Olezarsen is an agent acetylgalactoseamine conjugated, anti-sense oligonucleotide targeting APOC3 and mRNA for a genetically validated target for triglyceride lowering.
Olezarsen (brand name Tryngolza) is an FDA-approved, once-monthly injectable antisense oligonucleotide therapy designed to treat Familial Chylomicronemia Syndrome (FCS) in adults by reducing apolipoprotein C-III (apoC-III) production in the liver, thereby lowering triglyceride levels.
Olezarsen is an N-acetlgalactosamine-conjugated antisense oligonucleotide that targets the messenger RNA of apolipoprotein C-III, which inhibits triglyceride clearance.
Its targets are hepatocytes the site of APOC3 production.
Olezarsen binds to APOC3 Messenger, RNA, resulting in degradation and reduction in the production of a POC3-it effectively reduces triglyceride levels and the risk of hike per triglyceridemia induced acute pancreatitis.
In patients with predominantly moderate hypertriglyceridemia at elevated, cardiovascular risk, Olezarsen significantly reduced levels of triglycerides, apolipoprotein B, and non-HDL cholesterol, with no major concerns for safety.
Among patients with moderate hypertriglyceridemia and elevated cardiovascular risk, treatment olezarsen resulted in significantly greater reduction in triglyceride levels at six months than placebo.
It is used alongside diet to manage this high-risk condition.
Olezarsen is indicated for adults with FCS, a rare genetic condition preventing the proper breakdown of fats, which can lead to life-threatening acute pancreatitis.
It is an RNA-targeted therapy that inhibits the production of apoC-III, a protein that regulates triglyceride metabolism.
Side Effects: The most common adverse reactions (incidence >5%) include injection site reactions, decreased platelet count and joint pain.
Administration: It is administered as a subcutaneous injection, typically once per month.
Upon its U.S. launch in 2025, the annual list price for Tryngolza (olezarsen) was reported to be $595,000.
Studies have shown that olezarsen can significantly reduce triglyceride levels.
It also demonstrated a substantial reduction in the risk of acute pancreatitis, with data suggesting it can prevent severe, painful events in high-risk patients.
Monitoring: The drug requires monitoring for potential platelet decreases.