New Drugs for Cancer

The long development process of lab screening and animal tests is expensive and weeds out most compounds. Only about 0.1% of potential medicines eventually make it to clinical trials in human subjects. Nevertheless, the pharmaceutical industry continues to produce new oncology products. Hundreds are in the drug development pipeline.  New drugs approved by the FDA are sometimes combinations of old drugs or incorporate new delivery methods to get the active medicine into the body.  On this page we cover New Molecular Entities, which are truly new at the molecular level, and include both small molecule drugs and biologics.

MedicineTrade NameCategoryTargetManufacturer
2022
Lutetium (177Lu) vipivotide tetraxetanPluvictoradio-immunotherapy conjugateprostate cancerNovartis
Nivolumab/relatlimabOpdualagmonoclonal antibodymelanomaBristol Myers Squibb
PacritinibVonjokinase inhibitormyelofibrosisCTI Biopharma
Tebentafusp-tebnKimmtrakmonoclonal antibodyuveal melanomaImmunocore
2021
AsciminibScemblixkinase inhibitorleukemiaNovartis
Tisotumab vedotin-tftvTivdakMonoclonal Antibodycervical cancerSeagen
MobocertinibExkivitykinase inhibitorlung cancerTakeda
InfigratinibTruseltiqkinase inhibitorcholangiocarcinomaQED Therapeutics
Amivantamab-vmjwRybrevantMonoclonal Antibodynon-small cell lung cancerJanssen Pharmaceutical
SotorasibLumakrasKinase Inhibitornon-small cell lung cancerAmgen
Loncastuximab tesirine-lpylZynlontaAntibody ConjugatelymphomaADC Therapeutics
Dostarlimab-gxlyJemperliMonoclonal Antibodyendometrial cancerGlaxo SmithKline
TivozanibFotivdakinase inhibitorrenal cell carcinomaAveo Pharmaceuticals
Melphalan flufenamidePepaxtoalkylating agentmyelomaOncopeptides AB
UmbralisibUkoniqPI3K inhibitorlymphomaTG Therapeurtics
TepotinibTepmetkokinase inhibitornon-small cell lung cancerEMD Serono (Merck)

In recent years the FDA has approved on average 13 new molecular entities (NME) per year for cancer treatment. Additional new therapies include combinations of existing drugs and new delivery methods for existing drugs.  About a quarter of the new drugs approved by the FDA in the past decade are for cancer treatment.

The nature of the newer drugs differs from the chemotherapy medicines of decades ago.  Most new NMEs deliver targeted therapy; they would not even be recognizable as chemotherapy to people in the 1960s.  Even today, the public thinks of chemotherapy as a treatment with severe side effects.  While “old-style” chemo treatment do indeed cause serious side effects, targeted therapies tend to be easier on the body.  Of the 80 NMEs for cancer approved from 2015 to 2020:

Monoclonal Antibodies (unconjugated)19 NMEs
Antibody-drug Conjugates7
Small molecule kinase inhibitors27
Other signal induction inhibitors15
Alkylating agents2
Other10

 

How New are the New Drugs?

Most new drugs approved by the FDA aren’t totally new. They are variations or combinations of old drugs – new methods of delivery, etc. A survey of new drugs approved from 1989 to 2000 found only 35% were new molecular entities (NMEs). The rest were combinations with other drugs or differed from previously approved medicines in method of administration or dosage. The 1984 Hatch-Waxman Act allows the FDA to give market exclusivity to new medicines which are not NMEs and therefore cannot be patented.

New Immunotherapy

In recent years, development of new immunotherapy agents has revolutionized cancer treatment. These drugs induce or potentiate the anti-tumor activity of the immune system.

The immune system identifies tumors and triggers an anti-tumor response. Such response, however, is modulated by stimulating and inhibiting factors. The new immunomodulating agents work by slowing down the action of natural immune system inhibitors and by speeding up natural stimulatory factors. The novelty of this approach stems from the fact that the drug targets the host immune cells and not the tumor itself. Antibody-drug conjugates combine an antibody and a toxin to target malignant cells. Following therapeutic and commercial successes, a lot of efforts are being put in the development of new ADCs.

More on immnotherapy.

See more on notch inhibitors.

