References for First-In-Class Agents and Pharmacological Similars

Part 1: First-In-Class Agents and Part 2: Pharmacological Similars
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1. +
Blumenthal  DK. Chapter 3. Pharmacodynamics: Molecular Mechanisms of Drug Action. In: Brunton  LL, Hilal-Dandan  R, Knollmann  BC. eds. Goodman & Gilman's: The Pharmacological Basis of Therapeutics, 13e. New York, NY: McGraw-Hill; 2018. Available from McGraw Hill Education. accessmedicine.mhmedical.com/content.aspx?bookid=2189&sectionid=170349571 (accessed Feb. 27, 2019).
+
2. +
Yan  L, Zhang  W. Precision medicine becomes reality-tumor type-agnostic therapy. Cancer Commun (Lond). 2018;38(1):6.
[PubMed: 29764494] ; PubMed Central PMCID: PMC5953403.
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3. +
ABLYSINOL (dehydrated alcohol) injection, for cardiac septal branch intra-arterial use [product information]. Revised: 6/2018. Belcher Pharmaceuticals, LLC, Largo, FL. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/207987lbl.pdf (accessed Feb. 27, 2019).
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4. +
Food and Drug Administration Center for Drug Evaluation and Research: NDA 207987 [APPROVAL Letter]. Dated 06/21/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/appletter/2018/207987Orig1s000ltr.pdf (accessed Feb. 27, 2019).
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5. +
Azedra® (iobenguane I 131) injection, for intravenous use [product information]. Revised: 7/2018. Progenics Pharmaceuticals, Inc.; New York, NY. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/209607s000lbl.pdf (accessed Feb. 27, 2019).
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6. +
FDA approves first treatment for rare adrenal tumors [FDA News Release]. July 30, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm615155.htm (accessed Jan. 5, 2019).
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7. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves iobenguane I 131 for rare adrenal gland tumors [webpage]. Last Updated: 07/30/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm615157.htm (accessed Jan. 3, 2019).
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8. +
Lutathera® (lutetium Lu 177 dotatate) injection, for intravenous use [product information]. Revised: 01/2018. Advanced Accelerator Applications USA, Inc., NJ. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/208700s000lbl.pdf (accessed Jan. 1, 2019).
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9. +
FDA approves new treatment for certain digestive tract cancers [FDA News Release]. January 26, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm594043.htm (accessed Jan. 1, 2019).
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10. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves lutetium Lu 177 dotatate for treatment of GEP-NETS [webpage]. Last Updated: 01/26/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm594105.htm (accessed Jan. 3, 2019).
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11. +
Drug Trials Snapshot: Lutathera [webpage]. Last Updated: 02/20/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ucm597090.htm (accessed Jan. 1, 2019).
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12. +
Primatene Mist (epinephrine) 0.125 mg/inhalation [OTC Drug Facts label]. Armstrong Pharms. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=205920 (accessed Feb. 27, 2019).
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13. +
Epinephrine CFC Metered-dose Inhalers—Questions and Answers [webpage]. Last Updated: 02/22/2016 Available from: U.S. Department of Health and Human Services, Food and Drug Administration. https://wayback.archive-it.org/7993/20170723104117/https://www.fda.gov/Drugs/DrugSafety/InformationbyDrugClass/ucm080427.htm (accessed Feb. 28, 2019).
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14. +
Statement from FDA Commissioner Scott Gottlieb, M.D., and Janet Woodcock, M.D., director of the Center for Drug Evaluation and Research, on approval of OTC Primatene Mist to treat mild asthma [FDA News Release]. November 8, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm625338.htm (accessed Feb. 4, 2019).
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15. +
Statement from FDA Commissioner Scott Gottlieb, M.D., on FDA’s efforts to foster discovery and development of new tools to fight antimicrobial-resistant infections [FDA News Release]. June 12, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm610503.htm (accessed Jan. 5, 2019).
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16. +
CY 2018 CDER Breakthrough Therapy Calendar Year Approvals: Data as of December 31, 2018 [webpage]. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/downloads/drugs/developmentapprovalprocess/howdrugsaredevelopedandapproved/drugandbiologicapprovalreports/ndaandblaapprovalreports/ucm494010.pdf (accessed Feb. 21, 2019).
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17. +
Food and Drug Administration Safety and Innovation Act (FDASIA)—Fact Sheet: Breakthrough Therapies [webpage]. Last Updated: 3/28/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendmentstotheFDCAct/FDASIA/ucm329491.htm (accessed Feb 21, 2019).
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18. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: 2018 Cancer Approvals [webpage]. Last Updated: 2/26/2019. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/drugs/informationondrugs/approveddrugs/ucm279174.htm (accessed Feb 27, 2019)
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19. +
FDA approves a new antibacterial drug to treat a serious lung disease using a novel pathway to spur innovation [FDA News Release]. September 28, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm622048.htm (accessed Feb. 21, 2019).
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20. +
Safety Announcement: FDA warns about severe worsening of multiple sclerosis after stopping the medicine Gilenya (fingolimod) [webpage]. Last Updated: 12/20/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/DrugSafety/ucm626095.htm (accessed Feb. 27, 2019).
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21. +
Orphan Drug Product designation database: Search Orphan Drug Designations and Approvals [webpage]. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/ (accessed Feb. 27, 2019).
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22. +
Noshi  T, Kitano  M, Taniguchi  K,  et al. In vitro characterization of baloxavir acid, a first-in-class cap-dependent endonuclease inhibitor of the influenza virus polymerase PA subunit. Antiviral Res. 2018;160:109–117.
[PubMed: 30316915] .
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23. +
FDA approves the first drug with an indication for treatment of smallpox [FDA News Release]. July 13, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm613496.htm (accessed Feb. 3, 2019).
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24. +
DrugBank: Stiripentol (Accession number = DB09118) [open database]. Available from: Wishart Research Group. University of Alberta; Edmonton, Canada. www.drugbank.ca/drugs/DB09118 (accessed Feb. 3, 2019).
