Effects of Lomitapide on HoFH and Stock Prices


Marina Cuchel MD, Assistant Professor of Medicine at the University Of Pennsylvania School Of Medicine recently provided Rare Disease Report with an exclusive look at her poster presentation of lomitapide's efficacy and safety in patients with homozygous familial hypercholesterolemia (HoFH) at the DIA/NORD Conference on Rare Diseases and Orphan Products.  This data was recently published in The Lancet.

Here is a link to her poster presentation.

The data she presented is not only of great interest to caregivers of HoFH patients, it is also of interest to investors of either Aegerion Pharmaceuticals (the makers of  lomitapide) and Isis Pharmaceuticals [the makers of mipomersen sodium (Kynamro) (along with Genzyme)]. Both lomitapide and Kynamro are under review by the FDA for treatment of patients with HoFH.  And both drugs were recently recommended by FDA advisory committees although the recommendation for lomitapide was almost unanimous (13-2) while the recommendation for Kynamro was less decisive (9-6). The differences in voting may also explain Aegerion’s and Isis’s recent stock performances (see below). The data by Dr. Cuchel is the first time the public has had an opportunity to see some of what the FDA and the FDA advisory committees saw.

aegerion one month stock 248x147.jpgisis one month stock 248x147.jpg

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 of 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 in many cases, regular and costly LDL-apheresis is necessary. These treatments are often ineffective in reducing LDL-C to recommended levels in patients with HoFH.  Another form of FH, heterozygous FH can usually be treated with statins and diet modification.

The efficacy and safety of lomitapide of adults with HoFH

The presentation by Dr. Cuchel focused on the safety and efficacy of lomitapide. In this single-arm, open-label, phase 3 study, 29 adults with HoFH from 11 centers in four countries (USA, Canada, South Africa, and Italy) were recruited. Of those,  23 completed both the efficacy phase (26 weeks) and the full study (78 weeks). At the end of the 26 week efficacy phase, LDL cholesterol was reduced by 50% (baseline mean = 8.7 mmol/L vs week 26 mean =4.3 mmol/L; p<0·0001). Concentrations of LDL cholesterol remained reduced by 44% at week 56 (p<0·0001) and 38% at week 78 (p<0·0001). Gastrointestinal symptoms were the most common adverse event.

To see Dr. Cuchel’s discussion of this study, please go to

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