WASHINGTON (AP) -- Shares of Aegerion Pharmaceuticals Inc. rose Monday after regulators said the company's drug for a rare cholesterol disorder appears effective and could be used safely if physicians follow the company's guidance.
THE SPARK: The Food and Drug Administration posted a favorable review of Aegerion's experimental drug lomitapide, ahead of a public meeting Wednesday where a panel of experts will vote on the drug. The FDA is not required to follow the drug panel's advice, though it often does.
THE BIG PICTURE: Aegerion has been studying lomitapide's effectiveness and safety as a treatment for homozygous familial hypercholesterolemia, a rare genetic condition that causes severely elevated levels "bad" LDL cholesterol. The disease occurs in about 1 in every 1 million people in the U.S., according to the FDA. Patients usually develop symptoms during childhood, which can become fatal if left untreated. Lomitapide is designed to prevent the liver and intestines from secreting lipids into the bloodstream.
The FDA said in its review that 69 percent of patients in a company trial lowered their bad cholesterol by about 15 percent after 26 weeks taking the drug.
Side effects of the drug include potential for liver toxicity. The company said it plans to minimize the risk of the drug by educating prescribers about the need to monitor patients for side effects during treatment. The company also recommends requiring special certification for all doctors and pharmacies that dispense the drug.
The FDA said the plan outlined by the company "would support appropriate use of lomitapide, allowing it to be approved for use in the targeted patient population, a group with life threatening illness and limited therapeutic options.
SHARE ACTION: Shares of Cambridge, Mass.-based Aegerion rose $1.94, or 12.2 percent, to $17.90 in afternoon trading after rising as high as $18.15 earlier in the day, its highest level since June 2011.
- Pharmaceuticals & Drug Trials
- Food and Drug Administration