Aegerion Pharmaceuticals Inc. (AEGR) rose after a U.S. regulatory staff report said a plan to manage the risks of the company’s drug supports its approval in adults with the most life-threatening forms of severely high cholesterol.
Aegerion gained 9.4 percent to $17.46 at the close of New York trading following the Food and Drug Administration’s staff report today. An advisory panel will meet Oct. 17 on the medicine lomitapide, Cambridge, Massachusetts-based Aegerion’s lead product candidate, with the FDA scheduled to make a final decision by the end of December.
The once-daily pill aims to treat a genetic condition known as 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 advisory panel will consider a similar drug from Isis Pharmaceuticals Inc. (ISIS) and Sanofi (SAN) on Oct. 18.
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.
The FDA can require risk evaluation and mitigation strategies, or REMS, that go beyond drug labeling to ensure the benefits of a medication outweigh the risks.
“We do not believe that larger studies will be necessary given the REMS,” Kimberly Lee, an analyst at Think Equity Partners in San Francisco said in a telephone interview. “The FDA recommended the REMS program to better assess the drug safety.”
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 FDA suggested Aegerion’s risk management plan include educating prescribers about liver risks and the need to monitor patients with blood tests.
William Tanner, an analyst with Lazard Capital Markets LLC in New York, who rates Aegerion shares as neutral, estimates lomitapide may generate $300 million or more in annual sales. The estimate is based on a price of about $300,000 a year per patient that is similar to the costs of other drugs for diseases with few treatment options, he said. Lee said peak sales may reach $450 million.
Isis, based in Carlsbad, California, gained 4 percent to $13.15.
People with familial hypercholesterolemia don’t respond to standard cholesterol-lowering drugs such as Pfizer Inc. (PFE)’s Lipitor.
Bristol-Myers Squibb Co. originally began development of lomitapide and abandoned the compound after patients dropped out of a trial at a high rate because gastrointestinal side effects made it tough to tolerate the drug, according to Aegerion’s website.
Three patients out of 29 enrolled in a different clinical trial conducted by Aegerion left because of gastrointestinal side effects, the company said in a statement in May 2011 when the results of the study were released.
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