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vutrisiran   Click here for help

GtoPdb Ligand ID: 13616

Synonyms: ALN-65492 | ALN-TTRSC02 | Amvuttra® | votrisiran
Approved drug
vutrisiran is an approved drug (EMA, FDA & UK MHRA (2022))
Compound class: Nucleic acid
Comment: Vutrisiran (ALN-65492) is a GalNAc conjugated double-stranded small interfering ribonucleic acid (siRNA) drug. It acts as a transthyretin (TTR) synthesis inhibitor.
We have been unable to locate a full chemical SMILES for the drug, or its HELM notation.
No information available.
Summary of Clinical Use Click here for help
Vutrisiran was designated as an orphan drug (by the FDA) for the treatment of polyneuropathy in patients with hereditary transthyretin-mediated amyloidosis (hATTR-PN), and was granted full approval for this indication in June 2022. Approvals for hATTR-PN were granted by the EU and UK drug regulatory agencies in the same year. Following further clinical evaluation, vutrisiran was FDA approved to treat ATTR-related cardiomyopathy (ATTR-CM) in March 2025.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT03759379 HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) Phase 3 Interventional Alnylam Pharmaceuticals 1
NCT04153149 HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Phase 3 Interventional Alnylam Pharmaceuticals
External links Click here for help
For extended ADME data see the following:

Drugs.com