New agents in the treatment of familial hypercholesterolemia: Lomitapide vs. Mipomersen

Familial hypercholesterolemia (FH) is caused by genetic deficiency of LDL receptors leading to extremely high cholesterol levels and atherosclerosis at early ages.
For the prevention of early atherosclerotic cardiovascular events, effective reduction of LDL-cholesterol is necessary from the early ages. However, particularly in homozygous
patients, it’s almost impossible to achieve target LDL-cholesterol levels with antilipid agents including statin agents, due to the severe LDL receptor dysfunction. LDL apheresis
is an effective treatment modality in severe FH patients. However, the invasive, chronic time consuming nature of this treatment decreases the compliance of these patients.
Moreover, atherosclerosis progress in 25% of the patients undergoing regular and effective apheresis even though since early ages. Clinical data also indicate that there is
still an unmet medical need for new effective treatments for FH patients. This review will address new therapeutic strategies targeting Apolipoprotein (Apo) B including MTTP
inhibitor Lomitapide and oligonucleotide Mipomersen. As both agents are targeted against ApoB, they are expected to be effective even in receptor negative homozygous FH
patients.