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eplontersen   Click here for help

GtoPdb Ligand ID: 13606

Synonyms: AKCEA-TTR-LRx | Ionis-TTR-LRx | Wainua® | Wainzua®
Approved drug
eplontersen is an approved drug
Compound class: Nucleic acid
Comment: Eplontersen is a ligand-conjugated transthyretin (TTR)-directed antisense oligonucleotide. It was designed to reduce the hepatic expression of transthyretin protein and pathological TTR amyloid accumulation in patients with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN).

Full sequence as provided in the agent's INN record is: all-P-ambo-5'-O-(28-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-16,16-bis{[3-({6-[(2-acetamido-2-deoxy-β-D -galactopyranosyl)oxy]hexyl}amino)-3-oxopropoxy]methyl}- 1-hydroxy-1,10,14,21-tetraoxo-2,18-dioxa-9,15,22-triaza-1λ 5-phosphaoctacosan-1-yl)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methylcytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyluridylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyluridylyl-(3'→5')-2'-O-(2-methoxyethyl)guanylyl-(3'→5')-2'-deoxy-P-thioguanylyl-(3'→5')-P-thiothymidylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-deoxy-5-methyl-P-thiocytidylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-P-thiothymidylyl-(3'→5')-2'-deoxy-P-thioguanylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-deoxy-P-thioadenylyl-(3'→5')-2'-O-(2-methoxyethyl)adenylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyluridylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'→5')-2'-O-(2- methoxyethyl)-5-methylcytidine, but we have been unable to resolve this to a SMILES or HELM notation.
No information available.
Summary of Clinical Use Click here for help
Eplontersen was designated as an orphan medicine for the treatment of transthyretin-mediated amyloidosis (ATTRv) by the EMA in October 2023. It was FDA approved in December 2023, to treat adults with (ATTRv). The UK MHRA authorised use of eplontersen for this indication in October 2024, and EU EMA approval followed in March 2025.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT04136184 NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Phase 3 Interventional Ionis Pharmaceuticals, Inc. 2
NCT01737398 Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy Phase 2/Phase 3 Interventional Ionis Pharmaceuticals, Inc. 1
External links Click here for help
For extended ADME data see the following:

Drugs.com
European Medicines Agency (EMA)