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EC approves gene therapy Glybera

November 3, 2012 1:08 AM UTC

The European Commission approved Glybera alipogene tiparvovec from uniQure B.V. (Amsterdam, the Netherlands) to treat lipoprotein lipase (LPL) deficiency in patients with recurring, acute pancreatitis. Glybera is the first gene therapy approved outside of China. The company plans to scale up manufacturing for Glybera and incorporate EMA's request for an additional purification step before launching it in Europe in 2H13, initially in Germany. uniQure said it plans to set Glybera's price at about EUR 250,000 ($322,625) per year for the "duration of clinical benefit," which was about five years in clinical trials. 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. ...