Table 7 Approved small nucleic acid-based therapies
Target | Generic name | Brand name | Company | Mechanism | Delivery system/Chemistry | Delivery route | Disease | Approved |
|---|---|---|---|---|---|---|---|---|
CMV IE2 | Fomivirsen | Vitravene® | Novartis | ASO | 2ʹ-deoxy | Intravitreal injection | CMV retinitis | 1988 USA (Delist) |
VEGF | Pegaptanib | Macugen® | Pfizer/Eyetech | Aptamer | Pegylated | Intravitreal injection | AMD | 2004 USA (Delist) |
ApoB-100 | Mipomersen (delist) | Kynamro® | Kynamro | ASO | 2ʹ-O-methoxyethyl | Subcutaneous injection | HoFH | 2013 USA |
DMD | Eteplirsen | Exondys 51® | Sarepta | ASO | PMO | Intravenous injection | DMD | 2016 USA |
SMN2 | Nusinersen | Spinraza® | Biogen | ASO | 2ʹ-O-methoxyethyl | Intravenous injection | SMA | 2016 USA 2019 CN |
TTR | Inotersen | Tegsedi® | Ionis Pharmaceuticals | ASO | 2ʹ-O-methoxyethyl | Subcutaneous injection | hATTR | 2018 USA 2018 EUR |
TTR | Patisiran | Onpattro® | Alnylam | siRNA | LNP | Intravenous injection | hATTR | 2018 USA 2018 EUR |
ALAS1 | Givosiran | Givlaari® | Alnylam | siRNA | GalNAc | Subcutaneous injection | AHP | 2019 USA |
DMD | Golodirsen | Vyondys 53® | Sarepta | ASO | PMO | Intravenous injection | DMD | 2019 USA |
ApoC-3 | Volanesorsen | Waylivra® | Ionis Pharmaceuticals | ASO | 2ʹ-O-methoxyethyl | Subcutaneous injection | FCS | 2019 EUR |
DMD | Viltolarsen | Viltepso® | Nippon Shinyaku Co | ASO | PMO | Intravenous injection | DMD | 2020 USA 2020 JPN |
HAO1 | Lumasiran | Oxlumo® | Alnylam | siRNA | GalNAc | Subcutaneous injection | PH1 | 2020 USA 2020 EUR |
PCSK9 | Inclisiran | Leqvio® | Alnylam Novartis | siRNA | GalNAc | Subcutaneous injection | CVD | 2020 EUR 2021 USA |
DMD | Casimersen | Amondys45® | Sarepta | ASO | PMO | Intravenous injection | DMD | 2021 USA |
TTR | Vutrisiran | Amvuttra® | Alnylam | siRNA | GalNAc | Subcutaneous injection | hATTR | 2022 USA |
C5 | Avacincaptad pegol | Izervay® | Iveric Bio | Aptamer | Naked | Intravitreal injection | GA | 2023 USA |
SOD1 | Tofersen | Qalsody® | Biogen | ASO | Naked | Spinal cord injection | ALS | 2023 USA |
TTR | Eplontersen | Wainua® | Ionis Pharmaceuticals | ASO | GalNAc | Subcutaneous injection | hATTR | 2023 USA |
LDHA | Nedosiran | Rivfloza® | NovoNordisk | siRNA | GalNAc | Subcutaneous injection | PH1 | 2023 USA |
