olezarsen   Click here for help

GtoPdb Ligand ID: 13658

Synonyms: AKCEA-APOCIII-LRX | IONIS-APOCIII-LRX | ISIS-678354 | ISIS-APOCIII-LRX | Tryngolza®
Approved drug
olezarsen is an approved drug (FDA (2024))
Compound class: Nucleic acid
Comment: Olezarsen is an antisense GalNAc3-conjugated oligonucleotide (ASO) that targets the messenger RNA for apolipoprotein C-III (APOC3) as a mechanism to treat dyslipidemias [1-3,5]. The nucleotide sequence is chemically modified to optimise in vivo stability and hepatic delivery. We have been unable to resolve a full SMILES or HELM notation for the entire ASO.
No information available.
Summary of Clinical Use Click here for help
The FDA approved olezarsen in December 2024, to treat familial chylomicronemia syndrome [4]. It is also being tested for efficacy in atherosclerotic cadiovascular diseases and other dyslipidemias.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT05130450 A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) Phase 3 Interventional Ionis Pharmaceuticals, Inc.
NCT05185843 A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen Phase 3 Interventional Ionis Pharmaceuticals, Inc.
NCT05610280 A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia Phase 3 Interventional Ionis Pharmaceuticals, Inc.
NCT05681351 CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG) Phase 3 Interventional Ionis Pharmaceuticals, Inc.