Drug intelligence / Profile preview

inclisiran

Development stage
Approved
Lead developer
Novartis
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

Inclisiran is a long-acting small interfering RNA (siRNA) therapeutic used to lower low-density lipoprotein cholesterol (LDL-C) in adults with primary hyperlipidemia, including heterozygous familial hypercholesterolemia and atherosclerotic cardiovascular disease. It works by specifically targeting the mRNA of proprotein convertase subtilisin/kexin type 9 (PCSK9), leading to its degradation via the RNA-induced silencing complex (RISC). This results in decreased hepatic production of PCSK9 protein, increased recycling and expression of LDL receptors on hepatocytes, and enhanced clearance of circulating LDL-C. Inclisiran is administered as a subcutaneous injection every six months and is typically used alongside statins or other lipid-lowering therapies when additional LDL-C reduction is needed. The drug was developed as part of efforts to provide durable cholesterol lowering through gene silencing mechanisms[2][3][4][5][6][7].

Brand names
Leqvio
Other names
inclisiran sodium
02

Targets

PCSK9 (Proprotein convertase subtilisin/kexin type 9)

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