Table 1 Targeted drugs approved by the FDA in 2019
From: Novel targeted drugs approved by the NMPA and FDA in 2019
No. | Brand name | Active ingredient | Approval date | Target/Activity | FDA-approved use on approval date* | Drug class | Company |
|---|---|---|---|---|---|---|---|
1 | Egaten | Triclabendazole | 2/13/2019 | Microtubule/tubulin | To treat fascioliasis, a parasitic infestation caused by two species of flatworms or trematodes that mainly affect the liver, sometimes referred to as “liver flukes” | Small molecule | Novartis Inc. |
2 | Zulresso | Brexanolone | 3/19/2019 | GABA receptor | To treat postpartum depression (PPD) in adult women | Small molecule | Sage Therapeutics |
3 | Sunosi | Solriamfetol | 3/20/2019 | Dopamine and norepinephrine transporters | To treat excessive sleepiness in adult patients with narcolepsy or obstructive sleep apnea | Small molecule | Jazz Pharmaceuticals |
4 | Mayzent | Siponimod | 3/26/2019 | Sphingosine-1-phosphate (S1P) receptor | To treat adults with relapsing forms of multiple sclerosis | Small molecule | Novartis Inc. |
5 | Evenity | Romosozumab-aqqg | 4/9/2019 | Sclerostin | To treat osteoporosis in postmenopausal women at high risk of fracture | Monoclonal antibody (mAb) | Amgen |
6 | Balversa | Erdafitinib | 4/12/2019 | FGFR family | To treat adult patients with locally advanced or metastatic bladder cancer | Small molecule | Janssen Biotech |
7 | Skyrizi | Risankizumab-rzaa | 4/23/2019 | Interleukin-23 (IL-23) | To treat moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy | Monoclonal antibody (mAb) | AbbVie Inc. |
8 | Vyndaqel | Tafamidis meglumine | 5/3/2019 | Selective transthyretin (TTR) stabilizer | To treat heart disease (cardiomyopathy) caused by transthyretin-mediated amyloidosis (ATTR-CM) in adults | Small molecule | FOLDRX Pharms |
9 | Piqray | Alpelisib | 5/24/2019 | PI3Kα inhibitor | To treat breast cancer | Small molecule | Novartis Inc. |
10 | Polivy | Polatuzumab vedotin-piiq | 6/10/2019 | Anti-CD79b antibody-drug conjugate (ADC) | To treat adult patients with relapsed or refractory diffuse large B-cell lymphoma | Antibody-drug conjugate (ADC) | Genentech Inc. |
11 | Vyleesi | Bremelanotide | 6/21/2019 | Melanocortin 4 receptor agonist | To treat hypoactive sexual desire disorder in premenopausal women | Synthetic peptide analog | AMAG Pharmaceuticals |
12 | Xpovio | Selinexor | 7/3/2019 | Selective CRM1 inhibitor | To treat adult patients with relapsed or refractory multiple myeloma (RRMM) | Small molecule | Karyopharm Therapeutics Inc. |
13 | Recarbrio | Imipenem, cilastatin and relebactam | 7/16/2019 | Imipenem: β-lactamase inhibitor (previously FDA-approved antibiotic) cilastatin: dehydropeptidase inhibitor (previously FDA-approved antibiotic) relebactam: new β-lactamase inhibitor | To treat complicated urinary tract and intra-abdominal infections | Small molecule | Merck & Co., Inc. |
14 | Nubeqa | Darolutamide | 7/30/2019 | Androgen receptor (AR) antagonist | To treat adult patients with nonmetastatic castration-resistant prostate cancer | Small molecule | Bayer Healthcare |
15 | Turalio | Pexidartinib | 8/2/2019 | Colony-stimulating factor 1 receptor (CSF1R) | To treat adult patients with symptomatic tenosynovial giant cell tumor | Small Molecule | Daiichi Sankyo Inc. |
16 | Wakix | Pitolisant | 8/14/2019 | Histamine H3 receptor inverse agonist | To treat excessive daytime sleepiness (EDS) in adult patients with narcolepsy | Small molecule | Harmony Biosciences |
17 | Rozlytrek | Entrectinib | 8/15/2019 | pan-Trk, ROS1, and ALK inhibitor | To treat adult patients with metastatic, ROS1-positive non-small-cell lung cancer (NSCLC) | Small molecule | Genentech Inc. |
18 | Inrebic | Fedratinib | 8/16/2019 | JAK2 inhibitor | To treat adult patients with intermediate-2 or high-risk primary or secondary myelofibrosis | Small molecule | Impact Biomedicines, Inc. |
19 | Rinvoq | Upadacitinib | 8/16/2019 | Janus kinase 1 (JAK1) inhibitor | To treat adults with moderately to severely active rheumatoid arthritis | Small molecule | AbbVie Inc. |
