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Synonyms: ALN-60212 | ALN-PCSsc | Leqvio®
inclisiran is an approved drug (EMA (2020), FDA & UK MHRA (2021))
Compound class:
Nucleic acid
Comment: Inclisiran is a small interfering RNA (siRNA) class drug that targets the proprotein convertase subtilisin kexin type 9 (PCSK9) mRNA, to reduce production of the PCSK9 protein [6].
Individual nucleotides are chemically modified to improve stability, and the structure has terminal GalNac (sugar) conjugation for hepatic targeted delivery. We have been unable to locate a full chemical SMILES for the drug, or its HELM notation. |
| No information available. |
Summary of Clinical Use  |
| Used as an adjunct to diet and statin therapy in the treatment of primary hyperlipidemia (including heterozygous familial hypercholesterolemia) [2-3]. Limiting PCSK9 synthesis has the effect of lowering low-density lipoprotein cholesterol (LDL-C) concentrations in the blood, and thereby reduces atherosclerotic cardiovascular disease risk. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT05763875 | Efficacy and Safety of Inclisiran as Monotherapy in Patients With Primary Hypercholesterolemia Not Receiving Lipid-lowering Therapy. | Phase 3 Interventional | Novartis | ||
| NCT06494501 | The Prevent Coronary Artery Disease Trial | Phase 3 Interventional | Icahn School of Medicine at Mount Sinai | ||
| NCT06597006 | Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia | Phase 3 Interventional | Novartis | ||
| NCT03400800 | Inclisiran for Subjects With ASCVD or ASCVD-Risk Equivalents and Elevated Low-density Lipoprotein Cholesterol | Phase 3 Interventional | The Medicines Company | 5 | |
| NCT03399370 | Inclisiran for Participants With Atherosclerotic Cardiovascular Disease and Elevated Low-density Lipoprotein Cholesterol | Phase 3 Interventional | The Medicines Company | 5 | |
| NCT00000189 | Gepirone vs Placebo in Treatment of Cocaine Dependence - 3 | Phase 2 Interventional | University of Pennsylvania | 4 | |
| NCT02963311 | A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | Phase 2 Interventional | The Medicines Company | 1 | |
| External links |
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For extended ADME data see the following: Electronic Medicines Compendium (eMC) Drugs.com |
