Table 1.
Marketed RNA products.
Type | Product | Gene Target | Indication | Approval Year | Company |
---|---|---|---|---|---|
ASO | Vitravene (Fomivirsen) | Cytomegalovirus gene (UL123) | Cytomegalovirus infection | 1998 | Ionis Pharmaceuticals |
Kynamro (Mipomersen) | ApoB-100 | Hypercholesterolemia | 2013 | Ionis Pharmaceuticals | |
Exondys 51 (Eteplirsen) | Dystrophin (exon 51) |
Duchenne muscular dystrophy | 2016 | Sarepta Therapeutics | |
Spinraza (Nusinersen) | SMN2 | Spinal muscular atrophy | 2016 | Ionis Pharmaceuticals | |
Tegsedi (inotersen) | TTR | TTR-mediated amyloidosis | 2018 | Ionis Pharmaceuticals | |
Waylivra (Volanesorsen) | ApoCIII | Familial chylomicronemia syndrome | 2019 | Ionis Pharmaceuticals/Akcea | |
Vyondy 53 (Golodirsen) | Dystrophin (exon 53) |
Duchenne muscular dystrophy | 2019 | Sarepta Therapeutics | |
Viltepso (viltolarsen) | Dystrophin (exon 53) |
Duchenne muscular dystrophy | 2020 | Nippon Shinyaku | |
AMONDYS 45 (casimersen) | Dystrophin (exon 45) |
Duchenne muscular dystrophy | 2021 | Sarepta Therapeutics | |
siRNA | Onpattro (patisiran) | TTR | TTR-mediated amyloidosis | 2018 | Alnylam Pharmaceuticals |
Givlaari (givosiran) | ALAS1 | Acute hepatic porphyrias | 2019 | Alnylam Pharmaceuticals | |
OXLUMO (lumasiran) | Glycolate oxidase | Primary hyperoxaluria type 1 | 2020 | Alnylam Pharmaceuticals | |
Inclisiran (Leqvio) | PCSK9 | Hypercholesterolemia | 2020 | Novartis/ Alnylam Pharmaceuticals | |
Aptamer | Macugen (Pegaptanib) | VEGF-165 | Age-related macular degeneration and diabetic macular edema | 2004 | Eyetech Pfizer |
mRNA | Comirnaty (tozinameran) | SARS-CoV-2 spike protein mRNA | COVID-19 | 2020 | BioNTech/Pfizer |
mRNA-1273 | SARS-CoV-2 spike protein mRNA | COVID-19 | 2020 | Moderna/NIAID/BARDA |
Abbreviation: Apo: Apolipoprotein; SMN2: Survival of motor neuron 2, TTR: Transthyretin, ALAS1: Aminolevulinate synthase 1, PCSK9: Proprotein convertase subtilisin/kexin type 9, VEGF: Vascular endothelial growth factor, COVID-19: Coronavirus disease 2019.