Actions and Spectrum:
mipomersen is a medication used to lower LDL cholesterol levels in certain patients with homozygous familial hypercholesterolemia, a rare genetic disorder that causes extremely high LDL cholesterol levels.
The mechanism of action of mipomersen involves inhibiting the production of apolipoprotein B (apoB), a protein necessary for the formation of LDL cholesterol. By reducing apoB production, mipomersen lowers LDL cholesterol levels in the blood.
mipomersen is administered weekly and works by binding to messenger RNA (mRNA) molecules in the liver, which are responsible for producing apoB. By binding to these mRNA molecules, mipomersen prevents the formation of apoB, ultimately leading to a decrease in LDL cholesterol levels.
mipomersen has a narrow spectrum of activity, as it is only effective in patients with homozygous familial hypercholesterolemia (HoFH) who have extremely high LDL cholesterol levels. It is not used to treat other high cholesterol types or prevent heart disease.
