Aegerion Lomitapide drug found to treat hypercholesterolemia

Aegerion Pharmaceuticals has reported positive Phase III clinical study results of its Lomitapide drug, used to treat patient with homozygous familial hypercholesterolemia (HoFH).

Lomitapide is a small molecule microsomal triglyceride transfer protein inhibitor (MTP-I), in clinical development to examine its ability to reduce low density lipoprotein (LDL-C) or bad cholesterol levels in patients with HoFH.

The 78-week single-arm open label pivotal Phase III study involving 29 patients with a mean LDL-C of 337 mg/dL (352 mg/dL for completers) on a variety of background lipid-lowering therapies.

The Phase III study is designed to assess the efficacy and long-term safety of lomitapide for the treatment of patients with HoFH, and found the study results were consistent with data previously reported at the 26- and 56-week time points.

The trial demonstrated long-term safety and efficacy results and found that Lomitapide showed reduction in LDL-C cholesterol from baseline was maintained at 78-weeks.

Aegerion CEO Marc Beer said they intend to submit the applications containing the 56-week data to the FDA and EMA this quarter and continue to build the commercial organization in preparation for their anticipated launch.

Lomitapide has been granted an orphan drug designation by the US Food and Drug Administration for treating this condition.