Table 1.

FDA oncology drug approvals (January 2002–December 2012)

ProductApproval dateApproval typeaIndication(s)Primary endpointAA?Single-arm vs. randomized trialTrial size
Imatinibb2/1/2002SFirst-line GISTRRYesRandomized74
12/20/2002SFirst-line Ph+ CMLPFSYesRandomized1,106
5/20/2003SSecond-line pediatric Ph+ CMLRRYesExtrapolated from 2 single-arm studies39
9/27/2006SFirst-line pediatric Ph+ CMLRRYesSingle-arm51
10/19/2006SDermafibrosarcoma protuberansRRNoSingle-arm12
10/19/2006SMyelodysplastic syndromeRRNoSingle-arm7
10/19/2006SAdult Ph+ ALLRRNoSingle-arm48
10/19/2006SAdult aggressive systemic mastocytosisRRNoSingle-arm5
10/19/2006SHypereosinophilic syndromeRRNoSingle-arm14
12/19/2008SAdjuvant therapy for GISTDFSYesRandomized713
Ibritumomab2/19/2002IRelapsed follicular lymphomaRRYesRandomized143
9/3/2009SFirst-line NHLPFSNoRandomized414
Fulvestrant4/25/2002ISecond-line breast cancerTTPNo2 Randomized400; 451
Oxaliplatin8/9/2002ISecond-line metastatic CRCRRYesRandomized463
1/9/2004SFirst-line advanced CRCOSNoRandomized531
11/4/2004SAdjuvant stage III CRCDFSNoRandomized2,246
Anastrozoleb9/5/2002SAdjuvant HER+ breast cancerDFSYesRandomized9,366
Docetaxelb11/27/2002SNSCLC combination therapyOSNoRandomized1,218
5/19/2004SMetastatic prostate cancerOSNoRandomized1,006
8/18/2004SAdjuvant node + breast cancerDFSNoRandomized1,491
3/22/2006SGastric cancerOSNoRandomized457
10/17/2006SInoperable SCCHNPFSNoRandomized358
9/28/2007SInduction treatment of SCCHNOSNoRandomized501
Gefitinib5/5/2003IcThird-line NSCLCRRYesSingle-arm142
Bortezomib5/13/2003IThird-line multiple myelomaRRYesSingle-arm202
3/25/2005SSecond-line multiple myelomaOSNoRandomized669
12/8/2006SSecond-line mantle cell lymphomaRRNoSingle-arm155
6/20/2008SFirst-line multiple myelomaTTPNoRandomized682
Tositumomab6/27/2003IcRelapsed NHLRRNo2 Single-arm40; 60
12/22/2004SRefractory low-grade lymphomaRRYesSingle-arm60
Pemetrexed2/4/2004IMalignant pleural mesotheliomaOSNoRandomized456
8/19/2004ScSecond-line NSCLCRRYesRandomized571
9/26/2008SFirst-line NSCLC combination therapyRRYesRandomized1,725
7/2/2009SMaintenance NSCLCOSNoRandomized663
Cetuximab2/12/2004ISingle agent for second-line CRCRRYes1 Randomized, 1 Single-arm329; 57
2/12/2004ISecond-line CRC combination therapyRRYes1 Randomized, 1 Single-arm329; 138
3/1/2006SSCCHN combination therapyOSNoRandomized424
3/1/2006SSecond-line SCCHN as single agentRRNoSingle-arm103
11/7/2011SFirst-line SCCHN combination therapyOSNoRandomized442
7/6/2012SFirst-line mCRCOSNo3 Randomized1,217; 453; 315
Bevacizumab2/26/2004IFirst-line mCRCOSNoRandomized813
6/20/2006SSecond-line mCRCOSNoRandomized829
10/11/2006SFirst-line NSCLCOSNoRandomized878
2/22/2008SdFirst-line HER2breast cancerPFSYesRandomized712
5/5/2009ScGlioblastomaRRYes2 Single-arm85; 56
7/31/2009SMetastatic RCCPFSNoRandomized649
Gemcitabineb5/19/2004SFirst-line metastatic breast cancer combination therapyTTPNoRandomized529
