Mipomersen.

Genzyme Corporation and Isis Pharmaceuticals have a worldwide licensing and collaboration agreement for the development of subcutaneous mipomersen (ISIS 147764; ISIS 301012; ISIS301012; mipomersen-sodium). Mipomersen, an oligonucleotide antisense inhibitor directed against apolipoprotein B-100 (apoB-100) mRNA, is in phase III clinical evaluation for the treatment of familial hypercholesterolemia, a form of type IIa hyperlipoproteinemia, and hypercholesterolemia in patients with severely high cholesterol levels or at high risk for coronary heart disease in the US, European Union, Brazil, Canada, South Africa, and South East Asia. This review discusses the development history and scientific profile of this new compound.