Chemistry:Olezarsen

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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] Olezarsen is an antisense oligonucleotide which inhibits the formation of apolipoprotein C3 (apoC-III), a protein that regulates both triglyceride metabolism and liver clearance of chylomicrons and other triglyceride-rich lipoproteins.[4] By reducing serum apoC-III, olezarsen increases clearance of plasma triglycerides.[4]

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][5] The US Food and Drug Administration considers it to be a first-in-class medication.[6]

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).[7]

History

The US Food and Drug Administration (FDA) granted the application of olezarsen orphan drug designation in February 2024.[8] In August 2024, the European Medicines Agency granted olezarsen an orphan drug designation.[9]

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][5][10]

In July 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Tryngolza, intended for the treatment of adults with familial chylomicronemia syndrome.[4] The applicant for this medicinal product is Ionis Ireland Limited.[4] Olezarsen was authorized for medical use in the European Union in September 2025.[4][11]

Names

Olezarsen is the international nonproprietary name.[12]

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

References

  1. ↑ 1.0 1.1 1.2 1.3 1.4 1.5 Cite error: Invalid <ref> tag; no text was provided for refs named Tryngolza FDA label
  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. ↑ 3.00 3.01 3.02 3.03 3.04 3.05 3.06 3.07 3.08 3.09 3.10 3.11 3.12 3.13 3.14 "Drug Trials Snapshot: Tryngolza". 19 December 2024. https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshot-tryngolza.  Public Domain This article incorporates text from this source, which is in the public domain.
  4. ↑ 4.0 4.1 4.2 4.3 4.4 "Tryngolza EPAR". 25 July 2025. https://www.ema.europa.eu/en/medicines/human/EPAR/tryngolza.  Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  5. ↑ 5.0 5.1 "Novel Drug Approvals for 2024". 1 October 2024. https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024. 
  6. ↑ (PDF) New Drug Therapy Approvals 2024 (Report). January 2025. https://www.fda.gov/media/184967/download. Retrieved 21 January 2025. 
  7. ↑ Stroes, Erik S.G.; Alexander, Veronica J.; Karwatowska-Prokopczuk, Ewa; Hegele, Robert A.; Arca, Marcello; Ballantyne, Christie M.; Soran, Handrean; Prohaska, Thomas A. 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. 
  8. ↑ "Olezarsen Orphan Drug Designations and Approvals". https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=953423. 
  9. ↑ "EU/3/24/2973 - orphan designation for treatment of familial chylomicronaemia syndrome". 21 August 2024. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2973. 
  10. ↑ "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.
  11. ↑ "Tryngolza PI". 18 September 2025. https://ec.europa.eu/health/documents/community-register/html/h1969.htm. 
  12. ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87". WHO Drug Information 36 (1). 2022. 

Further reading



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