Aegerion application for lead rare disease drug lands at FDA

Cambridge, MA-based Aegerion Pharmaceuticals has cleared an important milestone. The rare disease drug developer submitted an NDA for its lead drug, dubbed lomitapide, which is designed to reduce lethal levels of cholesterol in patients with Homozygous Familial Hypercholesterolemia.

The Boston Business Journal notes that Aegerion's application beats out the arrival of Genzyme's NDA for a rival therapy. And the biotech--which launched in 2005 and went public ($AEGR) in 2010--is hoping that the once-daily oral med can get through the FDA's review process in 6 months rather than the standard 10-month review.

Patients who are diagnosed with HoFH often have as much as three to 6 times the normal amount of LDL-C while on a variety of lipid-lowering drug treatments, putting them at risk for a major cardiovascular event, the developer noted. Most patients die around the age of 30.

"The submission of our NDA and MAA filings represents a significant corporate accomplishment," said CEO Marc D. Beer. "In 2011, we assembled a team of experts with deep experience in orphan and genetic diseases, dedicated to working with the worldwide HoFH community. This is an important step towards making lomitapide available to patients suffering from HoFH who currently have inadequate treatment options."

- here's the press release
- get the story from the Boston Business Journal