December 26th, 2012

FDA Approves Lomitapide for Homozygous Familial Hypercholesterolemia

Aegerion Pharmaceuticals said today that the FDA had approved lomitapide (Juxtapid) to help further lower cholesterol in patients with homozygous familial hypercholesterolemia. The approval comes with a box warning about the risk of hepatotoxicity and a Risk Evaluation and Mitigation Strategy (REMS) Program which will require certification of health care providers and pharmacies before the drug can be prescribed and dispensed. The drug is expected to cost between $200,000-$300,000 per year.

The novel drug, which is a microsomal triglyceride transfer protein inhibitor, is indicated, according to the label, “as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).” The label notes that the safety and effectiveness of the drug has not been established in hypercholesterolemia patients who do not have HoFH and that the effect of the drug on cardiovascular morbidity and mortality has not been determined.

See Matt Herper’s post on Forbes: Aegerion Cholesterol Drug Approved. Will It Treat 300 Patients, Or 3,000?

One Response to “FDA Approves Lomitapide for Homozygous Familial Hypercholesterolemia”

  1. Hard clinical end point,such as, MI,ACS,stroke,cardiovascular mortality & all-cause mortality were not tested.Until those data are available it’s clinical utility should be interpreted with caution.