7/14/2006SdSecond-line ovarian cancer combination therapyPFSNoRandomized356
Azacitidine5/19/2004IMyelodysplastic syndromeRRNo1 Randomized, 2 Single-arm191; 120
Letrozoleb10/29/2004SExtended adjuvant breast cancerDFSYesRandomized5,187
12/28/2005SAdjuvant breast cancerDFSYesRandomized8,000+
Erlotinib11/18/2004ISecond-line NSCLCOSNoRandomized731
11/2/2005ScMetastatic pancreatic cancerOSNoRandomized569
4/16/2010SdMaintenance therapy for NSCLCOSNoRandomized889
Clofarabine12/28/2004IcRelapsed pediatric ALLRRYesSingle-arm49
Paclitaxel1/7/2005ISecond-line breast cancerRRNoRandomized460
10/11/2012SFirst-line locally advanced NSCLCRRNoRandomized1,052
Nelarabine10/28/2005IcT-cell ALL or T-cell lymphoblastic lymphomaRRYes2 Single-arm39; 28
Sorafenib12/20/2005IAdvanced RCCPFSNoRandomized769
11/16/2007SHepatocellular carcinomaOSNoRandomized602
Lenalidomide12/27/2005IcMyelodysplastic syndromesRRNoSingle-arm148
6/29/2006SSecond-line multiple myelomaTTPNo2 Randomized341; 351
Pegaspargaseb7/24/2006SFirst-line ALLDRNoRandomized118
Topotecanb6/14/2006SCarcinoma of the cervixOSNoRandomized293
Rituximabb2/10/2006SDiffuse large B-cell, CD20+, NHLOSNo3 Randomized632; 399; 823
9/29/2006SNHL combination therapyPFSNoRandomized322
9/29/2006SNHL following chemotherapyPFSNoRandomized322
2/18/2010SFirst-line CLL combination therapyPFSNo2 Randomized817; 522
1/28/2011SMaintenance therapy for NHLPFSNoRandomized1,018
Thalidomideb5/26/2006SMultiple myelomaORRYesRandomized207
Trastuzumabb11/16/2006SAdjuvant node+ breast cancerDFSNo2 Randomized(Total) 3,752
10/20/2010SAdenocarcinomaOSNoRandomized594
Vorinostat1/6/2006IThird-line CTCLRRNoSingle-arm74
Sunitinib1/26/2006ISecond-line GISTTTPNoRandomized312
1/26/2006IAdvanced RCCRRYesRandomized750
5/20/2011ScAdvanced pNET tumorsPFSNoRandomized171
Decitabine5/2/2006IMyelodysplastic syndromesRRNoRandomized170
Dasatinib6/28/2006IcSecond-line CMLRRYes3 Single-arm186; 107; 74
6/28/2006IcSecond-line Ph+ ALLRRNoSingle-arm78
10/28/2010SFirst-line Ph+ CMLRRYesRandomized519
Panitumumab9/27/2006ISecond-line CRCPFSYesRandomized463
Lapatinib3/13/2007ISecond-line HER2+ metastatic breast cancer combination therapyTTPNoRandomized399
1/29/2010SFirst-line HER2+ metastatic breast cancer combination therapyPFSYesRandomized1,286
Doxorubicinb5/17/2007SMultiple myeloma combination therapyTTPNoRandomized646
Temsirolumus5/30/2007IAdvanced RCCOSNoRandomized626
Ixabepilone10/16/2007ISecond-line metastatic breast cancer combination therapyPFSNoRandomized752
10/16/2007SSecond-line breast cancer monotherapyRRNoSingle-arm126
Nilotinib10/29/2007ISecond-line Ph+ CMLRRYesSingle-arm105
6/17/2010SNewly diagnosed Ph+ CMLRRYesRandomized846
Bendamustine3/20/2008ISecond-line CLLPFSNoRandomized301
10/31/2008SIndolent B-cell NHLRRNoSingle-arm100
Fludarabine12/18/2008ISecond-line B-cell CLLRRYesSingle-arm78
Degarelix12/24/2008IAdvanced prostate cancerDRNoRandomized620
Everolimus3/30/2009ISecond-line advanced RCCPFSNoRandomized416
