Drug news
CHMP recommends Glybera (uniQure) gene therapy for LPLD
uniQure announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera (alipogene tiparvovec) as a treatment for Lipoprotein Lipase Deficiency (LPLD) under exceptional circumstances. LPLD is a very rare, inherited disease. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain and pancreatitis. Glybera is the first gene therapy for LPL deficient patients, a severe disease with no alternative treatment