uniQure's Glybera® First Gene Therapy Approved by European Commission

uniQure's Glybera® First Gene Therapy Approved by European Commission PR Newswire AMSTERDAM, The Netherlands, November 2, 2012 AMSTERDAM, The Netherlands, November 2, 2012 /PRNewswire/ -- Glybera becomes the first gene therapy approved by regulatory authorities in the Western world First medication approved for patients with rare metabolic disorder Lipoprotein Lipase Deficiency Commercial roll-out to begin second half of2013 Validates uniQure's unique AAV-based gene therapy platform uniQure announced today it has received approval from the European Commission for the gene therapy Glybera® (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD, also called familial hyperchylomicronemia) suffering from recurring acute pancreatitis. Patients with LPLD, a very rare, inherited disease, are unable to metabolize the fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), an extremely serious, painful, and potentially lethal condition. The approval makes Glybera the first gene therapy approved by regulatory authorities in the Western world. "Glybera's approval means LPLD patients, for the first time, have a medical treatment option for a very complex and severe disease," said Professor John Kastelein of the Department of Vascular Medicine at the Academic Medical Center of the University of Amsterdam, the Netherlands.