Because it is intended to treat a disease that affects fewer than 200,000 people in the United States at any one time, lomitapide was designated an orphan drug for the treatment of HoFH, and in June 2011, the FDA agreed that the data from the pivotal study would be adequate to support the submission of the approval application. Dr. James Smith, a medical officer in the FDA’s division of metabolism and endocrinology products, pointed out that lomitapide is one of the first lipid-lowering drugs that is being considered for approval for an orphan disease, "without the benefit of having a large phase III safety database derived from a broader population."
If approved, Aegerion plans to study the drug in a pediatric population. The company has submitted an application for approval of lomitapide for the adult indication in the European Union.
The FDA is expected to make a decision on approval by Dec. 29, according to the company. The agency usually follows the recommendations of its advisory panels, which are not binding. Panelists have been cleared of potential financial conflicts of interest related to the topic of the meeting. Occasionally, a panelist may be given a waiver, but not at this meeting.