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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 [4-5].
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.
No information available.
Summary of Clinical Use Click here for help
Fitusiran was evaluated for efficacy as prophylaxis (bleed prevention) in patients with hemophilia A or B [1,6,8]. The US FDA approved fitusiran for hemophilia A/B in March 2025.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT03549871 A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis Phase 3 Interventional Sanofi 3
NCT03417245 A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors Phase 3 Interventional Sanofi 7
NCT03417102 A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors Phase 3 Interventional Sanofi 9