Glybera alipogene tiparvovec regulatory update
The European Commission approved Glybera alipogene tiparvovec from uniQure to treat lipoprotein lipase (LPL) deficiency in patients with recurring, acute pancreatitis. Glybera is the first gene therapy approved by regulatory authorities outside of China. uniQure plans to launch Glybera in Europe in 2H13 starting in Germany. The company said it first has to scale up manufacturing for Glybera and incorporate an additional purification step that was requested by EMA. uniQure said it is aiming to set Glybera's price at about €250,000 ($322,625) per year for the "duration of clinical benefit," which in clinical trials has been about 5 years. This translates into a cost for Glybera of about €1.3 million ($1.6 million). The adeno-associated virus (AAV) vector encoding LPL gene is given as a single intramuscular injection. uniQure said it plans to submit a BLA to FDA for Glybera by next quarter. ...