Near-term future of chemotherapy

Gene Therapy

Gene therapy has been talked about by futurists for decades and only in the past few years have appeared in the clinic. The idea is that by changing the DNA in the body’s cells, the genetic cause of illness can be addressed. Strategies include inactivating a bad gene, changing a mutated gene to a healthy gene, and putting a new gene in the body to fight disease. Researchers are making progress in employing gene therapy against sickle cell anemia, connective tissue disorders, and retinal disease. Some gene therapy for cancer is have entered clinical use. The most advanced area in anti-cancer gene therapy is CAR-T therapies, chimeric antigen receptor T-cell, which involves genetic modification of T-cells removed from the patient and then re-introduced to the body. In 2017 the FDA approved tisagenlecleucel and axicabtagene ciloleucel which are CAR-T processes, and in 2020 brexucabtagene autoleucel was approved.

 

Recently Approved Oncology Drugs

Oncology drugs (chemotherapy and immunotherapy agent) approved in recent years:

2020
MedicineTrade NameCategoryTargetManufacturer
AvapritinibAyvakitkinase inhibitorgastrointestinal stromal tumorBlueprint Medicines
TazemetostatTazverikenzyme inhibitorepithelioid sarcomaEpizyme
IsatuximabSarclisaMonoclonal Antibodymultiple myelomaSanofi
TucatinibTukysakinase inhibitorbreast cancerCascadian Therapeutics
PemigatinibPemazyrekinase inhibitorcholangio-carcinomaIncyte
Sacituzumab govitecan-hziyTrodelvyTopoisomerase inhibitorbreast cancerImmunomedics
CapmatinibTabrectakinase inhibitornon small-cell lung cancerNovartis
SelpercatinibRetevmokinase inhibitorthyroid cancer, lung cancerLoco Oncology (Eli Lilly)
RipretinibQinlockkinase inhibitorgastrointestinal stromal tumorsDeciphera Pharmaceuticals
LurbinectedinZepzelcaoncogenic transcription inhibitorsmall-cell lung cancerJazz and PharmaMar
PralsetinibGavretokinase inhibitornon small-cell lung cancerBlueprint Medicines
Belantamab mafodotin-blmfBlenrepmonoclonal antibodymultiple myelomaGlaxoSmith Kline
Tafasitamab-cxixMonjuvimonoclonal antibodydiffuse large B-cell lymphomaMorphosus US
NaxitamabDanyelzamonoclonal antibodyneuroblastomaY-mAbs Therapeutics
Brexucabtagene autoleucelTecartusCAR-T therapymantle cell lymphomaKite Pharma
MargetuximabMargenzaMonoclonal AntibodyHER2+ breast cancerMacrogenics
RelugolixOrgovyxhormone suppressantprostate cancerMyovant Sciences

2019
MedicineTrade NameCategoryTargetManufacturer
ErdafitinibBalversakinase inhibitorbladder cancerJanssen Biotech
AlpelisibPiqrayPI3K inhibitorbreast cancerNovartis
Polatuzumab vedotinPolivyantibody conjugatediffuse large B-cell lymphomaGenentech
SelinexorXpovioselective inhibitor of nuclear exportmultiple myelomaKaryopharm Therapeutics
DarolutamideNubeqandrogen receptor antagonistcastration resistant prostate cancerBayer
EntrectinibRozlytrekkinase inhibitorNTRK-defect tumors; tissue-agnostic oncology drugGenentech
ZanubrutinibBrukinsaBTK inhibitormantle cell lymphomaBeigene
Enfortumab vedotin-ejfvPadcevAntibody-drug conjugatebladder cancerAstellas
Fam-trastuzumab deruxtecan-nxkiEnhertuAntibody-drug conjugatebreast cancerDaiichi Sankyo

MedicineTrade NameCategoryTargetManufacturer
2018
RibociclibKisqalikinase inhibitorbreast cancerNovartis
ApalutamideErleadahormone suppressantprostate cancerJanssen Biotech
Lutetium Lu 177 dotatateLutatheraradio-immunotherapy conjugategastro-enteropancreatic neuroendocrine tumorsAAA USA, Inc
BinimetinibMektovikinase inhibitormetastatic melanomaArray Biopharma
EncorafenibBraftovikinase inhibitormetastatic melanomaArray Biopharma
IvosidenibTibsovoIDH1 inhibitoracute myeloid leukemiaAgios Pharmaceuticals
MogamulizumabPoteligeoMonoclonal Antibodynon-Hodgkin lymphomaKyowa Kirin
Moxetumomab pasudotox-tdfkLumoxitiMonoclonal Antibodyhairy cell leukemiaInnate Pharma
DuvelisibCopiktraPI3K inhibitorleukemia and lymphomaSecura
DacomitinibVizimprokinase inhibitornon-small-cell lung cancerPfizer
Cemiplimab-rwlcLibtayoMonoclonal antibodysquamous cell carcinomaRegeneron Pharmaceuticals
TalazoparibTalzennaPARP Inhibitorbreast cancerPfizer
LorlatinibLorbrenakinase inhibitornon-small cell lung cancerPfizer
GlasdegibDaurismoHedgehog pathway inhibitoracute myeloid leukemiaPfizer
LarotrectinibVitrakvikinase inhibitorsolid tumors (only the second tissue-agnostic oncology drug approved by FDA)Pfizer
GilteritinibXospatakinase inhibitoracute myeloid leukemiaAstellas
Calaspargase pegol-mknAsparlasenzymeacute lymphoblastic leukemiaServier Pharmaceuticals
Tagraxofusp-erzsElzonrisconjugate of interferon and toxinblastic plasmacytoid dendritic cell neoplasmStemline Therapeutics

