The European Commission (EC) has given final approval to uniQure’s gene therapy Glybera® for the treatment of lipoprotein lipase deficiency (LPLD) patients with recurring acute pancreatitis. This reportedly marks the first approval in the Western world of a gene therapy. It follows the 2003 approval in China of Gendicine, a head and neck cancer therapy developed by Shenzhen SiBiono GeneTech Co. Ltd.
University of Amsterdam Professor John Kastelein reportedly said that the therapy will dramatically affect LPLD patients whose pancreatitis attacks often cause early onset diabetes and cardiovascular complications. “Glybera’s approval means LPLD patients, for the first time, have a medical treatment option for a very complex and severe disease,” he said. “By helping to normalize 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.”
uniQure CEO Jörn Aldag said, “The final approval of Glybera from the EC marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare diseases with a very high unmet medical need. The EC’s approval is an important validation of our innovative product platform and offers strong support for our other advanced development programs, which focus on acute intermittent porphyria, Sanfilippo B, hemophilia B and Parkinson’s disease.” See uniQure Press Release, November 2, 2012.