Europe approves first gene therapy

In a ground-breaking move, the first drug that can rectify faults in the genetic code has been given the go-ahead in Europe, directing the way for the development of new drugs in the future.

The Glybera drug, developed by UniQure, is to be used to counteract a disorder known as Lipoprotein Lipase Deficiency, a syndrome that affects around two people in every million and can cause critical inflammation of the pancreas. Sufferers can not handle fat particles in the blood and the end result can be lethal.

A single treatment of Glybera is enough to treat the disorder.

Although two gene therapies have previously been approved in China, the drug is the first to be endorsed by the European Medicines Agency with the European Commission and should be obtainable next year.

The drug will cost £1 million per patient, and governments are in debate over ways to pay for the drug before it is released on to the market in 2013. Some may decide to pay £200,000 per year over a five-year period.

This type of therapy will no doubt raise many questions and is just the beginning as some of the world’s leading pharmaceutical companies start researching different types of gene therapy.

Chief executive of UniQure John Aldag said: “This final approval of Glybera from the European Commission marks a major step forward in making gene therapies available not only for Lipoprotein Lipase Deficiency, but also for a large number of rare diseases with a very high unmet medical need.”

James Clark