Table 2.

FDA-approved nucleic acid therapeutics as of February 2020

Drug name (trade name)	Target	Modifications	Mechanism	Indication	Approval	Ref.
Fomivirsen (Vitravene)	mRNA of the CMV immediate-early (IE)-2 protein	PS	ASO (translation blocking)	Cytomegalovirus retinitis (CMV)	FDA (1998) and EMA (1999) approved. FDA (2001) and EMA (2002) withdrawn	[174]
Pegaptanib (Macugen)	Vascular endothelial growth factor (VEGF165)	2′F, 2′OMe, PEG conjugate	Aptamer	Neovascular (wet) age-related macular degeneration	FDA approved (2004)	[175]
Mipomersen (Kynamro)	Apolipoprotein B-100 mRNA	2′MOE, PS, 5mC	ASO (RNase H)	Homozygous familial hypercholesterolemia	FDA approved (2013)	[176]
Eteplirsen (Exondys 51)	Exon 51 in dystrophin mRNA	PMO	ASO (splicing modulation)	Duchenne muscular dystrophy	FDA approved (2016)	[177]
Nusinersen (Spinraza)	Survival of motor neuron 2 (SMN2) pre-mRNA	2′MOE, PS, 5mC	ASO (splicing modulation)	Spinal muscular atrophy	FDA (2016) and EMA (2017) approved	[178]
Patisiran (Onpattro)	Transthyretin (TTR) mRNA	2′OMe	siRNA	Hereditary transthyretin-mediated amyloidosis	FDA and EMA approved (2018)	[179]
Inotersen (Tegsedi)	Transthyretin (TTR) mRNA	2′MOE, PS, 5mC	ASO (RNase H)	Hereditary transthyretin-mediated amyloidosis	FDA and EMA approved (2018)	[180]
Volanesorsen (Waylivra)	Apolipoprotein C3 (apo-CIII) mRNA	2′MOE, PS, 5mC	ASO (RNase H)	Familial chylomicronemia syndrome	EMA approved (2019)	[181]
Givosiran (Givlaari)	Aminolevulinate synthase 1 (ALAS1) mRNA	PS, 2′F, 2′OMe, GalNAc conjugate	siRNA	Acute hepatic porphyria	FDA approved (2019)	[182]
Golodirsen (Vyondys 53)	Exon 53 in dystrophin mRNA	PMO	ASO (splicing modulation)	Duchenne muscular dystrophy	FDA approved (2019)	[2, 183]