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25. +
Guce  AI, Clark  NE, Rogich  JJ,  et al. The molecular basis of pharmacological chaperoning in human α-galactosidase. Chem Biol. 2011;18(12):1521–1526.
[PubMed: 22195554] ; PubMed Central PMCID: PMC3246215.
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26. +
FDA approves first targeted treatment for patients with relapsed or refractory acute myeloid leukemia who have a certain genetic mutation [FDA News Release]. July 20, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm614115.htm (accessed Jan. 1, 2019).
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27. +
McAdoo  SP, Tam  FW. Fostamatinib disodium. Drugs Future. 2011;36(4):273.
[PubMed: 23284223] ; PubMed Central PMCID: PMC3533134.
+
28. +
Blumenthal  GM, Pazdur  R. Supplementary Table 1 - Summary of FDA oncology drug approvals in 2018 (in decreasing chronological order). Supplementary Information to: Blumenthal  GM, Pazdur  R. Approvals in 2018: a histology-agnostic new molecular entity, novel end points and real-time review. Nat Rev Clin Oncol. 2019;16(3):139–141.
[PubMed: 30670830] . Available from Nature Publishing Group. https://static-content.springer.com/esm/art%3A10.1038%2Fs41571-019-0170-z/MediaObjects/41571_2019_170_MOESM1_ESM.pdf (accessed Feb. 27, 2019).
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29. +
FDA approves first cancer drug through new oncology review pilot that enables greater development efficiency [FDA News Release]. July 18, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm613801.htm (accessed Jan. 5, 2019).
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30. +
Oncology Center of Excellence: Real-Time Oncology Review Pilot Program [webpage]. Last Updated: 01/29/2019. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/OCE/ucm612927.htm (accessed Feb. 21, 2019).
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31. +
Morrison  C. Fresh from the biotech pipeline-2018. Nat Biotechnol. 2019;37(2) :118–123.
[PubMed: 30718867] .
+
32. +
FDA approves first-line treatment for peripheral T-cell lymphoma under new review pilot [FDA News Release]. November 16, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm626079.htm (accessed Feb. 21, 2019).
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33. +
Approved Drugs: FDA grants regular approval for pembrolizumab in combination with chemotherapy for first-line treatment of metastatic nonsquamous NSCLC [webpage]. Last Updated: 08/20/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm617471.htm (accessed Feb. 21, 2018).
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34. +
List of Cleared or Approved Companion Diagnostic Devices (In Vitro and Imaging Tools) [webpage]. Last Updated: 12/13/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/medicaldevices/productsandmedicalprocedures/invitrodiagnostics/ucm301431.htm (accessed Feb. 21, 2019).
+
35. +
Companion Diagnostics [webpage]. Last Updated: 12/07/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/MedicalDevices/ProductsandMedicalProcedures/InVitroDiagnostics/ucm407297.htm (accessed Feb 21, 2019).
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36. +
Approved Drugs: FDA updates prescribing information for Keytruda and Tecentriq [webpage]. Last Updated: 08/20/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm617378.htm (accessed Feb 21, 2019).
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37. +
Approved Drugs: FDA limits the use of Tecentriq and Keytruda for some urothelial cancer patients [webpage]. Last Updated: 07/05/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm612484.htm (accessed Feb. 21, 2019).
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38. +
Statement from FDA Commissioner Scott Gottlieb, M.D., on the FDA’s new effort for developing and class labeling of in vitro companion diagnostics for classes of oncology therapeutic products [FDA News Release]. December 6, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm627745.htm (accessed Jan. 4, 2019).
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39. +
Ju  H, Zhang  J, Huang  B,  et al. Inhibitors of Influenza Virus Polymerase Acidic (PA) Endonuclease: Contemporary Developments and Perspectives. J Med Chem. 2017;60(9):3533–3551.
[PubMed: 28118010] .
+
40. +
XOFLUZATM (baloxavir marboxil) tablets for oral use[product information]. Revised: 10/2018. Genentech USA, Inc.; South San Francisco, CA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210854s000lbl.pdf (accessed 1/1/2019).
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41. +
Drug Trials Snapshot: XOFLUZA [webpage]. Last Updated: 11/06/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ucm624981.htm (accessed 1/1/2019).
+
42. +
Ma  TK, McAdoo  SP, Tam  FW. Spleen tyrosine kinase: A crucial player and potential therapeutic target in renal disease. Nephron. 2016;133(4):261–269.
[PubMed: 27476075] .
+
43. +
TAVALISSETM (fostamatinib disodium hexahydrate) tablets for oral use [product information]. Revised: 04/2018. Rigel Pharmaceuticals, Inc.; South San Francisco, CA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/209299lbl.pdf (accessed 1/1/2019).
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44. +
Tegsedi (inotersen) injection, for subcutaneous use [product information]. Revised: 10/2018. Ionis Pharmaceuticals, Inc.; Carlsbad, CA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/211172lbl.pdf (accessed 1/1/2019).
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45. +
ONPATTRO (patisiran) lipid complex injection, for intravenous use [product information]. Revised: 8/2018. Alnylam Pharmaceuticals, Inc.; Cambridge, MA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210922s000lbl.pdf (accessed 1/1/2019).
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46. +
Dhillon  S. Ivosidenib: First Global Approval. Drugs. 2018;78(14):1509–1516. Erratum in: Drugs. 2019;79(1):107.
[PubMed: 30209701] ; PubMed Central PMCID: PMC6315051.
+
47. +
Tibsovo® (ivosidenib tablets) for oral use [product information]. Revised: 07/2018. Agios Pharmaceuticals, Inc.; Cambridge, MA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/211192s000lbl.pdf (accessed 1/1/2019).
+
48. +
Lange  AM, Lo  HW. Inhibiting TRK proteins in clinical cancer therapy. Cancers (Basel). 2018;10(4):E105.
[PubMed: 29617282] ; PubMed Central PMCID: PMC5923360.
+
49. +
Vitrakvi® (larotrectinib) capsules for oral use and oral solution [product information]. Revised: 11/2018. Loxo Oncology, Inc.; Stamford, CT. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210861s000lbl.pdf (accessed 1/1/2019).
