Aegerion Pharmaceuticals Inc. won the backing of U.S. regulatory advisers Wednesday for its drug to treat the most life-threatening forms of high cholesterol.
An advisory panel to the Food and Drug Administration voted 13-2 today that Cambridge, Massachusetts-based Aegerion submitted sufficient data on the safety and efficacy of lomitapide for use in patients with a rare genetic disease. The FDA, which isn’t obligated to follow the recommendation, is scheduled to decide on approval by the end of December.
The once-daily pill aims to treat homozygous familial hypercholesterolemia that causes abnormalities in liver cells responsible for clearing LDL, or low density lipoprotein, particles from the blood. The disease can lead to heart attack or death at an early age, according to the National Institutes of Health. The panel said doctors should monitor patients who use the drug for liver abnormalities.
“I was convinced that the efficacy is very impressive,” Edward Gregg, a panel member and chief of the epidemiology and statistics branch in the Division of Diabetes Translation at the Centers for Disease Control and Prevention, said during the meeting. “The liver disease is concerning but I think it’s trumped by this very grave condition and the need for options.”
Aegerion should implement a risk plan that mitigates the potential for liver damage, FDA staff said in an Oct. 15 report discussed at Wednesday’s meeting in Silver Spring, Maryland.
The risk plan “would support appropriate use of lomitapide, allowing it to be approved for use in the targeted patient population, a patient population with life-threatening illness and limited therapeutic options,” FDA staff wrote.
Of the 29 patients in a clinical trial, 17 percent experienced the presence of an increased enzyme in their blood that indicates liver damage. The panel suggested Aegerion’s risk management plan include educating prescribers about liver risks and the need to monitor patients with blood tests, said Abraham Thomas, acting chairman of the panel and division head of endocrinology at the Henry Ford Hospital in Detroit.
Aegerion also has proposed a registry to follow about 300 patients treated with lomitapide for side effects for five years.
Lomitapide may generate at least $300 million in annual sales, said William Tanner, an analyst at Lazard Capital Markets LLC in New York, who rates Aegerion shares neutral. The estimate is based on a price of $300,000 a year per patient that is similar to costs of other drugs for diseases with few treatment options, he said. Kimberly Lee, a Think Equity Partners analyst in San Francisco, said peak sales may reach $450 million.
Aegerion is in discussions with the FDA to study lomitapide in pediatric patients and plans to begin a trial as soon as possible after the drug is approved in adult patients, the company said.
Trading in Aegerion shares was halted Wednesday, after the stock gained 9.7% to end at $19.15 on Tuesday. The shares rose 14 percent this year through Tuesday. The advisory panel will consider a similar drug from Isis Pharmaceuticals Inc. and Sanofi Thursday.
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