Amsterdam Molecular Therapeutics (AMT), a leader in the field of human gene therapy, released data showing that its gene therapy Glybera (alipogene tiparvovec) significantly reduces the risk of pancreatitis in patients with Lipoprotein Lipase Deficiency (LPLD). Pancreatitis, or inflammation of the pancreas, is a major clinical symptom of LPLD. It causes severe abdominal pain and often leads to hospitalization of patients as well as other complications such as diabetes and early atherosclerosis. Data were presented at the 79th European Atherosclerosis Society Meeting (26-29 June 2011, Gothenburg, Sweden).

“These data demonstrate the dramatic impact Glybera can have in the lives of LPLD patients. Pancreatitis is a debilitating and extremely painful condition. Many patients end up in the hospital emergency room and try to control the pain by any means,” explained Janneke de Wal, director of Global Sales & Marketing at AMT. “By reducing the incidence of pancreatitis episodes, Glybera has the potential to help “normalize” the day to day lives of patients affected by this disease and prevent the often frequent trips to hospital.”

Historical data on hospital presentations due to abdominal pain from 17 patients already treated with Glybera were collected in this case control study. Blinded event assessment was done by an expert adjudication committee using a modified Atlanta Diagnostic Criteria for acute pancreatitis to allow for incomplete historical data. A statistically significant (p=0.0434) reduction in the risk of acute pancreatitis was seen when the period from the first pancreatitis event to administration of Glybera was compared with the post-therapy period (median = 2.9 years). The hazard ratio, a measure of how often an event occurs over time, indicated a 63% reduction in risk of acute pancreatitis after administration of Glybera (95% CI 0.142-0.971).

AMT has developed Glybera as a treatment for patients with the genetic disorder LPLD. The dossier for Glybera that was submitted to the European Medicines Agency is in the process of being submitted for re-evaluation. LPLD is an orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. Glybera has orphan drug status in the EU and US.

AMT is a world leader in the development of human gene based therapies. In addition to Glybera, AMT has a product pipeline of several gene therapy products in development for hemophilia B, Duchenne muscular dystrophy, acute intermittent porphyria, Parkinson’s disease and SanfilippoB.