Rare Disease Report

Juxtapid Safe and Effective After 126 Weeks. Data Presented at AHA Meeting

NOVEMBER 18, 2013

This afternoon, Aegerion Pharmaceuticals posted results from its open label phase 3 extension study showing patients with homozygous familial hypercholesterolemia (HoFH) continue to do well taking the orphan drug JuxtapidTM (lomitapide) for 126 weeks. The results were presented at the American Heart Association's Scientific Sessions in Dallas.

In the extension, 19 of the 23 patients who completed the 78 week pivotal study (we wrote about here) entered the extension study and continued Juxtapid. At the poster presentation which included data up to the cut off date of dec 2013, 17 of those patients had completed 126 weeks of treatment and their data was presented. The primary endpoint was mean percent change in low-density lipoprotein cholesterol (LDL-C) from baseline to Week 126.  

And after 126 weeks, they found the mean LDL-C levels were reduced by 45.5% (baseline: 356 +/- 127 mg/dL vs. week 126: 189 +/- 120 mg/dL; P <0.001). Similar reductions were observed for Apo B, non-HDL-C, and total cholesterol.  The table below was included in Aegerion’s press release showing the efficacy of Juxtapid to continue to improve over the course of the 126 week study.

According to the press release, the adverse event (AE) profile in the extension study was similar to the one in the pivotal trial. Gastrointestinal symptoms were the most common AE, reported in 63% (12/19) of patients in the extension study. Transient aminotransferase elevations (ALT or AST) greater or equal to 3x the upper limit of normal (ULN) occurred in 9 patients who completed Week 126 of the extension study, including 5 patients who had elevations greater than or equal to 5x ULN. One sudden cardiac death occurred in a 58 year old patient with known coronary artery disease.

At the time of this publication, the abstract was not available for review.

Homozygous familial hypercholesterolemia (HoFH)

HoFH is a rare genetic lipid disorder resulting in an accumulation of low-density lipoprotein (LDL-C) in the blood. Patients with untreated HoFH have extremely high LDL-C levels, typically between 400 mg/dL and 1,000 mg/dL. These patients are at severely high risk for premature cardiovascular events, such as heart attack or stroke. The current treatment options for these patients include dietary modifications, lipid-lowering agents (e.g., statins), and LDL-apheresis is necessary. This past year, two orphan drugs were approved to treat patients with hoFH – Juxtapid and Kynamro.


Cuchel M, Blom DJ, Averna MR, et al. Sustained LDL-C Lowering and Stable Hepatic Fat Levels in Patients with Homozygous Familial Hypercholesterolemia Treated with the Microsomal Triglyceride Transfer Protein Inhibitor, Lomitapide: Results of an Ongoing Long-Term Extension Study. American Heart Association's 2013 Scientific Sessions, Dallas, TX 2013

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