. 2023 Jul 10;55(7):1283–1292. doi: 10.1038/s12276-023-00998-y

Table 1.

siRNA drugs approved by the FDA as of 2022.

Drug/Trade name	Date of Approval	siRNA Carrier	Routes of administration	Indication and usage	Target organ	Target gene	Reference
Patisiran/Onpattro	August 10, 2018	Lipid nanoparticles	intravenous	Adult patients with hereditary transthyretin mediated (hATTR) amyloidosis	Liver	transthyretin (TTR)	²³
Givosiran/Givlaari	November 20, 2019	GalNAc-conjugation	subcutaneous	Adult patients with acute hepatic porphyria (AHP)	Liver	aminolevulinate synthase 1 (ALAS1)	²⁴
Lumasiran/Oxlumo	November 23, 2020	GalNAc-conjugation	subcutaneous	Adult and pediatric patients with primary hyperoxaluria type 1 (PH1)	Liver	hydroxy acid oxidase 1 (HAO1)	²⁵
Inclisiran/Leqvio	December 21, 2021	GalNAc-conjugation	subcutaneous	Adult patients with heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease.	Liver	proprotein convertase subtilisin/kexin type 9 (PCSK9)	²⁶
Vutrisiran/amvuttra	June 13, 2022	GalNAc-conjugation	subcutaneous	Adult patients with hereditary transthyretin mediated (hATTR) amyloidosis	Liver	transthyretin (TTR)	²⁷