+
50. +
GalafoldTM (migalastat) capsules for oral use [product information]. Revised: 08/2018. Amicus Therapeutics U.S., Inc.; Cranbury, NJ. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/208623lbl.pdf (accessed 1/1/2019).
+
51. +
Diacomit (stiripentol) capsules for oral use and powder for oral suspension [product information]. Revised: 8/2018. Biocodex; Beauvais—France. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/206709s000,207223s000lbl.pdf (accessed 1/1/2019).
+
52. +
Tpoxx (tecovirimat) capsules for oral use [product information]. Revised: 07/2018. SIGA Technologies, Inc.; Corvallis, OR. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/208627s000lbl.pdf (accessed 1/1/2019).
+
53. +
Preissner  S, Kroll  K, Dunkel  M,  et al. SuperCYP: a comprehensive database on Cytochrome P450 enzymes including a tool for analysis of CYP-drug interactions. Nucleic Acids Res. 2010;38:D237–D243.
[PubMed: 19934256] ; PubMed Central PMCID: PMC2808967.
+
54. +
FDA approves new drug to treat influenza [FDA News Release]. October 24, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm624226.htm (accessed 1/1/2019).
+
55. +
Flu News & Spotlights: CDC Responds to Approval of New Flu Antiviral Drug [webpage]. Last updated: December 4, 2018. Available from: U.S. Department of Health and Human Services, Centers for Disease Control and Prevention. www.cdc.gov/flu/spotlights/cdc-responds-approval-new-flu-drug.htm (accessed Feb. 2, 2019).
+
56. +
Influenza Antiviral Medications: Summary for Clinicians [webpage]. Last updated: December 27, 2018. Available from: U.S. Department of Health and Human Services, Centers for Disease Control and Prevention. www.cdc.gov/flu/professionals/antivirals/summary-clinicians.htm (accessed Feb. 2, 2019).
+
57. +
Mushtaq  A. Baloxavir: game-changer or much ado about nothing? Lancet Respir Med. 2018;6(12):903–904.
[PubMed: 30420246] .
+
58. +
Uyeki  TM. A step forward in the treatment of influenza. N Engl J Med. 2018;379(10):975–977.
[PubMed: 30184452] .
+
59. +
Kikuchi  T, Watanabe  A. Baloxavir heralds a new era in influenza virus biology. Respir Investig. 2019;57(1):1–2.
[PubMed: 30396860] .
+
60. +
Flu Activity & Surveillance: Fluview [webpage]. Last updated: August 23, 2018]. Available from: U.S. Department of Health and Human Services, Centers for Disease Control and Prevention. www.cdc.gov/flu/weekly/fluactivitysurv.htm (accessed Feb. 2, 2019).
+
61. +
Heo  YA. Baloxavir: First global approval. Drugs. 2018;78(6):693–697.
[PubMed: 29623652] .
+
62. +
Watanabe  A, Ishida  T, Hirotsu  N,  et al. Baloxavir marboxil in Japanese patients with seasonal influenza: Dose response and virus type/subtype outcomes from a randomized phase 2 study. Antiviral Res. 2019;163:75–81.
[PubMed: 30684563] .
+
63. +
Hayden  FG, Sugaya  N, Hirotsu  N,  et al: Baloxavir Marboxil Investigators Group. Baloxavir Marboxil for Uncomplicated Influenza in Adults and Adolescents. N Engl J Med. 2018;379(10):913–923.
[PubMed: 30184455] .
+
64. +
Mossad  SB. Influenza update 2018-2019: 100 years after the great pandemic. Cleve Clin J Med. 2018;85(11):861–869.
[PubMed: 30395523] .
+
65. +
Hawkes  N. Sixty seconds on…baloxavir. BMJ. 2018;363:k4531.
[PubMed: 30366947] .
+
66. +
National Center for Biotechnology Information. PubChem Compound Database. Baloxavir Marboxil: CID=124081896. https://pubchem.ncbi.nlm.nih.gov/compound/124081896 (accessed Feb. 2, 2019).
+
67. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves fostamatinib tablets for ITP [webpage]. Last updated: 04/18/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm604956.htm (accessed Jan. 3, 2019).
+
68. +
Markham  A. Fostamatinib: First global approval. Drugs. 2018;78(9):959–963.
[PubMed: 29869203] .
+
69. +
McKeage  K, Lyseng-Williamson  KA. Fostamatinib in chronic immune thrombocytopenia: a profile of its use in the USA. Drugs Ther Perspect. 2018;34(10):451–456.
[PubMed: 30459507] ; PubMed Central PMCID: PMC6223701.
+
70. +
Newland  A, Lee  EJ, McDonald  V, Bussel  JB. Fostamatinib for persistent/chronic adult immune thrombocytopenia. Immunotherapy. 2018;10(1):9–25.
[PubMed: 28967793] .
+
71. +
DrugBank: Fostamatinib (Accession number: DB12010) [open database]. Available from: Wishart Research Group. University of Alberta; Edmonton, Canada. www.drugbank.ca/drugs/DB12010 (accessed Feb. 2, 2019).
+
72. +
Drug Profile: Fostamatinib - Rigel Pharmaceuticals. AdisInsight Drugs [database]. Released 2005 January 13, Updated 2019 January 28. Available from: Springer Nature Switzerland AG. http://adisinsight.springer.com/drugs/800021937 (accessed Jan. 29, 2019).
+
73. +
Drug Trials Snapshot: Tavalisse [webpage]. Last Updated: 04/25/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ucm605411.htm (accessed Mar. 1, 2019).
+
74. +
National Center for Biotechnology Information. PubChem Compound Database. Fostamatinib disodium hexahydrate: CID=24828759. https://pubchem.ncbi.nlm.nih.gov/compound/24828759 (accessed Feb. 2, 2019).
+
75. +
FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease [FDA News Release]. August 10, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616518.htm (accessed 1/1/2019).
