Olezarsen

Olezarsen, sold under the brand name Tryngolza, is a medication used in the treatment of familial chylomicronemia syndrome. Olezarsen is an apolipoprotein C-III-directed antisense oligonucleotide. It is given by injection under the skin.

Olezarsen

Clinical data
Trade names	Tryngolza
Other names	IONIS-APOCIII-LRX
License data	US DailyMed: Olezarsen
Routes of administration	Subcutaneous
ATC code	None
Legal status
Legal status	US: ℞-only
Identifiers
IUPAC name 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'-O->5'-O)-2'-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3'-O->5'-O)-2'-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3'-O->5'-O)-2'-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3'-O->5'-O)-2'-DEOXY-5-METHYL-P-THIOCYTIDYLYL-(3'-O->5'-O)-P-THIOTHYMIDYLYL-(3'-O->5'-O)-P-THIOTHYMIDYLYL-(3'-O->5'-O)-2'-DEOXY-P-THIOGUANYLYL-(3'-O->5'-O)-P-THIOTHYMIDYLYL-(3'-O->5'-O)-2'-DEOXY-5-METHYL-P-THIOCYTIDYLYL-(3'-O->5'-O)-2'-DEOXY-5-METHYL-P-THIOCYTIDYLYL-(3'-O->5'-O)-2'-DEOXY-P-THIOADENYLYL-(3'-O->5'-O)-2'-DEOXY-P-THIOGUANYLYL-(3'-O->5'-O)-2'-DEOXY-5-METHYL-P-THIOCYTIDYLYL-(3'-O->5'-O)-2'-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3'-O->5'-O)-2'-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3'-O->5'-O)-2'-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3'-O->5'-O)-2'-O-(2-METHOXYETHYL)-P-THIOADENYLYL-(3'-O->5'-O)-2'-O-(2-METHOXYETHYL)-5-METHYLURIDINE
CAS Number	2097587-83-0
DrugBank	DB18728
UNII	S3RS2SA30L

Olezarsen was approved for medical use in the United States in December 2024.

Medical uses

Olezarsen is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome.

History

The US Food and Drug Administration (FDA) granted the application of olezarsen orphan drug designation in February 2024.

Society and culture

Legal status

Olezarsen was approved for medical use in the United States in December 2024.

Names

Olezarsen is the INN.

Olezarsen is sold under the brand name Tryngolza.

Further reading =

• Karwatowska-Prokopczuk, Ewa; Tardif, Jean-Claude; Gaudet, Daniel; Ballantyne, Christie M.; Shapiro, Michael D.; Moriarty, Patrick M.; et al. (2022). "Effect of olezarsen _targeting APOC-III on lipoprotein size and particle number measured by NMR in patients with hypertriglyceridemia". Journal of Clinical Lipidology. 16 (5): 617–625. doi:10.1016/j.jacl.2022.06.005.
• Tardif, Jean-Claude; Karwatowska-Prokopczuk, Ewa; Amour, Eric St; Ballantyne, Christie M; Shapiro, Michael D; Moriarty, Patrick M; et al. (6 April 2022). "Apolipoprotein C-III reduction in subjects with moderate hypertriglyceridaemia and at high cardiovascular risk". European Heart Journal. 43 (14): 1401–1412. doi:10.1093/eurheartj/ehab820.

External links

• "Olezarsen (Code C180652)". NCI Thesaurus.
• Clinical trial number NCT04568434 for "A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) (BALANCE)" at ClinicalTrials.gov