Olezarsen

Last updated

Olezarsen
Olezarsen sodium.svg
Clinical data
Trade names Tryngolza
Other namesIONIS-APOCIII-LRX
License data
Routes of
administration
Subcutaneous
Drug class Antisense oligonucleotide
ATC code
  • None
Legal status
Legal status
Identifiers
  • 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
DrugBank
UNII
KEGG

Olezarsen, sold under the brand name Tryngolza, is a medication used in the treatment of familial chylomicronemia syndrome. [1] [2] Olezarsen is an apolipoprotein C-III-directed antisense oligonucleotide. [1] It is given by injection under the skin. [1]

Contents

Olezarsen was approved for medical use in the United States in December 2024. [1] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4]

Medical uses

Olezarsen is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome. [1]

History

The US Food and Drug Administration (FDA) granted the application of olezarsen orphan drug designation in February 2024. [5]

Society and culture

Olezarsen was approved for medical use in the United States in December 2024. [3] [6]

Names

Olezarsen is the international nonproprietary name. [7]

Olezarsen is sold under the brand name Tryngolza. [1]

References

  1. 1 2 3 4 5 6 7 "Tryngolza- olezarsen sodium injection, solution". DailyMed. 19 December 2024. Retrieved 25 January 2025.
  2. Spagnuolo, Catherine M; Hegele, Robert A (2023). "Recent advances in treating hypertriglyceridemia in patients at high risk of cardiovascular disease with apolipoprotein C-III inhibitors". Expert Opinion on Pharmacotherapy. 24 (9): 1013–1020. doi: 10.1080/14656566.2023.2206015 . PMID   37114828.
  3. 1 2 "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 1 October 2024. Retrieved 20 December 2024.
  4. New Drug Therapy Approvals 2024 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2025. Archived from the original on 21 January 2025. Retrieved 21 January 2025.
  5. "Olezarsen Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). Retrieved 20 December 2024.
  6. "Tryngolza (olezarsen) approved in U.S. as first-ever treatment for adults living with familial chylomicronemia syndrome as an adjunct to diet" (Press release). Ionis Pharmaceuticals. 19 December 2024. Retrieved 20 December 2024 via PR Newswire.
  7. World Health Organization (2022). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87". WHO Drug Information. 36 (1). hdl: 10665/352794 .

Further reading