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Rader - Figure 22 - New therapies for homozygous FH that target VLDL production

This is an exciting time in the field of lipidology, because after a couple of decades of standard lipid-lowering agents, there are now two new mechanisms of action that have been developed and that are currently being evaluated for approval (Figure). Mipomersen is an antisense oligonucleotide (ASO) directed toward the ApoB mRNA, and lomitapide is a small molecule directed toward inhibiting the microsomal transfer protein (MTP). Both of these drugs reduce very-low-density lipoprotein (VLDL) secretion from the liver, resulting in reduced LDL-C levels in the blood, rendering them particularly applicable in people who lack functional LDL receptors, homozygous FH patients, for whom these drugs are currently under review.

Rader J Clin Lipidol. 2012;