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Olezarsen is an antisense oligonucleotide used in adjunct to diet to reduce triglyceride levels in adults with familial chylomicronemia syndrome. Olezarsen is an antisense oligonucleotide that targets the messenger RNA (mRNA) for apolipoprotein C-III (APOC3, apoC-III), which is a key regulator of triglyceride levels in plasma, in the liver.
- Mechanism
- Curator reviewed · 2 references
Apolipoprotein C-III (APOC3, apoC-III) regulates the uptake of triglyceride-rich lipoproteins in the liver via lipoprotein lipase (LPL)-dependent and LPL-independent mechanisms. Apart from this important role, apoC-III has also been implicated in pathophysiological reactions such as vessel wall inflammation and hypertriglyceridemia, making it a desirable therapeutic target in disease and drug research.
Olezarsen is an ASO-GalNAc3 conjugate that binds to apoC-III mRNA leading to mRNA degradation and resulting in a reduction of serum apoC-III protein. Reduction of apoC-III protein leads to increased clearance of plasma triglycerides and very-low-density lipoprotein (VLDL).
- Primary indication
- Olezarsen is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).Curator reviewed · 3 structured indications
- First approval
- Canada, 2026 · United States, 2024 · European Union, 2025
- Also known as
- ALL-P-AMBO-5'-O-(((6-(5-((TRIS(3-(6-(2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYLOXY)HEXYLAMINO)-3-OXOPROPOXYMETHYL))METHYL)AMINO-5-OXOPENTANAMIDO)HEXYL))PHOSPHO)-2'-O-(2-METHOXYETHYL)-P-THIOADENYLYL-(3'-O->5'-O)-2'-O-(2-METHOXYETHYL)-P-THIOGUANYLYL-(3 · DNA, D(P-THIO)((2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))M5RU-M5C-T-T-G-T-M5C-M5C-A-G-M5C-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETH · IONIS-APOCIII-LRX · ISIS-APOCIII-LRX
- Code names
- AKCEA-APOCIII-LRx · ISIS-678354
- Brand names
- Tryngolza
Resolves to
What you can answer from here — as of September 12, 2026
Which drugs share a target with Olezarsen, and which of those have an active Phase 3 trial?