10/29/2010SSEGA with tuberous sclerosisRRYesSingle-arm28
5/5/2011ScpNET tumorsPFSNoRandomized410
4/26/2012SRenal angiomyolipoma with tuberous sclerosisRRYesRandomized118
7/20/2012SHER2+ breast cancerPFSNoRandomized724
Romidepsin9/5/2009IcSecond-line CTCLRRNo2 Single-arm96; 71
6/16/2011SPTCLRRYesSingle-arm130
Eribulin11/15/2010IThird-line metastatic breast cancerOSNoRandomized762
Pralatrexate9/24/2009IcRelapsed or refractory PTCLRRYesSingle-arm115
Pazopanib10/19/2009IcAdvanced RCCPFSNoRandomized435
4/26/2012ScSecond-line soft-tissue sarcomaPFSNoRandomized369
Ofatumumab10/26/2009IcRefractory CLLRRYesSingle-arm154
Cabazitaxel6/17/2010ISecond-line prostate cancerOSNoRandomized755
Ipilimumab3/25/2011IMelanomaOSNoRandomized676
Peginterferon alfa-2bb3/29/2011ScMelanomaDFSNoRandomized1,256
Vandetanib4/6/2011IeMedullary thyroid cancerPFSNoRandomized331
Abiraterone4/28/2011ISecond-line prostate cancerOSNoRandomized1,195
12/10/2012SMetastatic prostate cancerPFSNoRandomized1,088
Vemurafenib8/17/2011IMelanoma with BRAF mutationOSNoRandomized675
Brentuximab8/19/2011IcThird-line Hodgkin lymphomaRRYesSingle-arm102
8/19/2011IcSecond-line ALCLRRYesSingle-arm58
Crizotinib8/26/2011INSCLC ALK+RRYes2 Single-arm136; 119
Asparaginase11/18/2011IALL combination therapyDRNoSingle-arm58
Axitinib1/27/2012IcSecond-line RCCPFSNoRandomized723
Vismodegib1/30/2012IMetastatic basal cell carcinomaRRNoSingle-arm104
Pertuzumab6/8/2012IHER2+ metastatic breast cancerPFSNoRandomized808
Carfilzomib7/20/2012IcRelapsed multiple myelomaRRYesSingle-arm266
Ziv-aflibercept8/3/2012ISecond-line mCRCOSNoRandomized1,226
Vincristine sulfate8/9/2012IcThird-line adult Ph ALLCRYesSingle-arm65
Enzalutamide8/31/2012ICastration-resistant prostate cancerOSNoRandomized1,199
Bosutinib9/4/2012ISecond-line Ph+ CMLRRNoSingle-arm546
Regorafenib9/27/2012IRefractory mCRCOSNoRandomized760
Omacetaxine mepesuccinate10/26/2012IfThird-line CMLRRYes2 Single-arm73; 35
Cabozantinib11/29/2012IMedullary thyroid cancerPFSNoRandomized330
Ponatinib12/14/2012ISecond-line chronic, accelerated, or blast-phase CMLRRYesSingle-arm449

Abbreviations: AA, accelerated approval; ALCL, anaplastic large-cell lymphoma; ALL, acute lymphoblastic leukemia; CLL, chronic lymphocytic leukemia; CML, chronic myelocytic leukemia; CRC, colorectal cancer; CTCL, cutaneous T-cell lymphoma; DR, durable response; NHL, non–Hodgkin lymphoma; NSCLC, non–small cell lung cancer; Ph, Philadelphia chromosome; pNET, pancreatic neuroendocrine tumors; PTCL, peripheral T-cell lymphoma; SCCHN, squamous cell carcinoma of the head and neck; SEGA, subependymal giant cell astrocytoma.

  • aI, initial approval; S, supplemental approval.

  • bInitial approval granted before January 1, 2002.

  • cODAC recommended approval.

  • dODAC did not recommend approval.

  • eODAC convened to discuss postmarketing safety studies.

  • fODAC did not recommend approval, and the drug was subsequently approved under a different new drug application.