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fitusiran   Click here for help

GtoPdb Ligand ID: 13630

Synonyms: ALN-57213 | ALN-AT3 | ALN-AT3SC | Qfitlia®
Approved drug
fitusiran is an approved drug
Compound class: Nucleic acid
Comment: Fitusiran is a small interference RNA (siRNA) class agent. It is intended to reduce the antithrombin (SERPINC1) protein level as a mechanism to promote thrombin synthesis and rebalance hemostasis (and prevent bleeds) in patients with hemophilia A or B [5-6].
GalNAc-conjugation is a strategy used to promote delivery of the drug to the liver [2]- this is annotated as 'R1' in the sequence provided here. We have been unable to locate a full chemical SMILES for the ligand, or its HELM notation.
Classification Click here for help
Compound class Nucleic acid
Compound subclass siRNA
Target antithrombin (SERPINC1) mRNA
Approved drug? Yes. US FDA (2025)
International Nonproprietary Names Click here for help
INN number INN
10177 fitusiran
Synonyms Click here for help
ALN-57213 | ALN-AT3 | ALN-AT3SC | Qfitlia®
Database Links Click here for help
CAS Registry No. 1499251-18-1 (source: WHO INN record)
ChEMBL Ligand CHEMBL4297754
DrugBank Ligand DB15002
GtoPdb PubChem SID 504705449
Search PubMed clinical trials fitusiran
Search PubMed titles fitusiran
Search PubMed titles/abstracts fitusiran