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Synonyms: AD-51547 | ALN-TTRSC
Compound class:
Nucleic acid
Comment: Revusiran is a double-stranded small interfering ribonucleic acid (siRNA) class ligand. It is proposed to reduce hepatic transthyretin (TTR) production, as a mechanism to treat hereditary transthyretin-mediated (hATTR) amyloidosis-related cardiomyopathy.
Nucleic acid bases are chemically modified to improve stability and GalNAc-conjugation is 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 ligand, or its HELM notation. |
| No information available. |
Summary of Clinical Use  |
| Clinical development of revusiran was discontinued at phase 3 due to an observed mortality imbalance between treatment arms, although no causative mechanism has been reported. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT02292186 | A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis | Phase 2 Interventional | Alnylam Pharmaceuticals | ||
| NCT02319005 | ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Phase 3 Interventional | Alnylam Pharmaceuticals | ||
| NCT02595983 | The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant | Phase 2 Interventional | Alnylam Pharmaceuticals | ||
