TABLE 1

Examples of United States FDA-approved cancer drugs that were developed from hit compounds identified in small-molecule screens

Wherever possible, the target of the small-molecule screen and assay type are indicated.

DrugDate ApprovedUS Brand NameApproved Use in CancerTarget of Small-Molecule ScreenAssay TypeReference(s)
Alectinib12/11/2015AlecensaNon-small cell lung cancerAnaplastic lymphoma kinase (ALK)Not availableSakamoto et al. (2011)
Ixazomib Citrate11/20/2015NinlaroMultiple myeloma20S proteasomeNot availableKupperman et al. (2010)
Sonidegib7/24/2015OdomzoBasal cell carcinomaHedgehog (Hh) signalingCell-based: Hh-responsive Gli-luciferase reporter assay in the TM3 mouse cell linePan et al. (2010)
Olaparib12/19/2014LynparzaOvarian cancerPoly(ADP-ribose) polymerase- 1 Biochemical: FlashPlate scintillation proximity assay Dillon et al. (2003), Loh et al. (2005), Menear et al. (2008)
Ceritinib4/29/2014ZykadiaNon-small cell lung cancerAnaplastic lymphoma kinaseCell-based: selective cytotoxicity of Ba/F3 NPM-ALK vs. nontransformed parental Ba/F3 cellsGalkin et al. (2007)
Ibrutinib11/13/2013ImbruvicaChronic lymphocytic leukemia, mantle cell lymphoma, Waldenstrom macroglobulinemiaBruton’s tyrosine kinaseBiochemical: Forster resonance energy transfer enzyme assayPan et al. (2007), Honigberg et al. (2010)
Trametinib5/29/2013MekinistMelanomaCyclin-dependent kinase 4 inhibitor B (p15INK4b)Cell-based: branched-DNA assay with p15INK4b-specific probes in HT-29 cellsYamaguchi et al. (2007), Abe et al. (2011)
Enzalutamide8/31/2012XTANDIProstate cancerAndrogen receptorCell-based: human prostate cancer cellsTran et al. (2009)
Vismodegib1/30/2012ErivedgeBasal cell carcinomaHh signalingCell-based: Hh-responsive Gli8x-luciferase reporter assay in C3H/10T1/2 cellsWilliams et al. (2003), Romer et al. (2004), Zhang et al. (2009)
Lapatinib3/13/2007TykerbBreast cancerEpidermal growth factor receptorNot availableFry et al. (1994), Macarron et al. (2011)
Dasatinib6/28/2006SprycelAcute lymphoblastic leukemia, chronic myelogenous leukemiaLymphocyte-specific protein tyrosine kinaseBiochemical: filter-binding assayPadmanabha et al. (1998), Das et al. (2006)
Sorafenib12/20/2005NexavarHepatocellular carcinoma, renal cell carcinoma, thyroid cancerRaf1 (C-Raf) kinaseBiochemical: scintillation proximity assayWilhelm et al. (2006)
Erlotinib11/18/2004TarcevaNon-small cell lung cancer, pancreatic cancerEpidermal growth factor receptorNot availableFry et al. (1994), Macarron et al. (2011)
Gefitinib5/5/2003IressaNon-small cell lung cancerEpidermal growth factor receptorNot availableFry et al. (1994), Macarron et al. (2011)
Zoledronic  acid8/20/2001ZometaMultiple myeloma, hypercalcemiaNot availableNot availableGreen et al. (1994)