+
76. +
Rizk  M, Tüzmen  Ş. Update on the clinical utility of an RNA interference-based treatment: focus on Patisiran. Pharmgenomics Pers Med. 2017;10:267–278.
[PubMed: 29184431] ; PubMed Central PMCID: PMC5689029.
+
77. +
Benson  MD, Waddington-Cruz  M, Berk  JL,  et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):22–31.
[PubMed: 29972757] .
+
78. +
Kristen  AV, Ajroud-Driss  S, Conceição  I,  et al. Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis. Neurodegener Dis Manag. 2019;9(1):5–23.
[PubMed: 30480471] .
+
79. +
Hoy  SM. Patisiran: First global approval. Drugs. 2018;78(15):1625–1631.
[PubMed: 30251172] .
+
80. +
Keam  SJ. Inotersen: First global approval. Drugs. 2018;78(13):1371–1376.
[PubMed: 30120737] .
+
81. +
Adams  D, Gonzalez-Duarte  A, O'Riordan  WD,  et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):11–21.
[PubMed: 29972753] .
+
82. +
Armstrong,  M. Tegsedi gets US green light, but still looks second best. Vantage October 8, 2018. Copyright 2019 Evaluate Ltd. http://www.evaluate.com/vantage/articles/news/snippets/tegsedi-gets-us-green-light-still-looks-second-best (accessed Feb. 28, 2019).
+
83. +
Approved Risk Evaluation and Mitigation Strategies (REMS): Tegsedi (Inotersen) NDA #211172 [webpage]. REMS last update: 10/05/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/Scripts/Cder/Rems/index.cfm?event=IndvRemsDetails.page&REMS=383 (accessed Feb. 28, 2019).
+
84. +
A triumph of perseverance over interference. Nat Biotechnol. 2018;36(9):775.
[PubMed: 30188535] .
+
85. +
Bennett  CF, Baker  BF, Pham  N,  et al. Pharmacology of antisense drugs. Annu Rev Pharmacol Toxicol. 2017;57:81–105.
[PubMed: 27732800] .
+
86. +
Titze-de-Almeida  R, David  C, Titze-de-Almeida  SS. The race of 10 synthetic RNAi-based drugs to the pharmaceutical market. Pharm Res. 2017;34(7):1339–1363. Erratum in: Pharm Res. 2018;35(3):53.
[PubMed: 28389707] .
+
87. +
DrugBank [open database, version 5.1.2]: Patisiran (Accession number = DB14582). Available from: Wishart Research Group. University of Alberta; Edmonton, Canada. www.drugbank.ca/drugs/DB14582 (accessed Feb. 2, 2019).
+
88. +
National Center for Biotechnology Information. PubChem Compound Database. Patisiran: SID=350082694. https://pubchem.ncbi.nlm.nih.gov/substance/350082694 (accessed Feb. 2, 2019).
+
89. +
Dhillon  S. Ivosidenib: First global approval. Drugs. 2018;78(14):1509–1516. Erratum in: Drugs. 2019;79(1):107.
[PubMed: 30209701] ; PubMed Central PMCID: PMC6315051.
+
90. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves ivosidenib for relapsed or refractory acute myeloid leukemia [webpage]. Last updated: 10/18/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm614128.htm (accessed Jan. 3, 2019).
+
91. +
DrugBank [open database, version 5.1.2]: Ivosidenib (Accession number = DB14568). Available from: Wishart Research Group. University of Alberta; Edmonton, Canada. www.drugbank.ca/drugs/DB14568 (accessed Feb. 2, 2019).
+
92. +
Dang  L, White  DW, Gross  S,  et al. Cancer-associated IDH1 mutations produce 2-hydroxyglutarate. Nature. 2009;462(7274):739–744.
[PubMed: 19935646] ; PubMed Central PMCID: PMC2818760.
+
93. +
Garber  K. Oncology's energetic pipeline. Nat Biotechnol. 2010;28(9):888–891.
[PubMed: 20829819] .
+
94. +
Drug Trials Snapshot: Tibsovo [webpage]. Last Updated: 08/02/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ucm615501.htm (accessed Mar. 1, 2019).
+
95. +
National Center for Biotechnology Information. PubChem Compound Database. Ivosidenib: CID=71657455. https://pubchem.ncbi.nlm.nih.gov/compound/71657455 (accessed Feb. 2, 2019).
+
96. +
FDA approves an oncology drug that targets a key genetic driver of cancer, rather than a specific type of tumor [FDA News Release]. November 26, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm626710.htm (accessed 1/1/2019).
+
97. +
DrugBank [open database, version 5.1.2]: Larotrectinib (Accession number = DB14723). Available from: Wishart Research Group. University of Alberta; Edmonton, Canada. www.drugbank.ca/drugs/DB14723 (accessed Feb. 2, 2019).
+
98. +
Scott  LJ. Larotrectinib: First global approval. Drugs. 2019;79(2):201–206.
[PubMed: 30635837] .
+
99. +
Kummar  S, Lassen  UN. TRK inhibition: A new tumor-agnostic treatment strategy. Target Oncol. 2018;13(5):545–556.
[PubMed: 30276762] .
+
100. +
Dolgin  E. Twenty four gene fusions in one drug approval: Loxo’s tissue-agnostic TRK-inhibitor gets a green light. Nat Biotechnol. 2018. www.nature.com/articles/d41587-018-00002-3 (accessed Jun. 24, 2019).
+
101. +
Garber  K. Oncologists await historic first: a pan-tumor predictive marker, for immunotherapy. Nat Biotechnol. 2017;35(4):297–298.
[PubMed: 28398313] .
+
102. +
Mullard  A. 2018 FDA drug approvals. Nat Rev Drug Discov. 2019;18(2):85–89.
[PubMed: 30710142] .
+
103. +
TruSightTM Tumour 170 [product information]. Available from: Illumina, Inc. www.illumina.com/content/dam/illumina-marketing/documents/products/datasheets/trusight-tumor-170-data-sheet-1170-2016-017.pdf (accessed Feb. 28, 2019).
