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Synonyms: ALN-AS1 | Givlaari®
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.
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Classification  |
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| Compound class | Nucleic acid |
| Compound subclass | siRNA |
| Target | 5'-aminolevulinic acid synthase (ALAS1) mRNA |
| Approved drug? | Yes. FDA (2019) |
| International Nonproprietary Names |
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| INN number | INN |
| 10280 | givosiran |
| Synonyms |
| ALN-AS1 | Givlaari® |
| Database Links |
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| CAS Registry No. | 1639325-43-1 (source: WHO INN record) |
| ChEMBL Ligand | CHEMBL4594265 |
| DrugBank Ligand | DB15066 |
| GtoPdb PubChem SID | 504705433 |
| Search PubMed clinical trials | givosiran |
| Search PubMed titles | givosiran |
| Search PubMed titles/abstracts | givosiran |
