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To the Market

The European Commission has given its seal of approval to the continent’s first gene therapy — a treatment for a lipid-processing disorder — which will be sold in Europe beginning in late 2013, The Scientist reports.

According to the article, the EC has approved the use of Glybera, a therapy that “utilizes viral vectors to deliver DNA encoding a lipid-processing enzyme to patients lacking a functional copy due to a gene mutation.”

Glybera was developed by Amsterdam-based uniQure for patients suffering from a rare lipid-processing disease, called lipoprotein lipase deficiency, caused by loss-of-function mutations in the liproprotein lipase gene, The Scientist adds. The therapy is indicated for LPLD patients with severe symptoms such as life-threatening bouts of pancreatitis.

Katherine High, a pediatrician at the University of Pennsylvania and Children’s Research Hospital of Philadelphia and who was not involved in research on Glybera, notes that this marks the first time that a gene therapy has been “judged safe and efficacious for human clinical use,” according to regulatory standards used in Europe and the United States.

The Scientist also reports that UniQure is applying for approval from the US Food and Drug Administration and is also looking to bring Glybera to Korea, Israel, and countries in South America. Currently, no gene therapies have been approved for use in the United States.

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