Table 1.
Commercially Approved Oligonucleotide Therapeutics
| Therapeutic | INN name | Class of oligonucleotide | Drug product container | Route of administration | Year approved |
|---|---|---|---|---|---|
| Vitravenea | Fomivirsen | Antisense | Vial | Intravitreal | 1998 |
| Macugen | pegaptanib sodium | RNA aptamer | Prefilled syringe | Intravitreal | 2004 |
| Kynamro | Mimopersen | Antisense | Prefilled syringe | Subcutaneous | 2013 |
| Defitelio | Defibrotide | Polydisperse mix of SS and DS | Vial | Intravenous | 2016 |
| Spinraza | nusinersen | MOE antisense | Vial | Intrathecal | 2016 |
| Exondys 51 | eteplirsen | Morpholino | Vial | Intravenous | 2016 |
| Heplisav-B | Hepatitis B surface antigen | Immunosimulatory | Vial/prefilled syringe | Intramuscular | 2017 |
| Onpattro | patisiran | siRNA | Vial | Intravenous | 2018 |
| Tegsedi | inotersen | Gapmer antisense | Prefilled syringe | Subcutaneous | 2018 |
| Waylivra | volanesorsen | Gapmer antisense | Prefilled syringe | Subcutaneous | 2019 |
| Givlaari | givosiran | siRNA | Vial | Subcutaneous | 2019 |
a
Withdrawn in Europe in 2002 and in the United States in 2006.
DS, double stranded; Inn, international nonproprietary number; MOE, methoxyethyl; siRNA, small/short interfering RNA; SS, single stranded.