In briefing documents ahead of an advisory committee meeting on October 17, an FDA panel stated, "In considering a risk management program for lomitapide, FDA must keep in mind that the HoFH patient population currently has limited therapeutic options. On the other hand, the risk-benefit profile of lomitapide in the larger patient population with hypercholesterolemia has not been established, and there is reason for concern should this larger patient population be exposed to lomitapide. The REMS proposed above 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, while protecting the larger hypercholesterolemic patient population. We believe that this proposed REMS is needed to ensure that the benefits of lomitapide outweigh the potential risk of serious liver injury." Aegerion's (AEGR) Lomitapide is being evaluated for its ability to reduce low density lipoprotein or bad cholesterol levels in patients with Homozygous Familial Hypercholesterolemia, or HoFH. Shares of Aegerion are responding favorably to the FDA panel's comments, up 13%, or $2.04, to $18.00 in pre-market trading.
- Government Agencies