The Committee for Medicinal Products for Human Use (CHMP) has adopted a negative opinion, recommending that a marketing authorisation should not be granted for the orphan medicine Glybera® (alipogene tiparvovec), on the basis of the opinion of the Committee for Advanced Therapies (CAT).  Glybera is a gene-therapy product using an adeno-associated viral vector intended for the treatment of adult patients diagnosed with lipoprotein lipase deficiency demonstrating hyperchylomicronaemia or having a history of acute pancreatitis.

An accompanying Q&A document notes that the Committee, taking into account the assessment performed by the CAT, concluded that there was currently insufficient evidence of the benefits and safety of Glybera to recommend approval at this stage.  The studies provided had not shown a consistent long-lasting benefit of treatment, and there were, at present, too few patients for whom sufficiently long-term data were available. There was also insufficient evidence at this stage of a reduction in the rate of pancreatitis.