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Clinical Trials Update
November 26, 2014

Evolocumab Lowers LDL in Genetic High Cholesterol Disorder

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Copyright 2014 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA. 2014;312(20):2083. doi:10.1001/jama.2014.15519

Evolocumab (AMG145), inhibitor of an enzyme (PCSK9) that facilitates degradation of low-density lipoprotein (LDL) receptors, led to 60% greater reduction in LDL cholesterol than placebo in 331 patients with heterozygous familial hypercholesterolemia, reported a manufacturer-funded multicenter randomized trial (Raal FJ et al. Lancet. doi:10.1016/S0140-6736(14)61399-4 [published online October 2, 2014]).

Familial hypercholesterolemia is thought to be the most common disorder inherited in autosomal-dominant fashion, affecting between 1 in 250 to 300 people worldwide and more than 3 million people in the United States and Europe. Intensive statin therapy often fails to reduce LDL cholesterol to levels sufficient to prevent premature cardiovascular disease for people with the disorder.

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