Mipomersen

Mipomersen (ISIS 301012 free base) is an antisense oligonucleotide inhibitor of apolipoprotein B-100 (apoB-100) that causes selective degradation of apoB-100 mRNA, thereby inhibiting protein translation. This leads to reductions in low-density lipoprotein cholesterol (LDL-C) and other apoB-containing lipoprotein levels. Mipomersen is used as an adjunct treatment for homozygous familial hypercholesterolemia (FH) .