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Table 2 FDA-approved nucleic acid therapeutics as of February 2020

From: Modified nucleic acids: replication, evolution, and next-generation therapeutics

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 FDA (2001) and EMA (2002) withdrawn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]