Table 3 Regulatory agency-approved RNA modulating RNA drugs
Drug type | Generic name | Brand name | Originator company | Approval year | Regulatory agency | Disease | Route | Target-based actions |
|---|---|---|---|---|---|---|---|---|
ASO | Eteplirsen | Exondys 51 | Sarepta Therapeutics | 2016 | FDA | Duchenne muscular dystrophy (DMD) | i.v. injection | DMD gene modulator |
ASO | Nusinersen | Spinraza | Ionis Pharmaceuticals and Biogen | 2016 | FDA | Spinal muscular atrophy | Intrathecal injection | Survival motor neuron-2 (SMN2) gene modulator |
ASO | Inotersen | Tegsedi | Ionis Pharmaceuticals | 2018 | FDA | Hereditary transthyretin mediated amyloidosis | s.c. injection | Transthyretin (TTR) gene inhibitor |
siRNA | Patisiran | Onpattro | Alnylam Pharmaceuticals | 2018 | FDA | Amyloidosis, familial amyloid neuropathy, lipotoxic cardiomyopathy | i.v. injection | Amyloid protein deposition inhibitor, TTR gene inhibitor |
siRNA | Givosiran | Givlaari | Alnylam Pharmaceuticals | 2019 | FDA | Acute intermittent porphyria, hepatic porphyria | s.c. injection | 5-Aminolevulinate synthase 1 inhibitor |
ASO | Golodirsen | Vyondys 53 | Sarepta Therapeutics | 2019 | FDA | DMD | i.v. injection | DMD gene modulator |
siRNA | Lumasiran | Oxlumo | Alnylam Pharmaceuticals | 2020 | FDA | Hyperoxaluria | s.c. injection | Hydroxyacid oxidase 1 modulator |
ASO | Casimersen | Amondys 45 | Sarepta Therapeutics | 2021 | FDA | DMD | i.v. injection | DMD gene modulator |
siRNA | Inclisiran | Leqvio | Novartis | 2021 | FDA | Primary hypercholesterolemia | s.c. injection | Proprotein convertase subtilisin-kexin type 9 (PCSK9) inhibitor |
siRNA | Vutrisiran | Amvuttra | Alnylam Pharmaceuticals | 2022 | FDA | Familial amyloid neuropathy, Stargardt disease | s.c. injection | TTR gene inhibitor |