European Commission OKs first gene therapy, uniQure's Glybera

After a somewhat rocky past, and a change of opinion by the European Medicines Agency’s opinion in the summer (The Pharma Letter July 23), the European Commission has now granted final approval for venture capital backed uniQure’s gene therapy Glybera (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD, also called familial hyperchylomicronemia) suffering from recurring acute pancreatitis.

The approval makes Glybera the first gene therapy approved by regulatory authorities in the Western world, said uniQure, which acquired rights to the product along with its purchase of licensee Amsterdam Molecular Therapeutics, in what was seen as the only solution to gaining funding to continue the Dutch firm’s stem cell therapies R&D activities (TPL February 22). 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.

Drug expected to have a dramatic impact on patients’ lives