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. 2021 Mar 24;22(7):3295. doi: 10.3390/ijms22073295

Table 1.

Summary of currently approved oligonucleotide therapeutics.

Drug Names Market Names Companies FDA Approved Indications Drug Modality Mechanisms Targets
fomivirsen Vitravene Ionis Pharmaceuticals, Novartis 1998 Cytomegalovirus (CMV) retinitis ASO Translation block CMV protein IE2
pegaptanib Macugen OSI Pharmaceuticals 2004 Neovascular age-related macular degeneration Aptamer Binding and blocking Heparin-binding domain of VEGF-165
mipomersen Kynamro Kastle Therapeutics, Ionis Pharmaceuticals, Genzyme 2013 Homozygous familial hypercholesterolemia ASO RNase H degradation Apolipoprotein B100
eteplirsen Exondys 51 Sarepta Therapeutics 2016 Duchenne muscular dystrophy ASO Splicing modulation Exon 51 of DMD
nusinersen Spinraza Ionis Pharmaceuticals, Biogen 2016 Spinal muscular atrophy ASO Splicing modulation Exon 7 of SMN2
defibrotide Defitelio Jazz Pharmaceuticals 2016 Veno-occlusive disease in liver Aptamer Binding and activating Adenosine A1/A2 receptor
inotersen Tegsedi Akcea Therapeutics 2018 Polyneuropathy caused by hereditary transthyretin-mediated (hATTR) amyloidosis ASO RNase H degradation Transthyretin
milasen Not applicable Boston Children’s Hospital 2018 Mila Makovec’s CLN7 gene associated with Batten disease ASO Splicing modulation CLN7
patisiran Onpattro Alnylam 2018 Polyneuropathy caused by hATTR amyloidosis siRNA RNAi Transthyretin
golodirsen Vyondys 53 Sarepta Therapeutics 2019 Duchenne muscular dystrophy ASO Splicing modulation Exon 53 of DMD
givosiran Givlaari Alnylam 2019 Acute hepatic porphyria (AHP) siRNA RNAi 5-aminolevulinic acid synthase
volanesorsen Waylivra Akcea Therapeutics EMA approved in 2019 1 Familial chylomicronemia syndrome (FCS) ASO RNase H degradation Apolipoprotein C3
viltolarsen Viltepso NS Pharma 2020 Duchenne muscular dystrophy ASO Splicing modulation Exon 53 of DMD
casimersen Amondys 45 Sarepta Therapeutics 2021 Duchenne muscular dystrophy ASO Splicing modulation Exon 45 of DMD

1 Volanesorsen did not receive FDA approval in 2018 but was approved by European Medicines Agency (EMA) in 2019.