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article imageFirst gene therapy in Europe approved

By Tim Sandle     Nov 2, 2012 in Science
The European Commission has approved a treatment for patients suffering from acute pancreatitis. What is remarkable about this drug approval is that it is the first gene therapy treatment to be approved for use in Europe.
The European Commission's approval follows a recommendation from the European Medicine Agency, made on November 2.
The approval is for a drug called Glybera (alipogene tiparvovec). The drug is designed to combat a rare form of acute pancreatitis which develops due to a genetically inherited protein deficiency. The disease affects about 350-700 patients in Europe.
As a result of the condition, patients are unable to metabolite fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis). This can be a potentially lethal condition.
What is remarkable about Glybera is that, up to now, no gene therapy has been approved in the E.U. or the U.S. Glybera is a genetically engineered compound and consists of a virus (Adeno-associated Virus or AAV)).
Gene therapy is simply the use of DNA as a medicine to treat disease. It is based on the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. China was the first country to officially sanction a gene therapy.
Speaking about the newly approved medicine, Professor John Kastelein from Academic Medical Center of the University of Amsterdam is quoted by EuroBioTech News as saying:
"This therapy will have a dramatic impact on the lives of these patients. Currently their only recourse is to severely restrict the amount of fat they consume. By helping to normalise the metabolism of fat, Glybera prevents inflammation of the pancreas, thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities."
According to the BBC, the manufacturer of the drug (UniQure) indicates that it will be available next year.
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