Lomitapide has been evaluated in fourteen Phase I and eight Phase II clinical trials, as well as a pivotal Phase III clinical trial in HoFH completed in 2011. An extension study to assess long-term safety is ongoing. Over 900 patients have been treated with lomitapide as part of these clinical trials.

The most frequent adverse events in the Phase III clinical trial were gastrointestinal, and were generally mild to moderate. These events typically decreased after the patients were established on the maximally tolerated dose. Elevations in liver enzymes and hepatic fat were also observed in the Phase III trial. Four patients experienced elevations in liver enzymes of between five times to eleven times the upper limit of normal. Hepatic fat increased from a baseline of 1% to 8.3% at week 26, and then stabilized through week 78.

Aegerion submitted a NDA to the Food and Drug Administration (FDA), and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), requesting approval to market lomitapide as an adjunct to a low-fat diet and other lipid-lowering therapies, with or without apheresis, to reduce LDL-C, total cholesterol, apolipoprotein B, and triglycerides in adults with HoFH.

About Homozygous Familial Hypercholesterolemia (HoFH)

HoFH is a rare genetic lipid disorder that, if left untreated, results in extremely high cholesterol levels, typically between 400 mg/dL and 1,000 mg/dL. Those affected are at severely high risk of experiencing premature cardiovascular events, such as heart attack or stroke, often experiencing their first cardiovascular event in their twenties. Despite current treatments, many patients with HoFH do not survive beyond their mid-30's.

The disease is usually caused by defects in the low-density lipoprotein (LDL) receptor genes, resulting in impaired or total loss of function. The LDL receptor is a protein on the surface of cells that is responsible for binding to and removing cholesterol from the blood. A loss of LDL receptor function results in elevated accumulation of cholesterol in the blood.

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