroendocrine tumors (PMID: 21306237). 21306237 detail...
Unknown unknown thyroid cancer not applicable Lenvatinib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Lenvima (lenvatinib) improved progression free survival and response rates in patients with radioiodine-refractory thyroid cancer (PMID: 25671254). detail... 25671254
Unknown unknown thyroid cancer not applicable Lenvatinib Phase II Actionable In a Phase II clinical trial, Lenvima (lenvatinib) demonstrated partial response in 36% (21/59) of patients, partial response or stable disease in 80% (47/59), and median progression free survival of 9 months in patients with advanced medullary thyroid cancer (PMID: 26311725). 26311725
PDGFRA rearrange myelodysplastic/myeloproliferative neoplasm sensitive Imatinib FDA approved Actionable Gleevec (imatinib) is FDA approved for myelodysplastic/myeloproliferative cancers that are associated with PDGFR rearrangements (FDA.gov). detail...
Unknown unknown lymphocytic leukemia not applicable Ponatinib FDA approved Actionable In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). detail... 23935038
Unknown unknown gastrointestinal stromal tumor not applicable Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) improved median progression free survival to 27.3 weeks in GIST patients (PMID: 17332278). detail... 17332278
FLT3 act mut acute myeloid leukemia sensitive Gilteritinib FDA approved Actionable In a Phase III trial (ADMIRAL) that supported FDA approval, Xospata (gilteritinib) treatment resulted in complete remission (CR) or CR with partial hematologic recovery in 21% (29/138) of patients with relapsed or refractory acute myeloid leukemia harboring a FLT3 internal tandem duplication (ITD), D835, or I836 mutation (FDA.gov; NCT02421939). detail...
Unknown unknown renal cell carcinoma not applicable Pazopanib Phase III Actionable In a Phase III trial, adjuvant Votrient (pazopanib) therapy post nephrectomy did not improve disease-free survival (HR=0.862, p=0.165) compared to placebo in patients with renal cell carcinoma (J Clin Oncol 35, 2017 (suppl; abstr 4507)). detail...
Unknown unknown renal cell carcinoma not applicable Pazopanib FDA approved Actionable In a Phase III trial that supported FDA approval, Votrient (pazopanib) improved progression free survival in patients with advanced renal cell carcinoma (PMID: 20100962). detail... 20100962
Unknown unknown thyroid medullary carcinoma not applicable Vandetanib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Caprelsa (vandetanib) resulted in improved progression-free survival, an objective response rate of 45%, and a disease control rate of 87% in patients with medullary thyroid carcinoma (PMID: 22025146, PMID: 22723734). detail... 22723734 22025146
Unknown unknown dermatofibrosarcoma protuberans not applicable Imatinib FDA approved Actionable In a Phase II clinical trial that supported FDA approval, treatment with Gleevec (imatinib) resulted in a median time-to-progression of 23.9 months, and complete response in 33% (4/12) and partial response in 50% (6/12) of patients with dermatofibrosarcoma protuberans (PMID: 18451237). 18451237 detail...
Unknown unknown chronic myeloid leukemia not applicable Ponatinib FDA approved Actionable In a Phase II clinical trial which supported FDA approval, Iclusig (ponatinib) was effective in promoting disease regression in 52% of patients with accelerated phase chronic myeloid leukemia, 31% of patients with blast phase chronic myeloid leukemia, and 41% of patients with Philadelphia chromosome positive acute lymphoblastic leukemia (PMID: 23935038). detail... 23935038
Unknown unknown chronic myeloid leukemia not applicable Ponatinib Clinical Study Actionable In a meta-analysis, Iclusig (ponatinib) treatment was associated with increased rate of major molecular response compared with Gleevec (imatinib) (Odds Ratio (OR): 4.95 [0.97-25.19]), but not improved overall survival (OR: 2.00 [0.21-19.33]), and was associated with increased risk of vascular occlusive events (OR: 3.47 [1.23-9.78]) in patients with chronic myeloid leukemia (PMID: 26847662). 26847662
Unknown unknown colorectal cancer not applicable Regorafenib FDA approved Actionable In a Phase III clinical trial (CORRECT) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved overall survival compared to placebo (6.4 vs 5.0 months, HR=0.77, p=0.0052) in patients with refractory metastatic colorectal cancer (PMID: 23177514; NCT01103323). 23177514 detail...
