Aegerion’s Lomitapide Gets Standard Review For Rare Genetic Cholesterol Disease
This article was originally published in Pharmaceutical Approvals Monthly
Aegerion is preparing for an advisory committee meeting after submitting its LDL-C lowering oral drug lomitapide to FDA for patients with homozygous familial hypercholesterolemia. The NDA was submitted ahead of that for Isis/Genzyme’s injectable competitor, mipomersen.
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FDA will be using an upcoming advisory panel review of Aegerion Pharmaceuticals Inc.’s lomitapide to see if there is still support for traditional lipid endpoints in clinical trials of cholesterol drugs.
Aegerion submitted the lomitapide NDA Feb. 29 with 54-week data from a single-arm, open-label trial, hoping for priority review based on the lack of effective treatments for homozygous familial hypercholesterolemia. Meanwhile, Isis/Sanofi submitted their competing candidate mipomersen March 29 with two-year extension-study data.
Mipomersen, Genzyme/Isis Pharmaceuticals' second-generation antisense drug for severe high cholesterol, is four for four in terms of collecting positive outcomes in Phase III trials. But in addition to lowered cholesterol, patients in all of those studies had elevated liver enzymes, leaving open the question whether regulators will consider the side effect a manageable trade off for filling an unmet medical need