Table 1 FDA-approved ASO therapeutics
From: Nonviral delivery systems for antisense oligonucleotide therapeutics
Generic Name | Drug | Administration Route | Approval Year | Target | Indication | Applicant |
|---|---|---|---|---|---|---|
VITRAVENE | Fomivirsen | Intravitreal injection | 1998 | Cytomegalovirus | Cytomegalovirus retinitis | IONIS Pharmaceuticals |
MACUGEN | Pegaptanib | Intravitreal injection | 2004 | Vascular endothelial growth factor | Macular degeneration | EYETECH PHARMS |
KYNAMRO | Mipromersen | SC injection | 2013 | Apo B-100 synthesis | Heterozygous familial hypercholesterolemia | KASTLE THERAPS LLC |
SPINRAZA | Nusinersen | Intrathecal injection | 2015 | Mutations in chromosome 5q | Spinal Muscular Atrophy | BIOGEN IDEC |
DEFITELIO | Defibrotide | IV infusion | 2016 | P38 mitogen-activated protein kinase | Sinusoidal obstructive syndrome | JAZZ PHARMS INC |
EXONDYS 51 | Eteplirsen | IV infusion | 2016 | Exon 51 of the dystrophin gene | Duchenne muscular dystrophy | SAREPTA THERAPS INC |
TEGSEDI | Inotersen | SC injection | 2018 | Vascular endothelial growth factor | Macular degeneration | AKCEA THERAPY |
VYONDYS 53 | Golodirsen | IV infusion | 2019 | Exon 53 of the dystrophin gene | Duchenne muscular dystrophy | SAREPTA THERAPS INC |
VILTEPSO | Viltolarsen | IV infusion | 2020 | Exon 53 of the dystrophin gene | Duchenne muscular dystrophy | NIPPON SHINYAKU |
AMONDYS 45 | Casimersen | IV infusion | 2021 | Exon 45 of the dystrophin gene | Duchenne muscular dystrophy | SAREPTA THERAPS INC |