Aegerion Pharmaceuticals, Inc. an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat severe lipid disorders, announced it has received orphan drug designation from the US Food and Drug Administration's (FDA) Office of Orphan Product Development for its lead drug candidate, lomitapide, for the treatment of Familial Chylomicronemia (FC).

Lomitapide is currently being evaluated in an ongoing pivotal phase III clinical trial for the treatment of Homozygous Familial Hypercholesterolemia (HoFH), and the company expects to launch a separate clinical trial to evaluate lomitapide as a treatment for FC in the second half of 2011.

“The FDA's grant of orphan drug designation to lomitapide for the treatment of FC represents an important milestone in the overall development and commercialization plan for the compound, and it underscores the unmet need that exists. With this announcement, we are another step closer towards our goal of treating patients with these rare lipid disorders that currently have inadequate treatment options available,” commented Marc D Beer, chief executive officer of Aegerion.

Familial Chylomicronemia, a rare genetic disorder and severe form of hypertriglyceridemia that results in extremely high levels of triglycerides in the blood, is the second disease for which Aegerion is developing lomitapide and for which it plans to seek regulatory approval if development is successful. Lomitapide has already been granted orphan drug designation for the treatment of FC in the European Union, and for the treatment of HoFH, a rare genetic lipid disorder resulting in an accumulation of Low-Density Lipoprotein (LDL-C) in the blood, in the United States.

Lomitapide is a small molecule micro-somal triglyceride transfer protein inhibitor being developed as an oral, once-a-day treatment for patients with severe lipid disorders. It is being evaluated for its ability to reduce LDL-C levels in patients with HoFH and reduce triglyceride levels in patients with FC. It reduces lipid levels in the blood by preventing the liver and intestines from secreting lipids into the blood stream.

In the United States, under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug intended to treat a rare disease or condition, which is generally a disease that affects fewer than 200,000 individuals in the country. The designation grants US market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA requirements. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee. The designation does not change or shorten the duration of the regulatory review and approval process.