Olezarsen

Olezarsen
Clinical data
Trade namesTryngolza
Other namesIONIS-APOCIII-LRX
AHFS/Drugs.comMonograph
MedlinePlusa625020
License data
Routes of
administration		Subcutaneous
Drug classAntisense 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] It is given by injection under the skin.[1] Olezarsen is an apolipoprotein C-III-directed antisense oligonucleotide.[1][3]

The most common side effects include injection site reactions, low platelet counts, and joint pain.[3]

Olezarsen was approved for medical use in the United States in December 2024.[3][4] The US Food and Drug Administration considers it to be a first-in-class medication.[5]

Medical uses

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

Adverse effects

The most common side effects include injection site reactions, low platelet counts, and joint pain.[3] There are some reports of allergic (hypersensitivity) reactions, including difficulty breathing, rash, facial swelling, hives, chills, and muscle aches.[3]

Pharmacology

Olezarsen is an apolipoprotein C-III-directed antisense oligonucleotide.[1] By binding to apolipoprotein C-III mRNA, it causes its degradation, which in turn increases clearance of plasma triglycerides and very low-density lipoprotein (VLDL).[6]

History

The US Food and Drug Administration (FDA) granted the application of olezarsen orphan drug designation in February 2024.[7] In August 2024, the European Medicines Agency granted olezarsen an orphan drug designation.[8]

The FDA approved olezarsen based on evidence from a clinical trial (trial 1; NCT04568434) of 66 participants with familial chylomicronemia syndrome.[3] The trial was conducted at 29 sites in 11 countries including Canada, France, Italy, Netherlands, Norway, Portugal, Slovakia, Spain, Sweden, the United Kingdom, and the United States.[3] Of the 66 participants, 19 participants were from trial sites in the United States.[3] The benefits and side effects of olezarsen for participants with familial chylomicronemia syndrome were evaluated in the same single clinical trial.[3] Additional trials in participants with hypertriglyceridemia were used to support the safety assessment.[3] The number of participants representing efficacy findings may differ from the number of participants representing safety findings due to different pools of study participants analyzed for efficacy and safety.[3] Enrolled participants were already using other treatments to lower triglycerides, including a low-fat diet and medications (such as fenofibrates, omega-3 fatty acids, and statins).[3] Participants were randomly assigned to receive olezarsen or placebo every four weeks for one year.[3] Neither the participants nor the health care providers knew which treatment was being given.[3]

Society and culture

Olezarsen was approved for medical use in the United States in December 2024..[3][4][9]

Names

Olezarsen is the international nonproprietary name.[10]

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

References

  1. ^ a b c d e f g "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. ^ a b c d e f g h i j k l m n o "Drug Trials Snapshot: Tryngolza". U.S. Food and Drug Administration. 19 December 2024. Retrieved 24 June 2025. This article incorporates text from this source, which is in the public domain.
  4. ^ a b "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 1 October 2024. Retrieved 20 December 2024.
  5. ^ 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.
  6. ^ Stroes, Erik S.G.; Alexander, Veronica J.; Karwatowska-Prokopczuk, Ewa; Hegele, Robert A.; Arca, Marcello; Ballantyne, Christie M.; et al. (16 May 2024). "Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome". New England Journal of Medicine. 390 (19): 1781–1792. doi:10.1056/NEJMoa2400201.
  7. ^ "Olezarsen Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). Archived from the original on 27 December 2024. Retrieved 20 December 2024.
  8. ^ "EU/3/24/2973 - orphan designation for treatment of familial chylomicronaemia syndrome". European Medicines Agency (EMA). 21 August 2024. Retrieved 22 February 2025.
  9. ^ "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.
  10. ^ 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

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