Glybera alipogene tiparvovec regulatory update

EMA's CHMP recommended approval of an MAA from uniQure for Glybera alipogene tiparvovec to treat lipoprotein lipase (LPL) deficiency in patients with severe or multiple pancreatitis attacks, despite dietary fat restrictions. CHMP said it reversed its 3 prior negative opinions following a draft positive opinion in June from the Committee for Advanced Therapies (CAT), which conducted additional analyses of the data for the adeno-associated virus (AAV) vector encoding LPL gene. CHMP said Glybera should be approved under EMA's exceptional circumstances clause, and that the company be required to establish a patient registry to monitor outcomes. uniQure expects a final decision from the European Commission within 3 months. EMA said Glybera is the first gene therapy to receive a positive CHMP recommendation. ...