Lomitapide (Juxtapid) Shows Promise in Treatment of Hypercholesterolemia
Positive phase III data reported at heart meeting (November 19)
Positive results have been reported from an open-label phase III extension study of lomitapide (Juxtapid, Aegerion Pharmaceuticals) in patients with homozygous familial hypercholesterolemia (HoFH). The new data were presented Nov. 18 at the American Heart Association's Scientific Sessions taking place in Dallas, Texas.
Nineteen of 23 patients who completed the 78-week pivotal study entered the extension study and continued lomitapide at their individualized maintenance dose. Seventeen patients (89%) completed 126 weeks of treatment. The extension study’s primary efficacy endpoint was the mean percent change in low-density lipoprotein cholesterol (LDL-C) from baseline to week 126. The baseline was determined at the beginning of the pivotal study (week 0) after a 6-week run-in period, during which patients were stabilized on their other lipid-lowering therapies.
Patients remained on a stable regimen of lipid-lowering therapies during the 26-week efficacy phase of the pivotal study. Adjustments to concomitant lipid-lowering treatments were allowed after 26 weeks during the safety phase of the pivotal study and in the extension study.
Mean LDL-C levels were reduced by 45.5% from baseline at week 126 (356 +/– 127 mg/dL vs. 189 +/– 120 mg/dL; P < 0.001). Similar mean percent reductions were observed for apolipoprotein B (Apo B), non–high-density lipoprotein cholesterol (non–HDL-C), and total cholesterol (TC). On at least one time point during the study, an LDL-C of ≤ 100 or ≤ 70 mg/dL was achieved by 13 (68%) and nine (47%) patients, respectively.
Lomitapide is a microsomal triglyceride transfer protein (MTP) inhibitor that works independently of the LDL receptor functionality. It is indicated in the U.S. as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce LDL-C, TC, apo B, and non–HDL-C in patients with HoFH.
Source: Aegerion Pharmaceuticals; November 19, 2013.