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

ASO antisense oligonucleotide, siRNA small interfering RNA, FDA Food and Drug Administration, United States, i.v. intravenous, s.c. subcutaneous injection