Olezarsen
Medication
From Wikipedia, the free encyclopedia
Olezarsen, sold under the brand name Tryngolza, is a medication used in the treatment of familial chylomicronemia syndrome.[1][4] It is given by injection under the skin.[1] Olezarsen is an apolipoprotein C-III-directed antisense oligonucleotide.[1][5] 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.[2] By reducing serum apoC-III, olezarsen increases clearance of plasma triglycerides.[2]
| |
| Clinical data | |
|---|---|
| Trade names | Tryngolza |
| Other names | IONIS-APOCIII-LRX |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a625020 |
| License data | |
| Routes of administration | Subcutaneous |
| Drug class | Antisense oligonucleotide |
| ATC code |
|
| Legal status | |
| Legal status | |
| Identifiers | |
| |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C296H439N71O154P20S19 |
| Molar mass | 8684.73 g·mol−1 |
| |
| |
The most common side effects include injection site reactions, low platelet counts, and joint pain.[5]
Olezarsen was approved for medical use in the United States in December 2024.[5][6] The US Food and Drug Administration considers it to be a first-in-class medication.[7]
Medical uses
Olezarsen is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome.[1]
Adverse effects
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).[8]
History
The US Food and Drug Administration (FDA) granted the application of olezarsen orphan drug designation in February 2024.[9] In August 2024, the European Medicines Agency granted olezarsen an orphan drug designation.[10]
The FDA approved olezarsen based on evidence from a clinical trial (trial 1; NCT04568434) of 66 participants with familial chylomicronemia syndrome.[5] 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.[5] Of the 66 participants, 19 participants were from trial sites in the United States.[5] The benefits and side effects of olezarsen for participants with familial chylomicronemia syndrome were evaluated in the same single clinical trial.[5] Additional trials in participants with hypertriglyceridemia were used to support the safety assessment.[5] 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.[5] 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).[5] Participants were randomly assigned to receive olezarsen or placebo every four weeks for one year.[5] Neither the participants nor the health care providers knew which treatment was being given.[5]
Society and culture
Legal status
Olezarsen was approved for medical use in the United States in December 2024.[5][6][11]
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.[2] The applicant for this medicinal product is Ionis Ireland Limited.[2] Olezarsen was authorized for medical use in the European Union in September 2025.[2][3]
Names
Olezarsen is the international nonproprietary name.[12]
Olezarsen is sold under the brand name Tryngolza.[1]