+
104. +
Illumina and Loxo Oncology to Partner on Developing Next-Generation Sequencing-Based Pan-Cancer Companion Diagnostics [news release]. April, 10, 2019. Available from: Illumina, Inc. http://investor.illumina.com/mobile.view?c=121127&v=203&d=1&id=2341745 (accessed Feb. 2, 2019).
+
105. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves larotrectinib for solid tumors with NTRK gene fusions [webpage]. Last Updated: 12/17/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm626720.htm (accessed Jan. 3, 2019).
+
106. +
National Center for Biotechnology Information. PubChem Compound Database. Larotectinib: CID=46188928. https://pubchem.ncbi.nlm.nih.gov/compound/46188928 (accessed Feb. 2, 2019).
+
107. +
FDA approves new treatment for a rare genetic disorder, Fabry disease [FDA News Release]. August 10, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616598.htm (accessed Jan. 1, 2019).
+
108. +
Southern  KW, Patel  S, Sinha  IP, Nevitt  SJ. Correctors (specific therapies for class II CFTR mutations) for cystic fibrosis. Cochrane Database Syst Rev. 2018;8:CD010966.
[PubMed: 30070364] .
+
109. +
Gaggl  M, Sunder-Plassmann  G. Fabry disease: A pharmacological chaperone on the horizon. Nat Rev Nephrol. 2016;12(11):653–654.
[PubMed: 27665929] .
+
110. +
Moran  N. FDA approves Galafold, a triumph for Amicus. Nat Biotechnol. 2018;36(10):913.
[PubMed: 30307912] .
+
111. +
DrugBank [open database, version 5.1.2]: Migalastat (Accession number = DB5018). Available from: Wishart Research Group. University of Alberta; Edmonton, Canada. www.drugbank.ca/drugs/DB5018 (accessed Feb. 2, 2019).
+
112. +
Drug Approval Package: Galafold (migalastat) [Multi-Discipline Review Summary Review]. Application number: 208623Orig1s000. Reference ID: 4305409. Version date: February 1, 2016. Electronically signed 5/3/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration, Center For Drug Evaluation And Research. www.accessdata.fda.gov/drugsatfda_docs/nda/2018/208623Orig1s000MultidisciplineR.pdf (accessed Feb. 2, 2019).
+
113. +
Markham  A. Migalastat: First global approval. Drugs. 2016;76(11):1147–1152.
[PubMed: 27351440] .
+
114. +
FDA Approves GalafoldTM (migalastat) for the Treatment of Certain Adult Patients with Fabry Disease [Press Release]. Aug. 10, 2018. Copyright © 2019 Amicus Therapeutics, Inc. http://ir.amicusrx.com/news-releases/news-release-details/fda-approves-galafoldtm-migalastat-treatment-certain-adult (accessed Feb. 28, 2019).
+
115. +
Schiffmann  R, Bichet  DG, Germain  DP,  et al. Improvement in gastrointestinal symptoms observed in the phase 3 FACETS (AT1001-011) study of migalastat in patients affected with Fabry disease [abstract plus poster]. Mol Genet Metab. 2015;114(2):S103–104.
+
116. +
Hughes  DA, Nicholls  K, Shankar  SP,  et al. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. J Med Genet. 2017;54(4):288–296. Erratum in: J Med Genet. 2018;55(6):422–429.
[PubMed: 27834756] ; PubMed Central PMCID: PMC5502308.
+
117. +
Germain  DP, Hughes  DA, Nicholls  K,  et al. Treatment of Fabry's Disease with the pharmacologic chaperone migalastat. N Engl J Med. 2016;375(6):545–555.
[PubMed: 27509102] .
+
118. +
Clinical Review Report: Migalastat (Galafold): (Amicus Therapeutics): Indication: Fabry Disease [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Feb. Available from www.ncbi.nlm.nih.gov/books/NBK533676/.
[PubMed: 30457788] .
+
119. +
Ishii  S. Pharmacological chaperone therapy for Fabry disease. Proc Jpn Acad Ser B Phys Biol Sci. 2012;88(1):18–30.
[PubMed: 22241068] ; PubMed Central PMCID: PMC3278969.
+
120. +
CADTH Canadian Drug Expert Committee Recommendation: Migalastat (Galafold — Amicus Therapeutics): Indication: Fabry Disease [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Jan. No abstract available. Available from www.ncbi.nlm.nih.gov/books/NBK533471/.
[PubMed: 30457790] .
+
121. +
Pharmacoeconomic Review Report: Migalastat (Galafold): (Amicus Therapeutics): Indication: Fabry Disease [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Feb. Available from www.ncbi.nlm.nih.gov/books/NBK533451/.
[PubMed: 30457791] .
+
122. +
National Center for Biotechnology Information. PubChem Compound Database. Migalastat: CID=176077. https://pubchem.ncbi.nlm.nih.gov/compound/176077 (accessed Feb. 2, 2019).
+
123. +
Stiripentol (Diacomit): For Severe Myoclonic Epilepsy in Infancy (Dravet Syndrome) [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2015 Apr. Available from www.ncbi.nlm.nih.gov/books/NBK349342/.
[PubMed: 26962599] .
+
124. +
Nickels  KC, Wirrell  EC. Stiripentol in the management of epilepsy. CNS Drugs. 2017;31(5):405–416.
[PubMed: 28434133] .
+
125. +
Brigo  F, Igwe  SC, Bragazzi  NL. Stiripentol add-on therapy for focal refractory epilepsy. Cochrane Database Syst Rev. 2018;5:CD009887.
[PubMed: 29747241] .
+
126. +
National Center for Biotechnology Information. PubChem Compound Database. Stiripentol: CID= 5311454. https://pubchem.ncbi.nlm.nih.gov/substance/5311454 (accessed Feb. 3, 2019).
+
127. +
Nabbout  R, Chiron  C. Stiripentol: an example of antiepileptic drug development in childhood epilepsies. Eur J Paediatr Neurol. 2012;16 Suppl 1:S13–S17.
