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lumasiran   Click here for help

GtoPdb Ligand ID: 13613

Synonyms: AD-65585 | ALN-65585 | ALN-G01 | Oxlumo®
Approved drug
lumasiran is an approved drug (EMA, FDA & UK MHRA (2020))
Compound class: Nucleic acid
Comment: Lumasiran is a double-stranded small interfering ribonucleic acid (siRNA) drug. It targets HAO1 mRNA as a mechanism to reduce the hepatic oxalate overproduction that is responsible for disease pathology in patients with primary hyperoxaluria type 1 (PH1) [1]. We have been unable to locate a full chemical SMILES for the drug, or its HELM notation.
Classification Click here for help
Compound class Nucleic acid
Compound subclass siRNA
Target hydroxyacid oxidase 1 (HAO1)
Approved drug? Yes. UK MHRA (2020)  |  EU EMA (2020)  |  US FDA (2020)
International Nonproprietary Names Click here for help
INN number INN
10684 lumasiran
Synonyms Click here for help
AD-65585 | ALN-65585 | ALN-G01 | Oxlumo®
Database Links Click here for help
CAS Registry No. 1834610-13-7 (source: WHO INN record)
ChEMBL Ligand CHEMBL4297779
DrugBank Ligand DB15935
GtoPdb PubChem SID 504705432
Search PubMed clinical trials lumasiran
Search PubMed titles lumasiran
Search PubMed titles/abstracts lumasiran