When Aegerion Pharmaceuticals Inc.’s NDA for homozygous familial hypercholesterolemia therapy lomitapide is taken up by FDA’s Endocrinologic and Metabolic Drugs Advisory committee Oct. 17 the big question will be whether the company has enough data to convince regulators of the drug’s efficacy and safety, particularly in light of a liver toxicity signal that appears manageable but likely to set off warnings.
The lomitapide application was supported by one single-arm, open-label, dose-escalating Phase III trial in 29 patients averaging 31 years of...