Chemistry: Generation 2+ LICA
IONIS-APOCIII-LRx, formerly known as AKCEA-APOCIII-LRx, is a ligand-conjugated (LICA) investigational antisense medicine designed to inhibit the production of apoC-III, for patients who are at risk of disease due to elevated triglyceride levels. ApoC-III is a protein produced in the liver that regulates triglyceride metabolism in the blood. People with severely elevated triglycerides, such as people with familial chylomicronemia syndrome (FCS), are at high risk for acute pancreatitis and other serious conditions. ApoC-III is also the target of WAYLIVRA, the first approved medicine for patients with FCS.
About familial chylomicronemia syndrome (FCS)
FCS is a rare, genetic disease characterized by extremely elevated triglyceride levels that is estimated to affect 3,000 to 5,000 people worldwide. People with FCS are at high risk of unpredictable and potentially fatal acute pancreatitis. In addition to pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and pancreatogenic (type 3c) diabetes. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognition that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. Additional information on FCS is available at www.fcsfocus.com, through the LPLD Alliance at http://www.lpldalliance.org and through The FCS Foundation at http://www.livingwithfcs.org. For a full list of organizations supporting the FCS community worldwide, please click here.