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MedicineTrade NameCategoryTargetManufacturer
2017
MidostaurinRydaptkinase inhibitorleukemiaNovartis
BrigatinibAlunbrigkinase inhibitornon-small cell lung cancerTakeda
TisagenlecleucelKymriahCAR T-Cell therapylymphoma and leukemiaNovartis
AxicabtageneYescartaCAR T-Cell therapylymphomaKite Pharma
Gemtuzumab ozogamicinMylotargconjugateacute myeloid leukemiaPfizer
AvelumabBavenciomonoclonal antibodymetastatic Merkel cell carcinomaEMD Serono (Merck)
NiraparibZejulaPARP inhibitorepithelial ovarian, fallopian tube or primary peritoneal cancersGlaxo Smith Kline
DurvalumabImfinziMonoclonal Antibodyurothelial carcinomaAstraZeneca
Neratinib maleateIdhifaKinase inhibitoracute myeloid leukemiaPuma Biotech
Inotuzumab ozogamicinBesponsaAntibody drug conjugateacute lymphoblastic leukemiaWyeth Pharmaceuticals
CopanlisibAliqopaPI3K inhibitorfollicular lymphomaBayer Healthcare
EnasidenibIdhifaIDH2 inhibitoracute myeloid leukemiaCelgene
AbemaciclibVerzeniokinase inhibitormetastatic breast cancersEli Lilly
AcalabrutinibCalquencekinase inhibitormantle cell lymphomaAstraZeneca
2016
MedicineTrade NameCategoryTargetManufacturer
VenetoclaxVenclextaBCL-2 Inhibitorchronic lymphocytic leukemiaAbbVie
AtezolizumabTecentriqmonoclonal antibodyurothelial carcinomaGenentech
OlaratumabLartruvomonoclonal antibodysoft tissue sarcomaEli Lilly
RucaparibRubracaPARP inhibitorovarian cancerClovis Oncology

2015
MedicineTrade NameCategoryTargetManufacturer
IxazomibNinlaroproteosome inhibitormyelomaTakeda
Talimogene LaherparepvecImlygicimmunotherapymelanomaAmgen
PalbociclibIbrancekinase inhibitormetastatic breast cancerPfizer
LenvatinibLenvimakinase inhibitordifferentiated thyroid cancerEisai
PanobinostatFarydakHDAC inhibitormultiple myelomaSecura
DinutuximabUnituxinMonoclonal antibodyneuroblastomaUnited Therapeutics
SonidegibOdomzoHedgehog pathway inhibitorlocally advanced basal cell carcinomaSun Pharma
Trifluridine and tipiracilLonsurfthymidine phosphorylase inhibitor (antimetabolitecolorectal cancerTaiho Oncology
TrabectedinYondelisalkylating agentliposarcoma and leiomyosarcomaJanssen Pharmaceuticals
CobimetinibCotellickinase inhibitoradvanced melanomaGenentech
OsimertinibTagrissokinase inhibitornon-small cell lung cancerAstraZeneca
DaratumumabDarzalexmonoclonal antibodymultiple myelomaJanssen Biotech
IxazomibNinlaroProteasome inhibitormultiple myelomaMillenium Pharmaceuticals
NecitumumabPortrazzamonoclonal antibodysquamous non-small cell lung cancerEli Lilly
ElotuzumabEmplicitimonoclonal antibodymultiple myelomaBristol Myers Squibb
AlectinibAlecensakinase inhibitorALK-positive lung cancerHoffman LaRoche

Drugs currently approved by the FDA for cancer by year of approval:

Time periodNumber new drugs
Before 19607
1960s10
1970s11
1980s13
1990s30
2000-200935
2010-2019103
202016
202112