Table 2.
New human drugs with novel mechanisms of action, approved since the DrugCentral 2021 release
| Drug name(s) | Targeta | Target classb | Agency | Indication(s)c |
|---|---|---|---|---|
| inqovi | CDA | Enzyme | FDA | Myelodysplastic syndrome, CMML |
| lonafarnib | FNTA,B | Enzyme | FDA | Hutchinson-Gilford syndrome |
| umbralisib | CSNK1E | Kinase | FDA | Marginal zone & follicular lymphoma |
| fosdenopterin | MOCS1 | Enzyme | FDA | Molybdenum cofactor deficiency A |
| sotorasib | KRAS | Enzyme | FDA, EMA, PMDA | KRAS G12C-mutated locally NSCLC |
| ibrexafungerp | FKS1 | Enzyme | FDA | Vulvovaginal candidiasis |
| belzutifan | EPAS1 | TF | FDA | von Hippel-Lindau disease |
| avacopan | C5AR1 | GPCR | FDA, PMDA, EMA | ANCA-associated vasculitis, microscopic polyangiitis, and granulomatosis with polyangiitis |
| maribavir | UL97 | Kinase | FDA, EMA | Post-transplant CMV infection |
| pafolacianine | FOLR1 | MR | FDA | Imaging ovarian cancer & malignant lesions |
| mitapivat | PKLR | Enzyme | FDA | PK deficiency |
| mavacamten | MYH7 | Other | FDA | Obstructive HCM |
| gefapixant | P2RX3 | Ion channel | PMDA | Refractory chronic cough |
| tapinarof | AHR | TF | FDA | Plaque psoriasis |
| bulevirtide | SLC10A1 | Transporter | EMA | Chronic HBV with delta hepatitis |
| pegcetacoplan | C3 | Macroglobulin | FDA, EMA | Paroxysmal nocturnal hemoglobinuria |
| vosoritide | NPR2 | Enzyme | EMA, FDA | Achondroplasia |
| tirzepatide | GPR119 | GPCR | FDA | Diabetes mellitus type 2 |
| ansuvimab, atoltivimab, maftivimab, odesivimab | EBOV GP | Glycoprotein | FDA | Ebola virus disease |
| evinacumab | ANGPTL3 | Secreted | FDA, EMA | HoFH |
| bamlanivimab, etesevimab, casirivimab, imdevimab, regdanvimab, sotrovimab | S | Glycoprotein | FDA, EMA PMDA, | COVID-19 infection |
| remdesivir | RdRp | RNA polymerase | FDA, EMA PMDA, | COVID-19 infection |
| aducanumab | APP | Unclassified | FDA | Alzheimer's disease |
| bimekizumab | IL17F | Cytokine | EMA, PMDA | Plaque, erythrodermic and pustular psoriasis |
| tralokinumab | IL13 | Cytokine | EMA, FDA | Moderate to severe atopic dermatitis |
| tezepelumab | TSLP | Cytokine | FDA | Severe asthma |
| sutimlimab | C1S | Enzyme | FDA | Cold autoimmune hemolytic anemia |
| relatlimab | LAG3 | TAA | FDA | Unresectable or metastatic melanoma |
| faricimab | ANGPT2 | Secreted | FDA | EMD and diabetic macular edema |
| efgartigimod alfa | FCGRT | MR | FDA, EMA | Immune thrombocytopenia, and MG |
| sacituzumab govitecan | TACSTD2 | TAA | FDA, EMA | Triple negative breast neoplasms |
| belantamab mafodotin | TNFRSF17 | TAA | FDA, EMA | Multiple myeloma |
| tisotumab vedotin | F3 | MR | FDA | Recurrent or metastatic cervical cancer |
| imlifidase | IGHG1-4 | Antibody | EMA | Renal transplant |
| pabinafusp alfa | TFRC | MR | PMDA | Mucopolysaccharidosis type II |
| tebentafusp | HLA-A | Surface antigen | FDA, EMA | Unresectable metastatic uveal melanoma |
| lumasiran | HAO1 | RNA | FDA, EMA | Primary hyperoxaluria,type I |
| flortaucipir F 18 | MAPT | Structural | FDA | Positron emission tomography |
| borofalan (10B) | SLC7A5 | Transporter | PMDA | Neoplasm of head and neck |
| gallium (68Ga) gozetotide, lutetium (177Lu) vipivotide tetraxetan | KLK3 | Surface antigen | FDA | Positron emission tomography, PSMA-positive metastatic castration-resistant prostate cancer |
| piflufolastat F-18 | FOLH1 | Enzyme | FDA | Positron emission tomography |
aAHR, aryl hydrocarbon receptor; ANGPT2, angiopoietin-2; ANGPTL3, angiopoietin-related protein 3; APP, amyloid beta A4 precursor protein; C1S, complement C1s subcomponent; C3, complement C3; C5AR1, C5a anaphylatoxin chemotactic receptor 1; CDA, cytidine deaminase; CSNK1E, casein kinase I isoform epsilon; EPAS1, endothelial PAS domain-containing protein 1; F3, tissue factor; FCGRT, IgG receptor FcRn large subunit p51; FKS1, 1,3-beta-d-glucan-UDP glucosyltransferase; FNTA, protein farnesyltransferase/geranylgeranyltransferase type-1 subunit alpha; FNTB, protein farnesyltransferase subunit beta; FOLH1, glutamate carboxypeptidase 2; FOLR1, folate receptor alpha; EBOV GP, Ebola virus envelope glycoprotein; GPR119, glucose-dependent insulinotropic receptor; HAO1, hydroxyacid oxidase 1; HLA-A, HLA class I histocompatibility antigen, A-3 alpha chain; IGHG1, Ig gamma-1 chain C region; IGHG2, Ig gamma-2 chain C region; IGHG3, Ig gamma-3 chain C region; IGHG4, Ig gamma-4 chain C region; IL13, interleukin-13; IL17F, interleukin-17F; KLK3, prostate-specific antigen; KRAS, GTPase KRas; LAG3, lymphocyte activation gene 3 protein; MAPT, microtubule-associated protein tau; MOCS1, molybdenum cofactor biosynthesis protein 1; MYH7, Myosin-7; NPR2, atrial natriuretic peptide receptor 2; P2RX3, P2X purinoceptor 3; PKLR, pyruvate kinase PKLR; S, spike glycoprotein of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2); SLC10A1, sodium/bile acid cotransporter; SLC7A5, large neutral amino acids transporter small subunit 1; RdRp, SARS-CoV-2 RNA-dependent RNA polymerase; TACSTD2, tumor-associated calcium signal transducer 2; TFRC, transferrin receptor protein 1; TNFRSF17, tumor necrosis factor receptor superfamily member 17; TSLP, thymic stromal lymphopoietin; UL97, serine/threonine protein kinase UL97.
bMR, membrane receptor; TAA, tumour-associated antigen; TF, transcription factor.
cANCA, anti-neutrophil cytoplasmic autoantibody; CMML, chronic myelomonocytic leukemia; CMV, cytomegalovirus infection; HBV, viral hepatitis B infection; HCM, hypertrophic cardiomyopathy; HoFH, homozygous familial hypercholesterolemia; NSCLC, non-small cell lung cancer; PK, pyruvate kinase.