Overview

Lowering Cardiovascular Risk in Rare Lipid Disorders

Lipids are naturally occurring molecules that are transported in the blood. Excess levels of lipids such as low density lipoprotein (LDL) cholesterol and triglycerides can drastically increase the risk of potentially life threatening cardiovascular events, such as stroke or heart attack or in the case of excess triglycerides, pancreatitis. In several rare genetic lipid disorders, patients with extremely high lipid levels are at very high risk of these events at an early age. There is strong demand for a safe drug that can reduce LDL cholesterol and/or triglycerides in this patient population.

Aggressive Reduction of Lipids recommended by ACC and AHA

A direct relationship between reduced LDL-C and/or triglyceride levels and reduced risk of major cardiovascular events has been consistently demonstrated for more than a decade based on over 14 trials involving more than 90,000 patients, which showed about a 1% reduction in risk for every 2 mg/dL drop in LDL-C. As a result, physicians are highly focused on lowering levels of LDL-C and TG using combinations of diet and pharmacological therapy. In the US, for example, the American College of Cardiology and the American Heart Association have placed increased attention on aggressive LDL-C management and the National Cholesterol Education Program Guidelines recommend levels of less than 100 mg/dL of LDL-C and less than 150 mg/dL of TG. An optional target of <70 mg/dL (1.8mmol/L) for LDL-C has been recommended for patients deemed to be at "very high" risk requiring more aggressive LDL lowering.

Treating Fatal, Genetic Orphan Diseases

Yet there is a sub-section of patients that neither dietary modification nor maximum doses of currently approved lipid lowering drug therapies can address. Aegerion is focused on providing life-altering therapies for these debilitating and fatal, genetic orphan diseases affecting tens of thousands of patients worldwide.