[PubMed: 22695038] .
+
128. +
Drug Trials Snapshot: Diacomit [webpage]. Last Updated: 08/28/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ucm618455.htm (accessed Mar. 1, 2019).
+
129. +
Grosenbach  DW, Jordan  R, Hruby  DE. Development of the small-molecule antiviral ST-246 as a smallpox therapeutic. Future Virol. 2011;6(5):653–671.
[PubMed: 21837250] ; PubMed Central PMCID: PMC3151656.
+
130. +
Hoy  SM. Tecovirimat: First global approval. Drugs. 2018;78(13):1377–1382.
[PubMed: 30120738] .
+
131. +
Stower  H. An antiviral for smallpox [Infectious Disease Research Highlight]. Nat Med. 2018;24:1088.
+
132. +
Smallpox antiviral ends decades-long search. Nat Biotechnol. 2018;36(9):781.
[PubMed: 30188546] .
+
133. +
FDA Approves First Smallpox-Indicated Treatment, SIGA’s TPOXX. Genetic Engineering and Biotechnology News (GEN). July 13, 2018. Copyright © 2019 Genetic Engineering & Biotechnology News. www.genengnews.com/topics/drug-discovery/fda-approves-first-smallpox-indicated-treatment-sigas-tpoxx/ (accessed Feb 3, 2019).
+
134. +
The spectre of smallpox lingers. Nature. 2018;560(7718):281.
[PubMed: 30104592] .
+
135. +
Grosenbach  DW, Honeychurch  K, Rose  EA,  et al. Oral Tecovirimat for the treatment of smallpox. N Engl J Med. 2018;379(1):44–53.
[PubMed: 29972742] ; PubMed Central PMCID: PMC6086581.
+
136. +
Melamed  S, Israely  T, Paran  N. Challenges and achievements in prevention and treatment of smallpox. Vaccines (Basel). 2018;6(1):E8.
[PubMed: 29382130] ; PubMed Central PMCID: PMC5874649.
+
137. +
DrugBank [open database, version 5.1.2]: Tecovirimat (Accession number = DB12020 [DB06000]). Available from: Wishart Research Group. University of Alberta; Edmonton, Canada. www.drugbank.ca/drugs/DB12020 (accessed Feb. 3, 2019).
+
138. +
National Center for Biotechnology Information. PubChem Compound Database. Tecovirimat: CID= 16124688. https://pubchem.ncbi.nlm.nih.gov/compound/16124688 (accessed Feb. 3, 2019).
+
139. +
LucemyraTM (lofexidine) tablets for oral use [product information]. Revised: 2018. US WorldMeds, LLC.; Louisville, KY. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/209229s000lbl.pdf (accessed 1/1/2019).
+
140. +
FDA approves the first non-opioid treatment for management of opioid withdrawal symptoms in adults [FDA News Release]. May 16, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm607884.htm (accessed Jan. 1, 2019).
+
141. +
Zemdri (plazomicin) injection for intravenous use [product information]. Revised: 6/2018. Achaogen, Inc.; South San Francisco, CA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210303Orig1s000lbl.pdf (accessed 1/1/2019).
+
142. +
Xerava (eravacycline) for injection for intravenous use [product information]. Revised: 8/2018. Tetraphase Pharmaceuticals, Inc.; Watertown, MA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/211109lbl.pdf (accessed 1/1/2019).
+
143. +
Nuzyra (omadacycline) for injection for intravenous use and tablets for oral use [product information]. Revised: 10/2018. Paratek Pharmaceuticals, Inc.; Boston, MA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/209816_209817lbl.pdf (accessed 1/1/2019).
+
144. +
Honeyman  L, Ismail  M, Nelson  ML,  et al. Structure-activity relationship of the aminomethylcyclines and the discovery of omadacycline. Antimicrob Agents Chemother. 2015;59(11):7044–7053.
[PubMed: 26349824] ; PubMed Central PMCID: PMC4604364.
+
145. +
SeysaraTM (sarecycline) tablets for oral use [product information]. Revised: 10/2018. Allergan USA, Inc.; Irvine, CA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/209521s000lbl.pdf (accessed 1/1/2019).
+
146. +
Zhanel  G, Critchley  I, Lin  LY,  et al. Microbiological profile of sarecycline, a novel targeted spectrum tetracycline for the treatment of acne vulgaris. Antimicrob Agents Chemother. 2018;63(1):e01297.
[PubMed: 30397052] ; PubMed Central PMCID: PMC6325184.
+
147. +
Aemcolo (rifamycin) delayed-release tablets for oral use [product information]. Revised: 11/2018. Aries Pharmaceuticals, Inc.; San Diego, CA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210910s000lbl.pdf (accessed 1/1/2019).
+
148. +
FDA approves new drug to treat travelers’ diarrhea [FDA News Release]. November 16, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm626121.htm (accessed 1/4/2019).
+
149. +
Akynzeo® (netupitant and palonosetron) capsules for oral use [product information]. Revised: 04/2018. Helsinn Therapeutics, Inc.; Iselin, NJ. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210493s000lbl.pdf (accessed 1/1/2019).
+
150. +
Yupelri (revefenacin) solution for oral inhalation [product information]. Revised: 11/2018. Mylan Specialty L.P.; Morgantown, WV. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210598s000lbl.pdf (accessed 1/1/2019).
+
151. +
ErleadaTM (apalutamide) tablets for oral use [product information]. Revised: 2/2018. Janssen Products, LP; Horsham, PA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210951s000lbl.pdf (accessed 2/21/2019).
+
152. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves apalutamide for non-metastatic castration-resistant prostate cancer [webpage]. Last Updated: 05/03/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm596796.htm (accessed 1/3/2019).
+
153. +
FDA approves new treatment for a certain type of prostate cancer using novel clinical trial endpoint [FDA News Release]. February 14, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm596768.htm (accessed 1/1/2019).
