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 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]

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).[5]

Adverse effects

In a 66-patient trial, olezarsen was demonstrated to cause following side effects[5][6]:

  • injection site reactions
  • hypersensitivity reactions (due to immunogenic potential of the medication)
  • arthralgia
  • thrombocytopenia
  • hyperglycemia
  • elevation of liver enzymes

History

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

Society and culture

Olezarsen was approved for medical use in the United States in December 2024.[3][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 "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. ^ a b 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. ISSN 0028-4793.
  6. ^ Ionis Pharmaceuticals, Inc. (11 December 2024). A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS) (Report). clinicaltrials.gov.
  7. ^ "Olezarsen Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). Retrieved 20 December 2024.
  8. ^ "EU/3/24/2973 - orphan designation for treatment of familial chylomicronaemia syndrome | European Medicines Agency (EMA)". www.ema.europa.eu. 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

Allikas: https://en.wikipedia.org/wiki/Olezarsen
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