Table 2.
New active pharmaceutical ingredients with novel mechanisms of action approved since the 2018 release of DrugCentral
| Active Ingredient(s) | Target | Target Classa | Agency | Indication |
|---|---|---|---|---|
| crizanlizumab | SELP | Adhesion | FDA | Vaso-occlusive crisis in sickle cell disease |
| luspatercept | GDF11, MSTN | Cytokine | FDA | Beta thalassemia |
| emapalumab | IFNG | Cytokine | FDA | Primary hemophagocytic lymphohistiocytosis |
| prabotulinumtoxinA | SNAP25 | Cytosolic other | FDA | Rhytidectomy of glabellar frown lines |
| botulinum toxin type A | SNAP25 | Cytosolic other | EMA | Rhytidectomy of glabellar frown lines |
| andexanet alfa | rivaroxaban, apixaban | Drug | FDA, EMA | Direct-acting anticoagulant adverse reaction |
| roxadustat | EGLN1, EGLN2, EGLN3 | Enzyme | PMDA | Anemia in chronic kidney disease, refractory anemia |
| ivosidenib | IDH1 | Enzyme | FDA | Acute myeloid leukemia |
| romosozumab | SOST | Glycoprotein | FDA | Postmenopausal osteoporosis |
| fremanezumab | CALCA | GPCR | FDA, EMA | Migraine |
| galcanezumab | CALCA | GPCR | FDA | Migraine |
| erenumab | CALCRL | GPCR | FDA, EMA | Migraine |
| ubrogepant | CALCRL | GPCR | FDA | Migraine |
| lasmiditan | HTR1F | GPCR | FDA | Migraine |
| cannabidiol | GPR55 | GPCR | FDA, EMA | Lennox-Gastaut syndrome, severe myoclonic epilepsy in infancy |
| bremelanotide | MC4R | GPCR | FDA | Lack or loss of sexual desire |
| larotrectinib | NTRK2, NTRK3 | Kinase | FDA, EMA | Malignant neoplasm |
| entrectinib | NTRK2, NTRK3, ROS1 | Kinase | FDA, PMDA | Reactive oxygen species 1 positive non-small cell lung cancer, solid neoplasm with neurotrophic receptor tyrosine kinase gene fusion |
| duvelisib | PIK3CG | Kinase | FDA | Chronic lymphoid leukemia, malignant lymphoma - small lymphocytic, follicular non-Hodgkin's lymphoma |
| lorlatinib | ROS1, LTK, FER, FES, NTRK2, NTRK3, PTK2, PTK2B, TNK2 | Kinase | FDA, EMA, PMDA | Non-small cell lung cancer lung cancer |
| fostamatinib | SYK | Kinase | FDA, EMA | Immune thrombocytopenia |
| ibalizumab | CD4 | Membrane receptor | FDA, EMA | Human immunodeficiency virus infection |
| tagraxofusp | IL3RA | Membrane receptor | FDA | Blastic plasmacytoid dendritic cell neoplasm |
| selinexor | XPO1 | Nuclear other | FDA | Relapse multiple myeloma |
| givosiran | ALAS1 | RNA | FDA, EMA | Hepatic porphyria |
| volanesorsen | APOC3 | RNA | EMA | Chylomicronemia syndrome |
| golodirsen | DMD | RNA | FDA | Duchenne muscular dystrophy |
| burosumab | FGF23 | Secreted | FDA, EMA, PMDA | Familial x-linked hypophosphatemic vitamin D refractory rickets |
| voxelotor | HBA1 | Transporter | FDA | Sickle cell disease |
| sotagliflozin | SLC5A1 | Transporter | EMA | Diabetes mellitus type 1 |
| tenapanor | SLC9A3 | Transporter | FDA | Irritable bowel syndrome characterized by constipation |
| polatuzumab vedotin | CD79B | Tumor-associated antigen | FDA, EMA | Diffuse large B-cell lymphoma refractory |
| enfortumab vedotin | NECTIN4 | Tumor-associated antigen | FDA | Metastatic urothelial carcinoma |
| caplacizumab | VWF | Unclassified | EMA, FDA | Thrombotic thrombocytopenic purpura |
aSELP, P-selectin; GDF11, Growth/differentiation factor 11; MSTN, Growth/differentiation factor 8; IFNG, Interferon gamma; SNAP25, Synaptosomal-associated protein 25; EGLN1, Egl nine homolog 1; EGLN2, Egl nine homolog 2; EGLN3, Egl nine homolog 3; IDH1, Isocitrate dehydrogenase [NADP] cytoplasmic; SOST, Sclerostin; CALCA, Calcitonin gene-related peptide 1; CALCRL, Calcitonin-gene-related peptide receptor; HTR1F, 5-hydroxytryptamine receptor 1F; GPR55, G-protein coupled receptor 55; MC4R, Melanocortin receptor 4; NTRK2, BDNF/NT-3 growth factors receptor; NTRK3, NT-3 growth factor receptor; ROS1, Proto-oncogene tyrosine-protein kinase ROS; PIK3CG, Phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit gamma isoform; LTK, Leukocyte tyrosine kinase receptor; FER, Tyrosine-protein kinase Fer; FES, Tyrosine-protein kinase Fes/Fps; PTK2, Focal adhesion kinase 1; PTK2B, Protein-tyrosine kinase 2-beta;TNK2,Activated CDC42 kinase 1; SYK, Tyrosine-protein kinase SYK; CD4,T-cell surface glycoprotein CD4; IL3RA, Interleukin-3 receptor; XPO1,Exportin-1; ALAS1, aminolevulinate synthase1 (ALAS1) mRNA; APOC3, apolipoprotein C-III (apoC-III) mRNA; DMD, exon 53 of dystrophin pre-mRNA; FGF23, Fibroblast growth factor 23; HBA1, hemoglobin subunit alpha; SLC5A1, Sodium/glucose cotransporter 1; SLC9A3, Sodium/hydrogen exchanger 3; CD79B, B-cell antigen receptor complex-associated protein beta chain; NECTIN4, Nectin-4; VWF, von Willebrand factor;