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eChapter 69 (Part 1): The Goodman & Gilman Year in Review: 2013 New and Noteworthy FDA Approvals

Nelda Murri

PART 1 FIRST-IN-CLASS AGENTS

The FDA approved 40 new drugs and biologics of note in 2013. Twenty-two are pharmacologically similar to others already marketed and 10 are newly licensed biologics (see part 2 of this series). The remainder represent eight first-in-class agents indicated for the management of cancer, diabetes, hepatitis C infection, hypercholesterolemia, multiple sclerosis, and pulmonary hypertension (Table 69-1). Three drugs, ibrutinib (IMBRUVICA) for lymphoma (Table 69-1), obinutuzumab (GAZYVA) for chronic lymphocytic leukemia (see part 2 of this series), and sofosbuvir (SOVALDI) for hepatitis C infection (Table 69-1), were the first drugs to be approved under FDA's "breakthrough therapy" designation.1 Established in 2012, FDA reserves breakthrough designations for drugs exhibiting superior efficacy against serious diseases.

Table 69-1.New Pharmacological Drug Classes Introduced in 2013
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Table 69-1. New Pharmacological Drug Classes Introduced in 2013
Pharmacological Class First to be Marketed in the U.S. FDA Approved Indication Goodman & Gilman's The Pharmacological Basis of Therapeutics, 12e Reference
sodium-glucose co-transporter 2 (SGLT2) inhibitor* canagliflozin* (invokana) type 2 diabetes mellitus Chapter 43. Endocrine Pancreas And Pharmacotherapy Of Diabetes Mellitus And Hypoglycemia > Drugs in Development for Type 2 Diabetes; also see SGLT2 Inhibitors – An Update on Canagliflozin12
nuclear factor (erythroid-derived 2)-like 2 (Nrf2) transcriptional pathway activator

dimethyl fumarate

(tecfidera)

 multiple sclerosis; Friedreich's Ataxia† Chapter 35. Immunosuppressants, Tolerogens, and Immunostimulants; also see Dimethyl Fumarate (tecfidera): A New Small Molecule Disease-Modifying Drug for Multiple Sclerosis13
Bruton tyrosine kinase (BTK) inhibitor

ibrutinib†$

(imbruvica)

 mantle cell lymphoma; Waldenström macroglobulinemia Chapter 62. Targeted Therapies: Tyrosine Kinase Inhibitors, Monoclonal Antibodies, and Cytokines
apolipoprotein B-100 synthesis inhibitor

mipomersen†

(kynamro)

 homozygous familial hypercholesterolemia Chapter 31. Drug Therapy For Hypercholesterolemia And Dyslipidemia; also see Mipomersen: An antisense oligonucleotide for lowering LDL-cholesterol in patients with homozygous familial hypercholesterolemia14
alpha particle–emitting isotope

radium Ra 223 dichloride

(xofigo)

 prostate cancer Chapter 60. General Principles of Cancer Chemotherapy
soluble guanylate cyclase (sGC) activator*

riociguat†

(adempas)

 pulmonary hypertension Chapter 28. Pharmacotherapy of Congestive Heart Failure > Direct Activators of Soluble Guanylyl Cyclase; Chapter 36. Pulmonary Pharmacology
NS5B polymerase inhibitor

sofosbuvir$

(sovaldi)

 hepatitis C infection Chapter 58. Antiviral Agents (Nonretroviral)
MEK inhibitor

trametinib †

(mekinist)

 melanoma Chapter 3. Pharmacodynamics: Molecular Mechanisms of Drug Action > Receptor Tyrosine Kinases; also see Trametinib: a first in class oral MEK inhibitor approved for treatment of metastatic melanoma with BRAF V600 mutations15 Chapter 62. Targeted Therapies: Tyrosine Kinase Inhibitors, Monoclonal Antibodies, and Cytokines

*see specific mention in 12e

† Orphan drug

$ Breakthrough drug designation

Among previously approved drugs, one product, pertuzumab (PERJETA), became the first FDA-approved neoadjuvant treatment for breast cancer.2 Neoadjuvant treatments are given as a first step to shrink a tumor before the main treatment (usually surgery) is given. Another new product, clinolipid (see part 2 of this series) was granted a priority review to help alleviate the long-standing national shortage ...

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