+
154. +
Clinical Study Report. A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase III Study of ARN-509 in Men with Non-Metastatic (M0) Castration-Resistant Prostate Cancer: Selective Prostate AR Targeting with ARN-509 (SPARTAN) [Protocol ARN-509-003; Phase 3 JNJ-56021927 (apalutamide)]. Aragon Pharmaceuticals, Inc. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/nda/2018/rev_210951_arn-509-003_CSR_Redacted.pdf (accessed Jan. 6, 2019).
+
155. +
Vizimpro® (dacomitinib) tablets for oral use [product information]. Revised: 9/2018. Pfizer Labs; New York, NY. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/211288s000lbl.pdf (accessed 1/1/2019).
+
156. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves dacomitinib for metastatic non-small cell lung cancer [webpage]. Last Updated: 12/17/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm621967.htm (accessed Jan. 3, 2019).
+
157. +
DaurismoTM (glasdegib) tablets for oral use [product information]. Revised: 11/2018. Pfizer Labs; New York, NY. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210656s000lbl.pdf (accessed 1/1/2019).
+
158. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves glasdegib for AML in adults age 75 or older or who have comorbidities [webpage]. Last Updated: 11/23/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm626494.htm (accessed 1/3/2019).
+
159. +
Mektovi® (binimetinib) tablets for oral use [product information]. Revised: 06/2018. Array BioPharma Inc.; Boulder, Colorado. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210498lbl.pdf (accessed 1/1/2019).
+
160. +
FDA approves encorafenib and binimetinib in combination for unresectable or metastatic melanoma with BRAF mutations [webpage]. Last Updated: 06/27/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm611981.htm (accessed 1/1/2019).
+
161. +
BraftoviTM (encorafenib) capsules for oral use [product information]. Revised: 06/2018 Array BioPharma Inc.; Boulder, Colorado. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210496lbl.pdf (accessed 1/1/2019).
+
162. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves encorafenib and binimetinib in combination for unresectable or metastatic melanoma with BRAF mutations [webpage]. Last Updated: 06/27/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm611981.htm (accessed 1/3/2019).
+
163. +
FDA approves encorafenib and binimetinib in combination for unresectable or metastatic melanoma with BRAF mutations [webpage] Last Updated: 06/27/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm611981.htm (accessed 1/1/2019).
+
164. +
Antar  A, Otrock  ZK, El-Cheikh  J,  et al. Inhibition of FLT3 in AML: a focus on sorafenib. Bone Marrow Transplant. 2017;52(3):344–351.
[PubMed: 27775694] .
+
165. +
Xospata® (gilteritinib) tablets for oral use [product information]. Revised: 11/2018. Astellas Pharma US, Inc.; Northbrook, Il. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/211349s000lbl.pdf (accessed Jan. 1, 2019).
+
166. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves gilteritinib for relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation [webpage]. Last Updated: 12/17/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm627045.htm (accessed 1/3/2019).
+
167. +
FDA approves treatment for adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a certain genetic mutation [FDA News Release]. November 28, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm627072.htm (accessed 1/1/2019).
+
168. +
Lorbrena (lorlatinib) tablets for oral use [product information]. Revised: 11/2018. Pfizer Labs; New York, NY. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210868s000lbl.pdf (accessed 1/1/2019).
+
169. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves lorlatinib for second- or third-line treatment of ALK-positive metastatic NSCLC [webpage]. Last Updated: 12/17/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm625027.htm (accessed 1/3/2019).
+
170. +
Copiktra (duvelisib), capsules for oral use [product information]. Revised: 09/2018. Verastem, Inc.; Needham, MA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/211155s000lbl.pdf (accessed 1/1/2019).
+
171. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: duvelisib (COPIKTRA, Verastem, Inc.) for adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) [webpage]. Last Updated: 09/24/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm621503.htm (accessed 1/3/2019).
+
172. +
TalzennaTM (talazoparib) capsules for oral use [product information]. Revised: 10/2018. Pfizer Labs; New York, NY. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/211651s000lbl.pdf (accessed 1/1/2019).
+
173. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves talazoparib for gBRCAm HER2-negative locally advanced or metastatic breast cancer [webpage]. Last Updated: 12/17/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm623540.htm (accessed Jan. 3, 2019).
+
174. +
Bawa  S, Kumar  S, Drabu  S,  et al. Structural modifications of quinoline-based antimalarial agents: Recent developments. J Pharm Bioallied Sci. 2010;2(2):64–71.
[PubMed: 21814435] ; PubMed Central PMCID: PMC3147106.
+
175. +
ArakodaTM (tafenoquine) tablets for oral use [product information]. Revised: 8/2018. 60 DEGREES PHARMS; Washington, DC. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210607lbl.pdf (accessed Feb. 22, 2019).
+
176. +
FDA and DoD formalize collaboration to advance medical products in support of American military personnel [FDA News Release]. November 2, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm624970.htm (accessed Feb. 22, 2019).
+
177. +
Krintafel (tafenoquine) tablets for oral use [product information]. Revised: 7/2018. GlaxoSmithKline; Research Triangle Park, NC. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210795s000lbl.pdf (accessed 1/1/2019).
+
178. +
Moxidectin tablets for oral use [product information]. Revised: 6/2018 Medicines Development for Global Health; Melbourne, Victoria, Australia. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210867lbl.pdf (accessed 1/1/2019).
+
179. +
Biktarvy® (bictegravir, emtricitabine, and tenofovir alafenamide) tablets for oral use [product information]. Revised: 02/2018. Gilead Sciences, Inc.; Foster City, CA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210251s000lbl.pdf (accessed 1/1/2019).
+
180. +
PifeltroTM (doravirine) tablets for oral use [product information]. Revised: 08/2018. Merck & Co., Inc.; Whitehouse Station, NJ. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210806s000lbl.pdf (accessed 1/1/2019).
+
181. +
DelstrigoTM (doravirine, lamivudine, and tenofovir disoproxil fumarate) tablets for oral use [product information]. Revised: 8/2018. Merck & Co., Inc.; Whitehouse Station, NJ. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210807s000lbl.pdf (accessed Feb. 28, 2019).
