Table 2.

RNA therapeutic drug approved by FDA and/or EMA

Agent	Therapeutic drug	Disease	Mechanism of target gene	Approval agency/year
Oligonucleotides	Defibrotide	Severe hepatic veno-occlusive disease	Adenosine receptors (A1, A2a, A2b)	FDA/2016
	Mipomersen	Familial hypercholesterolemia	Apolipoprotein B mRNA	FDA/2013
	Pegaptanib	Neovascular age-related macular degeneration	Vascular endothelial growth factor 165	FDA/2004
Antisense oligonucleotides	Casimersen	Duchenne muscular dystrophy	Duchenne muscular dystrophy gene (exon 45)	FDA/2021
	Eteplirsen	Duchenne muscular dystrophy	Exclusion of exon 51 from the Duchenne muscular dystrophy mRNA	FDA/2016
	Fomivirsen	Cytomegalovirus retinitis	Cytomegalovirus IE-2 mRNA	FDA/1998
	Golodirsen	Duchenne muscular dystrophy	Dystrophin	FDA/2019
	Nusinersen	Spinal muscular atrophy	Survival motor neuron-2 protein	FDA/2016
	Inotersen	Hereditary transthyretin amyloidosis	Transthyretin mRNA	FDA/2018
	Viltolarsen	Duchenne muscular dystrophy	Duchenne muscular dystrophy gene	FDA/2020
	Volanesorsen	Familial chylomicronaemia syndrome	Apo C-III mRNA	EMA/2019
siRNAs	AMVUTTRA	Amyloidogenic transthyretin amyloidosis	Transthyretin mRNA	FDA/2022
	Givosiran	Acute hepatic porphyria	5-aminolevulinic acid synthase 1 mRNA	FDA/2019
	Inclisiran	Primary hypercholesterolemia	Inhibit hepatic translation proprotein convertase subtilisin-kexin type 9	EMA/2020
				FDA/2021
	Lumasiran	Primary hyperoxaluria type 1	Hydroxyacid oxidase-1	FDA/2020
	Patisiran	Hereditary transthyretin amyloidosis	Transthyretin mRNA	FDA/2018

Apo C-III: Apolipoprotein C-III; EMA: European Medicines Agency; FDA: U.S. Food and Drug Administration.