Table 1.
Summary of Approved Oligonucleotide Therapeutics (as of November 30, 2020)
| Generic name | Trade name | Type | Target | Indication | First approved |
|---|---|---|---|---|---|
| Fomivirsen | Vitravene | Antisense | CVM IE2 mRNA | Cytomegalovirus retinitis in patient with AIDS | US: 1998; EU: 1999 |
| Pegaptanib | Macugen® | Aptamer | VEGF protein | Neovascular (wet) age-related macular degeneration | US: 2004; EU: 2006; JPN: 2008 |
| Mipomersen sodium | Kynamro® | Antisense | ApoB-100 mRNA | Homozygous familial hypercholesterolemia | US: 2013 |
| Eteplirsen | Exondys 51 | Antisense | Dystrophin pre-mRNA | Duchenne muscular dystrophy | US: 2016 |
| Nusinersen | Spinraza® | Antisense | SMN2 pre-mRNA | Spinal muscular atrophy | US: 2016; EU: 2017; JPN: 2017 |
| Inotersen | Tegsedi | Antisense | TTR mRNA | Polyneuropathy of hereditary transthyretin-mediated amyloidosis | US: 2018; EU: 2018 |
| Patisiran | Onpattro® | siRNA | TTR mRNA | Polyneuropathy of hereditary transthyretin-mediated amyloidosis | US: 2018; EU: 2018; JPN: 2019 |
| Viltolarsen | Viltepso | Antisense | Dystrophin pre-mRNA | Duchenne muscular dystrophy | JPN: 2020; US: 2020 |
| Lumasiran | Oxlumo | siRNA | Glycolate oxidase mRNA | Primary hyperoxaluria type 1 | US: 2020; EU: 2020 |
mRNA, messenger RNA; siRNA, small interfering RNA.