UPDATED: Aegerion wins lopsided FDA panel vote backing rare cholesterol disease drug

A group of FDA advisers took a hard look at Aegerion's ($AEGR) new drug for homozygous familial hypercholesterolemia on Wednesday and concluded that the clear need for a new therapy outweighed any safety concerns they might have concerning liver disease. In a lopsided vote, 13 of the outside experts voted to recommend an approval of the drug, with only two voting against.

The vote fell right in line with the FDA's internal review for lomitapide, which concluded that the right risk evaluation and mitigation strategy "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." 

"I was convinced that the efficacy is very impressive," said panel member Dr. Edward Gregg, according to a report from Bloomberg. "The liver disease is concerning but I think it's trumped by this very grave condition and the need for options."

Aegerion shares shot up about 15% on the news.

Homozygous familial hypercholesterolemia is an extremely rare genetic condition characterized by dangerously high levels of LDL, which can trigger a lethal set of cardiovascular conditions. But a significant 17% of patients in the small study demonstrated a spike in liver enzymes, a classic red flag for drug developers. The likely REMS program that will be required to safeguard patients will include an education program for physicians and active monitoring of patients.

The vote marks another big step toward a potential launch of the drug, a major turning point for the biotech. CEO Marc Beer says an approval would put the company on a path to profitability in 2014, completing a turnaround he started two years ago. A rival therapy for the rare disorder from Sanofi's ($SNY) Genzyme and Isis Pharmaceuticals ($ISIS)--mipomersen--is up for review tomorrow.

"We're ready to launch based on an approval," Beer tells FierceBiotech. And even if mipomersen gains a panel endorsement today, Beer says that it would be good for these patients to have a pair of therapies to choose between.

Cambridge, MA-based Aegerion made the decision early on to keep control of this program, spearheading an app in Europe which could set the stage for a 2013 launch while getting ready to do its own development program in Japan uniquely tailored for that market. Aegerion is also prepping a pediatric study to broaden the approved patient population.

With the drug on course to a probable approval, Aegerion has been growing a small commercial operation, bringing its total staff count to about 70 now. That's a big change over just a couple of years. And the biotech will keep its eyes focused on lomitapide, with no other programs competing for attention.

- get the press release
- here's the report from Bloomberg

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