Table 1.
List of FDA approved oligonucleotide drugs.
| Name | Type | Modification | Mechanism | Indication/Target | FDA Approval |
|---|---|---|---|---|---|
| Fomivirsen | ASO | 21 nt PS DNA | RNase H1 | Cytomegalovirus retinitis/CMV UL123 | Aug 1998 |
| Pegaptanib | Aptamer | 27 nt 2ʹ-F/2ʹ-OME PEGylated | Blocking binding | Neovascular age-related macular degeneration/VEGF-165 | Dec 2004 |
| Mipomersen | ASO Gapmer | 20 nt PS 2ʹ-MOE | RNase H1 | Homozygous familial Hypercholesterolemia/APOB |
Jan 2013 |
| Defibrotide | ssDNA and dsDNA | Mixture of PO | Non single sequence dependent based mechanism | hepatic veno-occlusive disease |
Mar 2016 |
| Nusinersen | Steric block ASO | 18 nt PS 2′-MOE | Splicing, intron 7 | Spinal muscular atrophy/SMN2 exon 7 | Dec 2016 |
| Eteplirsen | Steric block ASO | 30 nt PMO | Splicing, exon 51 | Duchenne muscular dystrophy/DMD exon 51 | Sep 2016 |
| Milasen | ASO | 22 nt PS 2′-MOE | Splicing | Batten disease/CLN7 | Jan 2018 |
| Patisiran | siRNA LNP formulation | 19 + 2 nt 2′-OME | Ago2 | Hereditary transthyretin amyloidosis, polyneuropathy-TTR |
Aug 2018 |
| Inotersen | Gapmer ASO | 20 nt PS 2ʹ-MOE | RNase H1 | hereditary transthyretin amyloidosis, polyneuropathy/TTR |
Oct 2018 |
| Givosiran | Dicer substrate siRNA | 21/23 nt- GalNAc conjugate | Ago2 | Acute hepatic porphyria ALAS1 | Nov 2019 |
| Golodirsen | Steric block ASO | 25 nt PMO | Splicing, exon 53 | Duchenne muscular dystrophy/DMD exon 53 | Dec 2019 |
| Viltolarsen | ASO | 21 nt-PMO | Splicing | Duchenne muscular dystrophy/DMD exon 53 | Aug 2020 |
| Casimersen | ASO | 22-PMO | Splicing | Duchenne muscular dystrophy/DMD Exon 45 | Feb 2021 |
| Inclisiran | siRNA | 21/23 nt- GalNAc conjugate | Ago2 | Hypercholesterolaemia/PCSKK9 | Dec 2021 |