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givosiran   Click here for help

GtoPdb Ligand ID: 13614

Synonyms: ALN-AS1 | Givlaari®
Approved drug
givosiran is an approved drug (FDA (2019))
Compound class: Nucleic acid
Comment: Givosiran (ALN-AS1) is a double-stranded N-acetylgalactosamine (GalNAc)-conjugated small interfering ribonucleic acid (siRNA) drug. It inhibits hepatic synthesis of 5'-aminolevulinate synthase 1 as a mechanism to reduce accumulation of toxic heme intermediates in patients with acute hepatic porphyria (which arises from deficient aminolevulinic acid dehydratase and porphobilinogen deaminase activities). GalNAc-conjugation is a strategy used to promote delivery of the drug to the liver [1]- this is annotated as 'R1' in the sequence provided here. 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 5'-aminolevulinic acid synthase (ALAS1) mRNA
Approved drug? Yes. US FDA (2019)
International Nonproprietary Names Click here for help
INN number INN
10280 givosiran
Synonyms Click here for help
ALN-AS1 | Givlaari®
Database Links Click here for help
CAS Registry No. 1639325-43-1 (source: WHO INN record)
ChEMBL Ligand CHEMBL4594265
DrugBank Ligand DB15066
GtoPdb PubChem SID 504705433
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