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Synonyms: ALN-57213 | ALN-AT3 | ALN-AT3SC
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  |
| Fitusiran is being evaluated for efficacy as prophylaxis (bleed prevention) in patients with hemophilia A or B [1,6,8]. |
| 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 | |
