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Volanesorsen is a second-generation 2'-O-methoxyethyl (2'-MOE) chimeric antisense oligonucleotide designed to reduce triglyceride levels by targeting the messenger RNA (mRNA) for apolipoprotein C-III (apoC-III). By binding to the apoC-III mRNA, volanesorsen induces its degradation via RNase H1-mediated cleavage, thereby preventing translation of the apoC-III protein. ApoC-III normally inhibits triglyceride metabolism and hepatic clearance of chylomicrons; thus, inhibition of its production enables enhanced breakdown and clearance of triglycerides through both lipoprotein lipase-dependent and -independent pathways. Volanesorsen is primarily indicated as an adjunct to diet in adults with genetically confirmed familial chylomicronemia syndrome (FCS) who are at high risk for pancreatitis and have not responded adequately to other triglyceride-lowering therapies. The drug was discovered and developed by Ionis Pharmaceuticals[1][2][6].
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