Olezarsen, formerly known as IONIS-APOCIII-LRx and AKCEA-APOCIII-LRx, is an RNA-targeted investigational LIgand-Conjugated Antisense (LICA) medicine being evaluated for people at risk of disease due to elevated triglyceride levels, including those with familial chylomicronemia syndrome (FCS). Olezarsen is designed to inhibit the body’s production of apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood.

About Familial Chylomicronemia Syndrome (FCS)

FCS is a rare, genetic form of severe hypertriglyceridemia (sHTG) and is characterized by extremely elevated triglyceride levels and corresponding build-up of large triglyceride-containing particles in the blood called chylomicrons, which can result in severe health complications. It is caused by impaired function of the enzyme lipoprotein lipase (LPL). Because of limited LPL production or function, people with FCS cannot effectively break down chylomicrons, lipoprotein particles that are 90% triglycerides. FCS is estimated to impact one to 13 people per million in the U.S. People living with FCS are at high risk of acute pancreatitis (AP) in addition to other chronic health issues such as severe, recurrent abdominal pain. People living with FCS experience debilitating psychosocial symptoms, and are sometimes unable to work, adding to the burden of disease.

Safety and efficacy have not been evaluated by any regulatory authorities for the indication described.

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