Lipid Insights: New Therapies for HoFH

Lipid Insights: New Therapies for HoFH

Schedule: On Demand (The original live program aired on Tuesday, March 12, 2013)

Program Overview:
HoFH is a rare genetic condition that causes severe hypercholesterolemia and leaves patients at extremely high risk for premature coronary heart disease (CHD).

Controlling dyslipidemia through lowering low-density lipoprotein cholesterol (LDL-C) levels is extremely important in patients with HoFH. Unfortunately, hypercholesterolemia in FH patients is often resistant to a single drug therapy. Cholesterol levels may not normalize or decrease to target levels, even after combination therapy with two or three drugs. HoFH patients must pursue diet and lifestyle modifications and combination therapies with statins and other lipid lowering drugs, but these options are rarely sufficient to achieve target LDL-C levels. As a result, many HoFH patients must rely on LDL apheresis, which is a method of filtering LDL and other Apo B particles from the blood. Several compounds are under development to help FH patients achieve target LDL levels.

Mipomersen sodium injectable drug, an antisense inhibitor of apolipoprotein B synthesis, was recently approved by the US Food and Drug Administration (FDA) for use as an adjunct to treatment with lipid-lowering medications and diet modifications in patients with HoFH. Specifically, the FDA approved the administration of mipomersen as a 200 mg weekly subcutaneous injection, in order to reduce the levels of LDL-C, total cholesterol (TC), apolipoprotein B (Apo B), and non-high density lipoprotein cholesterol (non-HDL-C) in patients with HoFH.

Robert A. Wild, MD, PhD, MPH, FNLA

President, Southwest Lipid Association
Professor and Chief
Gynecology, Clinical Epidemiology and Medicine
University of Oklahoma Sciences Center
Oklahoma City, OK

Peter H. Jones, MD, FACP, FNLA

Directory, Weight Management Center
Methodist Diabetes & Metabolism Institute
Associate Professor of Medicine
Center for Cardiovascular Disease Prevention
Methodist DeBakey Heart and Vascular Center
Baylor College of Medicine
Houston, TX

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Education Information

This program, though not accredited in its archived form, is targeted towards specific content areas and learning objectives.

Content Domain
This activity falls under the following content domain(s):

  • 3.1.1 Familial hypercholesterolemia (FH) and other autosomal dominant hypercholesterolemias

Learning Objectives
This activity teaches to the following learning objective(s):

  1. Evaluate the clinical effects, safety and efficacy data for current and new treatment options for FH patients.
  2. Improve the diagnosis and treatment of patients with homozygous and heterozygous FH