Aegerion Rare-Disease Drug Wins U.S. Regulatory Approval
Aegerion Pharmaceuticals Inc. (AEGR)’s drug to treat the most life-threatening forms of high cholesterol won U.S. approval, and will carry a label warning about risk of liver toxicity.
The Food and Drug Administration cleared the treatment known as lomitapide for use in patients with a rare genetic disease that causes extreme elevation of blood cholesterol levels, the company said today in a statement. The drug is Cambridge, Massachusetts-based Aegerion’s first on the market.
The once-daily pill called Juxtapid is designed to treat homozygous familial hypercholesterolemia, a condition that causes abnormalities in liver cells responsible for clearing LDL, so-called bad cholesterol, from the blood. The disease can lead to heart attack or death at an early age, according to the National Institutes of Health.
“We are excited that Juxtapid will become a new treatment option for patients with HoFH,” Marc Beer, chief executive officer at Aegerion, said in the statement. “The approval of our first product also marks an important corporate milestone for Aegerion and reflects our commitment to help patients in need.”
Juxtapid will carry a boxed warning, the FDA’s strictest caution, alerting patients to the risk of liver toxicity. Of the 29 patients in a clinical trial, 34 percent had at least one elevation in an enzyme that indicates liver damage, according to the boxed warning.
Aegerion fell 1.8 percent to $25.25 at today's 1 p.m. close ahead of the Christmas holiday. The company has gained 52 percent this year.
Aegerion will make the drug available to physicians and pharmacies the company certifies as educated about the risk of liver toxicity and the need to monitor patients who use the product.
The market for the treatment could total as many as 3,000 patients in the U.S., Beer said today on a conference call. The drug will cost between $200,000 and $300,000 per patient per year, he said. Aegerion plans to offer more details on pricing at the JPMorgan Healthcare Conference beginning Jan. 7 in San Francisco.
When added to existing lipid-lowering therapy, Juxtapid helped reduce LDL cholesterol by about 50 percent in a trial of 23 patients after 26 weeks, according to the statement.
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