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
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: