First gene therapy in Europe

The European Commission confirmed the EMA’s recommendation for market authorisation for the gene therapy Glybera (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD) suffering from recurring acute pancreatitis. The rare, inherited disease affects about 350-700 patients in Europe. Patients are unable to metabolise fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), a potentially lethal condition. Up to now, no gene therapy has been approved in the EU or the US. Glybera consists of an Adeno-associated Virus (AAV) vector expressing the faulty LPL enzyme.

“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”, commented Professor John Kastelein from Academic Medical Center of the University of Amsterdam. “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.”