Six-year follow-up data from Glybera treated patients

Netherlands-based human gene company uniQure (Nasdaq: QURE) and privately-held Italian drugmaker Chiesi Farmaceutici have announced initial analysis of retrospectively collected individual patient data from lipoprotein lipase deficiency (LPLD) patients treated with Glybera (alipogene tiparvovec).

The analysis covers follow-up data for up to six years post treatment for 13 patients, all of whom met indication requirements for the current labeling of Glybera in the European Union. This analysis represents an extension of the case note review which formed a part of the data package upon which the European Commission approved Glybera (The Pharma Letter November 5, 2012) under exceptional circumstances for the treatment of a subset of patients with lipoprotein lipase deficiency, or LPLD, a potentially life-threatening, orphan metabolic disease. Glybera currently is not approved for use outside of the EU.

An external adjudication board of independent experts performed the review of the individual patient profiles. In the analysis equal time periods of up to six years before and after Glybera treatment were compared to evaluate the number and severity of attacks of pancreatitis in each LPLD patient with a history of severe or repeated pancreatitis. The review suggests that treatment with Glybera provides long-term beneficial effects with regard to the risk of encountering new pancreatitis events, including the occurrence of severe pancreatitis events. These results are in line with the trend exhibited in the first case note review performed up to three years after treatment with Glybera.