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Information about Mipomersen

Two new agents became available in 2013 for therapy of homozygous familial hypercholesterolemia, a rare but severe inborn error of metabolism marked by high levels of low density lipoprotein cholesterol and childhood onset of complications of atherosclerosis.

Mechanism of action of Mipomersen

  • Mipomersen (Kynamro) is an antisense oligonucleotide that binds to the mRNA that encodes apolipoprotein B.
  • Lomitapide (Juxtapid) is an inhibitor of microsomal triglyceride transport protein which blocks production of apolipoprotein B containing lipoproteins in the liver and intestine.

Liver safety of Mipomersen

Both agents can lower serum cholesterol levels by 25% to 30% in patients with familial hypercholesterolemia; however, both are hepatotoxic and their use is restricted. Both mipomersen and lomitapide can cause serum aminotransferase elevations and hepatic steatosis and thus may induce or exacerbate nonalcoholic fatty liver.

Lipid lowering medications

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