olezarsen

GtoPdb is requesting financial support from commercial users. Please see our sustainability page for more information.

Home
Ligands
olezarsen

GtoPdb Ligand ID: 13658

Synonyms: AKCEA-APOCIII-LRX | IONIS-APOCIII-LRX | ISIS-678354 | ISIS-APOCIII-LRX | Tryngolza®

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 [2-4,6]. 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.

References
1. Bergmark BA, Marston NA, Prohaska TA, Alexander VJ, Zimerman A, Moura FA, Kang YM, Murphy SA, Zhang S, Lu MT et al.. (2025) Olezarsen in patients with hypertriglyceridemia at high cardiovascular risk: Rationale and design of the Essence-TIMI 73b trial. Am Heart J, 286: 116-124. [PMID:40081744]
2. Chebli J, Larouche M, Gaudet D. (2024) APOC3 siRNA and ASO therapy for dyslipidemia. Curr Opin Endocrinol Diabetes Obes, 31 (2): 70-77. [PMID:38334488]
3. Hooper AJ, Bell DA, Burnett JR. (2024) Olezarsen, a liver-directed APOC3 ASO therapy for hypertriglyceridemia. Expert Opin Pharmacother, 25 (14): 1861-1866. [PMID:39305266]
4. Shamsudeen I, Hegele RA. (2022) Safety and efficacy of therapies for chylomicronemia. Expert Rev Clin Pharmacol, 15 (4): 395-405. [PMID:35762447]
5. Syed YY. (2025) Olezarsen: First Approval. Drugs, 85 (4): 571-576. [PMID:40074987]
6. Valdivielso P, Coca Prieto I. (2024) Apolipoprotein C-III, familial chylomicronemia syndrome, and olezarsen. Med, 5 (6): 493-494. [PMID:38878767]

References

1. Bergmark BA, Marston NA, Prohaska TA, Alexander VJ, Zimerman A, Moura FA, Kang YM, Murphy SA, Zhang S, Lu MT et al.. (2025)
Olezarsen in patients with hypertriglyceridemia at high cardiovascular risk: Rationale and design of the Essence-TIMI 73b trial.
Am Heart J, 286: 116-124. [PMID:40081744]

2. Chebli J, Larouche M, Gaudet D. (2024)
APOC3 siRNA and ASO therapy for dyslipidemia.
Curr Opin Endocrinol Diabetes Obes, 31 (2): 70-77. [PMID:38334488]

3. Hooper AJ, Bell DA, Burnett JR. (2024)
Olezarsen, a liver-directed APOC3 ASO therapy for hypertriglyceridemia.
Expert Opin Pharmacother, 25 (14): 1861-1866. [PMID:39305266]

4. Shamsudeen I, Hegele RA. (2022)
Safety and efficacy of therapies for chylomicronemia.
Expert Rev Clin Pharmacol, 15 (4): 395-405. [PMID:35762447]

5. Syed YY. (2025)
Olezarsen: First Approval.
Drugs, 85 (4): 571-576. [PMID:40074987]

6. Valdivielso P, Coca Prieto I. (2024)
Apolipoprotein C-III, familial chylomicronemia syndrome, and olezarsen.
Med, 5 (6): 493-494. [PMID:38878767]