Aegerion Pharmaceuticals Wins Approval for Cholesterol Drug

by Brett Chase, Minyanville staff writer
Aegerion Pharmaceuticals AEGR has won US approval to sell a drug that treats a rare genetic condition linked to heart disease in young people.

Aegerion’s cholesterol-lowering capsule drug Juxtapid was approved by the Food and Drug Administration to help fight homozygous familial hypercholesterolemia. It’s the first drug approved for the money-losing biotech company.

The medicine is aimed at a small patient population. Initially, Aegerion estimates about 3,000 patients in the US can be treated with the product, CEO Marc Beer says.

Beer told analysts on a conference call Monday morning that Juxtapid will be priced between $200,000 and $300,000 per patient per year. He noted that he was encouraged that insurers will pay for the drug as it addresses an unmet need for patients.

“These patients have new hope to reduce their cholesterol levels,” Beer says.

The drug will include warnings about potential side effects, including liver damage. Despite the safety issues, experts advisers to the FDA voted 13-2 in October to approve the treatment. (See also: Aegerion, Isis, Sanofi Make Case for Drugs to Treat Severe Cholesterol.)

Government advisers were split on another medicine developed by Isis Pharmaceuticals ISIS and Sanofi SNY during a separate review in October. The medicine, Kynamro, developed by those companies, was recommended by an FDA panel on a 9-6 vote. Split decisions often bode poorly for a drug’s prospects for approval. The FDA is expected to rule on Kynamro next month. A panel that recommends drug approval to European regulators refused to back the drug earlier this month due to safety concerns.

As part of Aegerion’s approval, the company agreed to a so-called risk evaluation and mitigation strategy for patients. The drug will be available through a restricted program.

Shares of Aegerion dropped 5% to $24.51 in morning trading Monday. The stock is still up 57% over the past three months. Shares of Isis fell 1% to $10.15 Monday morning. That stock declined 29% over the past three months. Sanofi’s US shares dropped less than 1% to $47.34. The French drug maker’s ADRs jumped 30% in the past six months. Sanofi gained the stake in Kynamro when it took over US-based Genzyme last year.

Juxtapid, also known as lomitapide, was originally owned by Bristol-Myers Squibb BMY, which was trying to develop a cholesterol drug for a broader audience in the 1990s. After a high rate of gastrointestinal side effects, the drug program was scrapped. Bristol-Myers donated the experimental drug to a University of Pennsylvania doctor, Daniel Rader, in 2003. Three years later, Aegerion licensed the drug from the university.

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