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

Abbreviations

AAV: adeno-associated virus

aTTP: acquired thrombotic thrombocytopenic purpura

ADC: antibody–drug conjugate

ASO: antisense oligonucleotide

ATTR: transthyretin amyloidosis

ATTR-CM: transthyretin amyloid cardiomyopathy

BCRP: breast cancer–resistant protein

BLAs: biologic license applications

CAR: chimeric antigen receptor

FDA: U.S. Food and Drug Administration

FGFR: fibroblast growth factor

GST: glutathione S transferase

hATTR-PM: polyneuropathy manifestations of hereditary ATTR

HRSA: Health Resources and Services Administration

HSDD: hypoactive sexual desire disorder

LRP: lipoprotein-related protein

MCR: melanocortin receptor

MMAE: monomethyl auristatin E (vedotin)

NDA: new drug application

NES: nuclear export signal

OA: onasemnogene abeparvovec

PV: polatuzumab vedotin

scAAV9: self-complementary AAV serotype 9

sd: single-domain

sdAb: single-domain antibody fragments

SINE: selective inhibitor of nuclear export

siRNA: small interfering double-stranded ribonucleic acid

SMA: spinal muscular atrophy

SMN: survival motor neuron

TTR: transthyretin

UGT: UDP-glucuronosyltransferase

vWF: von Willebrand factor

Wnt: wingless-type mouse mammary virus integration site

XPO1: exportin 1

Note: The following symbols are used throughout.

!accelerated approval (final FDA approval is contingent on demonstration of a clinical benefit in a confirmatory trial)

biologic license designation

$breakthrough therapy

#cancer drug

orphan indication

First-In-Class and Pharmacological Similars

In the first half of 2019, the FDA granted 11 noteworthy NDA licenses and approved 14 noteworthy new BLAs. Among the 11 NDA approvals are:

  • Five pharmacological “firsts” (bremelanotide for female hypoactive sexual desire disorder, erdafitinib!$# a breakthrough for urothelial carcinoma, selinexor!# for multiple myeloma, and tafamidis$ and tafamidis meglumine$ [free acid and salt forms, respectively], a breakthrough for cardiomyopathy of transthyretin-mediated amyloidosis) [Table P1-1]; and

  • Six new molecular entities that are pharmacologically similar to previously approved drugs (alpelisib# for breast cancer, brexanolone$ [C–IV], a breakthrough for postpartum depression, esketamine$ [C–III], a breakthrough nasal spray for adjunctive therapy of treatment-resistant depression, siponimod for multiple sclerosis, solriamfetol [C–IV] for narcolepsy- or obstructive sleep apnea–associated daytime sleepiness, and triclabendazole for fascioliasis [infection with liver flukes, typically Fasciola hepatica]) [Table P1-2].

Table P1-1New Pharmacological Drug Classes Introduced January-June, 2019

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