West’s First Gene Therapy Gets European Backing

Posted: July 21, 2012 at 8:10 pm

By Mark Long

The first gene-therapy drug in the Western world likely moved a step closer to the market Friday, as European regulators recommended approval of a medicine to treat a rare disease that causes fat to build up in the blood.

Dow Jones Newswires reports that a European Medicines Agency committee backed approval of Glybera, which was developed by Dutch biotech firm uniQure to treat patients with lipoprotein lipase deficiency, or LPLD.

The European Commission usually backs such recommendations from the drug regulator, which had rejected Glybera three times before.

In gene therapy, scientists try to correct a problem caused by a defective or non-functioning gene by replacing it with a functioning gene. Research efforts had faded for years amid concerns it was too risky and complex to be safe and effective. But findings from several studies rekindled interest in the approach, as WSJ reported in 2010.

No gene therapy drugs are on the market in the West, though one to treat cancer was approved in China in 2003, the Associated Press notes in its story on the thumbs up for Glybera. The AP says the agency recommended approval only with tough restrictions and uniQure will have to set up a registry to track patients.

Patients with LPLD cant handle fat particles in their blood plasma. This leads to recurring, severe abdominal pain and pancreatitis. The disease affects one or two out of every million people.

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West’s First Gene Therapy Gets European Backing

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