Unknown unknown colorectal cancer not applicable Regorafenib Phase III Actionable In a Phase III trial (CONSIGN), Stivarga (regorafenib) treatment demonstrated safety profile and efficacy consistent with previous studies, median progression-free survival (PFS) was 2.7 months overall, 2.8 months in KRAS wild-type, and 2.5 months in KRAS mutant colorectal cancer patients, and with no difference in KRAS status between long and short PFS groups (PMID: 30190299; NCT01538680). 30190299
Unknown unknown gastroesophageal junction adenocarcinoma not applicable Ramucirumab FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Cyramza (ramucirumab) increased progression-free and overall survival in patients with gastroesophageal junction adenocarcinoma (PMID: 24094768). detail... 24094768
Unknown unknown colorectal cancer not applicable Ramucirumab + FOLFIRI FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with a combination of Cyramza (ramucirumab) and the FOLFIRI chemotherapy regimen improved median progression free survival to 5.7 months and overall survival to 11.7 months in patients with metastatic colorectal cancer (PMID: 25877855). 25877855 detail...
Unknown unknown gastrointestinal stromal tumor not applicable Regorafenib FDA approved Actionable In a Phase III clinical trial (GRID) that supported FDA approval, Stivarga (regorafenib) demonstrated safety and improved progression free survival compared to placebo (4.8 vs 0.9 months, HR=0.27, p<0.0001) in patients with gastrointestinal stromal tumors (PMID: 23177515; NCT01271712). detail... 23177515
Unknown unknown Indication other than cancer not applicable Fingolimod FDA approved Actionable Gilenya (fingolimod) is FDA approved for use in patients with relapsing forms of multiple sclerosis (FDA.gov). detail...
KIT D816V Indication other than cancer resistant Imatinib FDA approved Actionable Gleevec (imatinib) is not indicated for patients with aggressive systemic mastocytosis harboring KIT D816V (FDA.gov). detail...
Unknown unknown colorectal cancer not applicable Panitumumab FDA approved Actionable In an open-label clinical trial that supported FDA approval, Vectibix (panitumumab) treatment in patients with metastatic colorectal cancer resulted in increased progression-free survival (13.8 weeks; SD=0.76) compared to best supportive care alone (8.5 weeks; SD=0.54) (PMID: 18316547). 18316547 detail...
Unknown unknown hepatocellular carcinoma not applicable Sorafenib FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with Nexavar (sorafenib) improved median progression free survival to 10.7 months in patients with unresectable hepatocellular carcinoma (PMID: 19144678). 19144678 detail...
Unknown unknown non-small cell lung carcinoma sensitive Bevacizumab-awwb + Carboplatin + Paclitaxel FDA approved Actionable In a Phase III trial that supported FDA approval, Mvasi (bevacizumab-awwb) treatment resulted in an objective response rate (ORR) of 39% (128/328) similar to the ORR of Avastin (bevacizumab), 41.7% (131/314), in non-squamous NSCLC patients receiving Carboplatin and Paclitaxel as first line therapy, and was pharmacokinetically similar to Avastin (bevacizumab) further supporting extrapolation to other indications (J Clin Oncol 34, 15_sup (2016) 9095, J Clin Oncol 35, 15_sup (2017) 9050, FDA.gov; NCT01966003). detail... detail... detail...
Unknown unknown non-small cell lung carcinoma not applicable Ramucirumab + Docetaxel FDA approved Actionable In a Phase III trial that supported FDA approval, treatment with a combination of Cyramza (ramucirumab) and Taxotere (docetaxel) resulted in improved overall survival compared to treatment with Taxotere (docetaxel) and a placebo in patients with metastatic non-small cell lung cancer (PMID: 24933332). detail... 24933332
Unknown unknown renal cell carcinoma not applicable Sunitinib Phase III Actionable In a Phase III clinical trial, treatment with Sutent (sunitinib) resulted in prolonged disease free survival (HR = 0.761) compared to placebo in post-nephrectomy patients with renal cell carcinoma (ESMO 2016 Congress in Copenhagen, Abstract LBA11_PR). detail...
Unknown unknown renal cell carcinoma not applicable Sunitinib FDA approved Actionable In a Phase III clinical trial that supported FDA approval, treatment with Sutent (sunitinib) resulted in improved median progression free survival (47.3 weeks) and objective response rate (27.5%) in patients with renal cell carcinoma (PMID: 19707433). 19707433 detail...