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eChapter 2018: Goodman & Gilman Year in Review New and Noteworthy FDA Approvals

Nelda Murri, PharmD, MBA

PART 1: FIRST-IN-CLASS DRUGS, PHARMACOLOGICAL SIMILARS, BREAKTHROUGH THERAPIES, ORPHAN INDICATIONS, CANCER APPROVALS, AND COMPANION DIAGNOSTIC TESTS

Note: The following symbols are used throughout.

† orphan indications

$ breakthrough therapies

# cancer therapies

biologic license designations

First-In-Class and Pharmacological Similars

In 2018, the U.S. Food and Drug Administration (FDA) granted 46 noteworthy new drug application (NDA) licenses and approved 29 noteworthy new biologic license applications (BLAs). Among the 46 NDA approvals are:

  • Nine pharmacological “firsts,” including two RNA-based gene silencing therapies (inotersen† [an antisense oligonucleotide] and patisiran†$ [a small interfering ribonucleic acid (siRNA) sequence])1 and a “tissue-agnostic” cancer drug, larotrectinib†$# (Table P1-1);2

  • Three ablative agents: dehydrated (99%) alcohol† (Ablysinol)3 used to induce controlled cardiac septal infarct in adults with hypertrophic obstructive cardiomyopathy;4 iobenguane I 131†$# (Azedra)5, a radioablative for pheochromocytoma or paraganglioma; 6,7 and lutetium Lu 177 dotatate†# (Lutathera),8 a radiolabeled somatostatin analog for somatostatin receptor–positive gastroenteropancreatic neuroendocrine tumors;9-11

  • Thirty-three new molecular entities that are pharmacologically similar to previously approved drugs (Table P1-2); and

  • Primatene Mist,12 a chlorofluorocarbon (CFC)-free inhaled epinephrine reformulation for the self-management of mild asthma, reintroduced after a 7-year absence from the market and representing the only OTC metered-dose inhaler available in the United States.13,14

Table P1-1.New Pharmacological Drug Classes Introduced in 2018
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Table P1-1. New Pharmacological Drug Classes Introduced in 2018
Pharmacological Class First to be Marketed in the U.S. FDA Approved Indication Goodman & Gilman's The Pharmacological Basis of Therapeutics, 13th Edition Reference
Polymerase acidic endonuclease inhibitor20,39 Baloxavir marboxil (Xofluza)40,41 Influenza Chapter 62. Antiviral Agents (Nonretroviral) > Anti-influenza Agents
Spleen tyrosine kinase inhibitor42 Fostamatinib disodium hexahydrate (Tavalisse)43 Immune thrombocytopenic purpura† Chapter 67. Pathway-Targeted Therapies: Monoclonal Antibodies, Protein Kinase Inhibitors, and Various Small Molecules
Transthyretin-directed antisense oligonucleotide1 Inotersen (Tegsedi)44 Polyneuropathy of hereditary transthyretin-mediated amyloidosis† Chapter 3. Pharmacodynamics: Molecular Mechanisms of Drug Action > Pharmacotherapies That Modify Specific Genes and Their Transcription and Translation
Transthyretin-directed short interfering RNA1 Patisiran$ (Onpattro)45
Isocitrate dehydrogenase-1 (IDH1) inhibitor46 Ivosidenib# (Tibsovo)47 IDH1 mutated myeloid leukemia† Chapter 67. Pathway-Targeted Therapies: Monoclonal Antibodies, Protein Kinase Inhibitors, and Various Small Molecules
Note: The Abbott RealTime IDH1 somatic gene mutation detection system is FDA approved to detect susceptible IDH1 mutations.34
Tropomyosin receptor kinase (TRK) inhibitor48 larotrectinib#$ (Vitrakvi)49 NTRK gene fusion-positive solid tumors† Chapter 14. Neurotransmission in the Central Nervous System > Figure 14–18
Alpha-galactosidase A chaperone25 Migalastat (Galafold)50 Fabry disease† Chapter 3. Pharmacodynamics: Molecular Mechanisms of Drug ...

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