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FDA Approves Kynamro (Mipomersen) for Inherited Cholesterol Disorder
First systemic antisense drug to reach the market (Jan. 29)
The FDA has approved Kynamro (mipomersen sodium, Genzyme/Isis Pharmaceuticals), given as a 200-mg weekly subcutaneous injection, as an adjunct to lipid-lowering medications and diet to reduce low-density lipoprotein cholesterol (LDL-C), apolipoprotein B (apoB), total cholesterol, and non–high-density lipoprotein cholesterol (non–HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).
HoFH is a rare inherited disorder that makes the body unable to remove LDL-C — often called the “bad” cholesterol — from the blood, causing abnormally high levels of circulating LDL-C. In the U.S., HoFH affects approximately one in one million individuals. For those with HoFH, heart attacks and death often occur before the age of 30.
The FDA’s approval of Kynamro (mipomersen) is based on results from a randomized, double-blind, placebo-controlled trial that enrolled 51 HoFH patients aged 12 to 53 years — including seven patients aged 12 to 16 years — who were maintaining a regimen of maximally tolerated lipid-lowering medications. Treatment with Kynamro further reduced LDL-C levels by an average of 113 mg/dL (or 25%) from a treated baseline of 439 mg/dL, and further reduced all measured endpoints for atherogenic particles. These findings were published in The Lancet in March 2010.
Kynamro (mipomersen) is an oligonucleotide inhibitor of apoB-100 synthesis and is the first systemic antisense drug to reach the market. It is metabolized without affecting the cytochrome P450 pathways used by commonly prescribed drugs and therefore has the potential for no drug–drug interactions. No clinically relevant pharmacokinetic interactions were reported between Kynamro and warfarin, or between Kynamro and simvastatin or ezetimibe.
Source: Isis Pharmaceuticals; January 29, 2013.