+
182. +
Omegaven (fish oil triglycerides) emulsion for intravenous use [product information]. Revised: 7/2018. Fresenius Kabi; Graz, Austria. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/0210589s000lbledt.pdf (accessed 1/1/2019).
+
183. +
Epidiolex® ( cannabidiol ) oral solution, CV [product information]. Revised: 12/2018. GW Research Ltd.; Research Triangle Park, NC. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210365lbl.pdf (accessed 2/21/2019).
+
184. +
FDA approves first drug comprised of an active ingredient derived from marijuana to treat rare, severe forms of epilepsy [FDA News Release]. June 25, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm611046.htm (accessed 1/1/2019).
+
185. +
Reddy  DS, Golub  VM. The pharmacological basis of cannabis therapy for epilepsy. J Pharmacol Exp Ther. 2016;357(1):45–55.
[PubMed: 26787773] .
+
186. +
Ibeas  Bih C, Chen  T, Nunn  AV,  et al. Molecular targets of cannabidiol in neurological disorders. Neurotherapeutics. 2015;12(4):699–730.
[PubMed: 26264914] ; PubMed Central PMCID: PMC4604182.
+
187. +
Zhornitsky  S, Potvin  S. Cannabidiol in humans—the quest for therapeutic targets. Pharmaceuticals (Basel). 2012;5(5):529–552.
[PubMed: 24281562] ; PubMed Central PMCID: PMC3763649.
+
188. +
LokelmaTM (sodium zirconium cyclosilicate) for oral suspension [product information]. Revised: 5/2018. AstraZeneca Pharmaceuticals LP; Wilmington, DE. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/207078s000lbl.pdf (accessed 1/1/2019).
+
189. +
Chaitman  M, Dixit  D, Bridgeman  MB. Potassium-binding agents for the clinical management of hyperkalemia. 2016;41(1):43–50.
[PubMed: 26765867] ; PubMed Central PMCID: PMC4699486.
+
190. +
SYMDEKOTM (tezacaftor/ivacaftor) tablets; (ivacaftor) tablets (copackaged) for oral use [product information]. Revised: 02/2018. Vertex Pharmaceuticals Inc.; Boston, MA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210491lbl.pdf (accessed 1/1/2019).
+
191. +
OrilissaTM (elagolix) tablets for oral use [product information]. Revised: 07/2018. AbbVie Inc.; North Chicago, IL. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210450s000lbl.pdf (accessed 1/1/2019).
+
192. +
Olumiant (baricitinib) tablets for oral use [product information]. Revised: 5/2018. Lilly USA, LLC; Indianapolis, IN. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/207924Orig1s000lbl.pdf (accessed 1/1/2019).
+
193. +
Apadaz (benzhydrocodone and acetaminophen) tablets for oral use, CII [product information]. Revised: 2/2018. Kempharm KemPharm, Inc.; Coralville, IA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/208653s000lbl.pdf (accessed 2/21/2019).
+
194. +
Approved Risk Evaluation and Mitigation Strategies (REMS): Opioid Analgesic REMS: Shared System REMS [webpage]. REMS last update: 09/18/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/scripts/cder/rems/index.cfm?event=RemsDetails.page&REMS=17 (accessed 2/21/2019).
+
195. +
Firdapse® (amifampridine) tablets for oral use [product information]. Revised: 11/2018. Catalyst Pharmaceuticals, Inc.; Coral Gables, FL. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/208078s000lbl.pdf (accessed 1/1/2019).
+
196. +
FDA approves first treatment for Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder [FDA News Release]. November 28, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm627093.htm (accessed Jan. 1, 2019).
+
197. +
AnnoveraTM (segesterone acetate and ethinyl estradiol vaginal system) [product information]. Revised: 08/2018. Population Council New York, NY. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/209627s000lbl.pdf (accessed 1/1/2019).
+
198. +
FDA approves new vaginal ring for one year of birth control [FDA News Release]. August 10, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616541.htm (accessed 1/1/2019).
+
199. +
NuvaRing® (etonogestrel/ethinyl estradiol vaginal ring) [product information]. Organon Inc. West Orange, NJ. Revised: 12/2018. Merck & Co., Inc.; Whitehouse Station, NJ. https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=017343fb-86c4-45ab-9c47-52cc5b9f3a02&type=display (accessed Feb. 28, 2019).
+
200. +
Motegrity (prucalopride) tablets for oral use [product information]. Revised: 12/2018. Shire US Inc.; Lexington, MA. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210166s000lbl.pdf (accessed Jan. 1, 2019).
+
201. +
Drug Safety and Availability: Zelnorm (tegaserod maleate) Information [webpage]. Last updated 7/26/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/DrugSafety/ucm103223.htm (accessed Jan. 18, 2019).
+
202. +
DOPTELET® (avatrombopag) tablets for oral use [product information]. Revised: 5/2018. Dova Pharmaceuticals, Inc.; Durham, NC. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210238s000lbl.pdf (accessed Jan. 1, 2019).
+
203. +
FDA approves new drug for patients with chronic liver disease who have low blood platelets and are undergoing a medical procedure [FDA News Release]. May 21, 2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm608320.htm (accessed Jan. 1, 2019).
+
204. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves avatrombopag for thrombocytopenia in adults with chronic liver disease [webpage]. Last Updated: 05/21/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm608323.htm (accessed Jan. 3, 2019).
+
205. +
Mulpleta® (lusutrombopag tablets) for oral use [product information]. Revised 8/2018. Shionogi Inc.; Florham Park, NJ. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.accessdata.fda.gov/drugsatfda_docs/label/2018/210923s000lbl.pdf (accessed Feb. 21, 2019).
+
206. +
Hematology/Oncology (Cancer) Approvals & Safety Notifications: FDA approves lusutrombopag for thrombocytopenia in adults with chronic liver disease [webpage]. Last Updated: 07/31/2018. Available from: U.S. Department of Health and Human Services, Food and Drug Administration. www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm615348.htm (accessed Jan. 3, 2019).