The European Commission has authorised the marketing of Glybera, a treatment for lipoprotein lipase (LPL) deficiency. This is the first gene therapy treatment approved in the 27 countries of the European Union.
LPL is a very rare disease caused by difficulties to metabolise fat particles in the blood. Until now, the only solution was a diet low in fat, less than 20g per day. However, patients continue to suffer from abdominal pain and severe and regular inflammations of the pancreas.
Developed by the Dutch laboratory UniQure, Glybera uses a vector to deliver the LPL gene to the muscle cells of the patient. The treatment will be available during the summer of 2013 and will be administered to adult patients at centres of excellence. The laboratory will conduct a follow-up to monitor the effects of the drug over time.
