Both lomitapide from Aegerion Pharmaceuticals Inc. (NASDAQ:AEGR) and Kynamro mipomersen from the Genzyme Corp. unit of Sanofi (Euronext:SAN; NYSE:SNY) and Isis Pharmaceuticals Inc. (NASDAQ:ISIS) significantly reduced LDL-C levels in their respective Phase III trials in homozygous familial cholesterolemia (hoFH). But big differences in the trial designs and a high degree of interpatient variability on efficacy and side effects for both compounds make it difficult to compare the data.

Aegerion's UP1002 was an open-label, single-arm, dose-escalation Phase III study in 29 patients. Subjects were on a low-fat diet and maximally tolerated lipid-lowering therapy, which could include LDL-C apheresis. Patients started on 5 mg lomitapide daily for two weeks then increased to daily doses of 10, 20, 40 and 60 mg at four