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Aegerion Pharmaceuticals, Inc. Beats Genzyme Corporation (GENZ) to FDA in Rare-disease Race

3/6/2012 8:04:11 AM

Business Journal by Julie M. Donnelly, Reporter

Aegerion Pharmaceuticals, Inc. (Nasdaq: AEGR) has submitted its potential drug for a rare form of extremely high cholesterol to the U.S. Food and Drug Administration and to European regulators for approval. The company expects that the FDA will review the drug within six months, rather than the customary 10 months, because there are currently no drugs on the market to treat patients with the genetic disease, called Homozygous Familial Hypercholesterolemia (HoFH). The disease, which usually kills patients by the age of 30, causes strokes and heart attacks.

The drug candidate, called lomitapide, is a once-daily oral pill that would be used in conjunction with a low fat diet and other lipid-lowering therapies. Patients diagnosed with HoFH typically have as much as three to six times the normal amount of bad cholesterol even while taking over cholesterol-lowering drugs.

With Monday's announcement, Aegerion has beaten its rival, Cambridge-based Genzyme Corp., in submitting its drug to the FDA first. Genzyme, the rare disease and multiple sclerosis unit of French drug maker Sanofi (NYSE: SNY), slightly delayed its planned 2011 submission of its experimental HoFH therapy until the first quarter of 2012, in order to prepare additional technical documents. Genzyme’s drug is an injectable, while Aegerion’s is an oral pill. Genzyme’s drug candidate would be marketed as Kynamro and was licensed from its partner Isis Pharmaceuticals (Nasdaq: ISIS).

If approved, lomitapide would be the first approved therapy for Cambridge, Mass.-based Aegerion. Founded in 2005, the company went public in 2010. Aegerion is focused on rare, genetic lipid disorders, like HoFH, and other orphan diseases.

“The submission ... represents a significant corporate accomplishment,” CEO Marc D. Beer, said in a statement. “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.”

“I really hope both products are approved,” Beer said in an interview with the BBJ in December. “We’re not in a competitive battle, we’re in a battle against the disease, and what keeps me up at night is that every day these products are not approved is a bad day for patients.”

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