Drug to Lower LDL

New Type of Drug to Lower LDL Looks Promising

Published: Oct 2, 2013

By Todd Neale, Senior Staff Writer, MedPage Today

Reviewed by Zalman S. Agus, MD; Emeritus Professor, Perelman School of Medicine at the University of Pennsylvania

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Action Points

• Loss-of-function mutations in PCSK9 resulted in reduced plasma LDL cholesterol and a decreased risk of coronary heart disease, a study found.
• This study suggests that inhibition of PCSK9 synthesis by RNA interference provides a novel, potentially safe mechanism to reduce LDL cholesterol concentration in healthy individuals with raised cholesterol.

Using RNA interference to inhibit the synthesis of PCSK9 in the liver may be a new approach to lowering LDL cholesterol levels, a phase I study in healthy volunteers with elevated cholesterol suggested.

A single infusion of the intravenous RNA interference drug ALN-PCS resulted in a rapid drop in circulating PCSK9 levels accompanied by significant reductions in LDL cholesterol, according to Kevin Fitzgerald, PhD, of Alnylam Pharmaceuticals, which is developing the drug, and colleagues.

At the highest dose evaluated, there was an average LDL cholesterol reduction of 40% from baseline relative to placebo (P lower than 0.0001) they reported online in The Lancet

Source reference:Burnett J, Hooper A "Running interference to lower cholesterol" Lancet2013; DOI: 10.1016/ S0140­6736(13)61910­8. ./.../