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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 U.S. Food and Drug Administration (FDA) granted 11 noteworthy new drug application (NDA) licenses and approved 14 noteworthy new biologic license applications (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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