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Cardiometabolic Health Congress Expert Faculty Evaluate the Clinical Implications of Newly FDA-Approved Treatment for Homozygous Familial Hypercholesterolemia
Date:1/8/2013

Boston, MA (PRWEB) January 08, 2013

The U.S. Food and Drug Administration (FDA) approved JUXTAPID™ (lomitapide), a new, first-in-class treatment option for clinical management of patients with homozygous familial hypercholesterolemia (HoFH) on December 28, 2012. The FDA decision prompted the Cardiometabolic Health Congress (CMHC) expert faculty to examine the implications this new treatment option may have on physician practice and patient care.

HoFH, an inherited genetic disorder caused by a mutation to the LDL receptor, is associated with early onset of atherosclerosis and premature cardiac death. Patients with HoFH tend to respond poorly to statin therapy because of low LDL receptor activity. For patients with HoFH who do not respond to statin therapy, LDL apheresis is indicated. This approach is expensive, invasive, and not universally available or effective alone – many patients do not achieve target LDL-C levels and as many as 30 percent of pediatric patients receiving long-term LDL apheresis progress to atherosclerotic disease of the coronary arteries and/or aorta.

Daniel Rader, MD, Cooper-McClure Professor of Medicine and Pharmacology and chief of the Division of Translational Medicine and Human Genetics in the Department of Medicine, the Perelman School of Medicine at the University of Pennsylvania, stated, “The magnitude of LDL-C and apoB reduction seen with lomitapide in patients with HoFH is substantially greater than that achieved with any other pharmacologic therapies in this disease and is expected to change the course of the disease and delay the onset and progression of atherosclerotic cardiovascular disease.”

For patients with HoFH, heart and vascular disease often develop in childhood, and the average age of death is approximately 30 years. For this reason, Robert H. Eckel, MD, co-chair of th
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