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Gene Silencing Drug Wins Approval

The US Food and Drug Administration has approved Kynamro, a gene silencing drug from Isis Pharmaceuticals, to treat homozygous familial hypercholesterolemia, The New York Times reports. The drug will be marketed by Sanofi's Genzyme division.

Kynamro, or mipomersen, inhibits the apolipoprotein B gene, whose gene product is involved in carrying cholesterol in the blood, working to lower levels of LDL cholesterol. In a trial, the drug decreased mean LDL levels by 24.7 percent, as compared with a 3.3 percent decrease in people who received a placebo, the Times says. The drug will, though, come with a warning about the risk of liver damage.

Homozygous familial hypercholesterolemia affects a small number of people, and the drug is unlikely, the Times notes, to be a blockbuster, especially as it can have some serious side effects. "Still, Kynamro could become the first commercial success for the gene silencing technique, which is known as antisense, and which some experts say is finally poised to fulfill its promise after over two decades of research and numerous disappointments," the Times adds.

Our sister publication Gene Silencing News has more on this here.

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