20 | Xenleta | Lefamulin | 8/19/2019 | 50S bacterial ribosome | To treat adults with community-acquired bacterial pneumonia | Small molecule | Nabriva Therapeutics |
21 | Nourianz | Istradefylline | 8/27/2019 | Adenosine A2A receptor antagonist | To treat adult patients with Parkinson’s disease experiencing “off” episodes | Small molecule | Kyowa Kirin, Inc. |
22 | Ibsrela | Tenapanor | 9/12/2019 | Na+/H+ exchanger NHE3 inhibitor | To treat irritable bowel syndrome with constipation in adults | Small molecule | Ardelyx, Inc. |
23 | Aklief | Trifarotene | 10/4/2019 | Retinoic acid receptor (RAR) agonist | For the topical treatment of acne vulgaris in patients 9 years of age and older | Small molecule | Galderma R&D |
24 | Beovu | Brolucizumab-dbll | 10/7/2019 | Vascular endothelial growth factor (VEGF) inhibitor | To treat wet age-related macular degeneration | Monoclonal antibody (mAb) | Novartis Pharmaceuticals Corporation |
25 | Scenesse | Afamelanotide | 10/8/2019 | Melanocortin 1 receptor | To increase pain-free light exposure in adult patients with a history of phototoxic reactions (skin damage) from erythropoietic protoporphyria | Synthetic peptide analog | CLINUVEL PHARMACEUTICALS LTD |
26 | Reyvow | Lasmiditan | 10/11/2019 | 5-HT1F receptor agonist | For the acute treatment of migraine with or without aura in adults | Small molecule | Eli Lilly |
27 | Trikafta | Elexacaftor/ivacaftor/tezacaftor | 10/21/2019 | Elexacaftor: new cystic fibrosis transmembrane conductance regulator (CFTR) modulator ivacaftor: CFTR potentiator tezacaftor: F508del CFTR corrector | To treat patients 12 years of age and older with the most common gene mutation that causes cystic fibrosis | Small molecule | Vertex Pharmaceuticals |
28 | Reblozyl | Luspatercept-aamt | 11/8/2019 | Binds several TGF-beta superfamily ligands | For the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions | Recombinant fusion protein | Celgene Corporation and Acceleron Pharma Inc. |
29 | Brukinsa | Zanubrutinib | 11/14/2019 | Bruton tyrosine kinase (BTK) inhibitor | To treat certain patients with mantle cell lymphoma, a form of blood cancer | Small molecule | BeiGene USA Inc. |
30 | Adakveo | Crizanlizumab-tmca | 11/15/2019 | P-selectin | To treat patients with painful complications of sickle cell disease | Monoclonal antibody (mAb) | Novartis Inc. |
31 | Givlaari | Givosiran | 11/20/2019 | Aminolevulinic acid synthase 1 (ALAS1) | To treat acute hepatic porphyria, a rare blood disorder | Gene therapy: RNA interference (RNAi) | Alnylam Pharmaceuticals |
32 | Xcopri | Cenobamate | 11/21/2019 | Sodium channel blocker | To treat partial onset seizures | Small molecule | SK Life Science, Inc. |
33 | Oxbryta | Voxelotor | 11/25/2019 | Sickle hemoglobin (HbS) polymerization inhibitor | To treat sickle cell disease | Small molecule | Oxbryta to Global Blood Therapeutics |
34 | Vyondys 53 | Golodirsen | 12/12/2019 | Exon 53 | To treat certain patients with Duchenne muscular dystrophy | Gene therapy: antisense oligonucleotide | Sarepta Therapeutics |
35 | Padcev | Enfortumab vedotin-ejfv | 12/18/2019 | Nectin-4 | To treat refractory bladder cancer | Antibody-drug conjugate (ADC) | Seattle Genetics |
36 | Caplyta | Lumateperone tosylate | 12/20/2019 | 5-HT2A receptor antagonist | To treat schizophrenia | Small molecule | Intra-Cellular Therapies, Inc. |
37 | Dayvigo | Lemborexant | 12/20/2019 | Dual antagonist of the orexin OX1 and OX2 receptors | To treat insomnia | Small molecule | Eisai Inc. |
38 | Enhertu | Fam-trastuzumab deruxtecan-nxki | 12/20/2019 | Trastuzumab: epidermal growth factor receptor 2 (HER2) deruxtecan: DNA topoisomerase I inhibitor | To treat metastatic breast cancer | Antibody-drug conjugate (ADC) | AstraZeneca and Daiichi Sankyo Company, Limited |
39 | Ubrelvy | Ubrogepant | 12/23/2019 | Calcitonin gene-related peptide receptor (CGRP) antagonist | For the acute treatment of migraine with or without aura in adults | Small molecule | Allergan USA, Inc. |
