Amsterdam Molecular Therapeutics (AMT), a leader in the field of human gene therapy, announced new data showing that a one-time administration of its lead product Glybera results in significant long-term health benefits. Long-term follow-up data from two clinical trials show that one administration with Glybera brings significant and clinically important reduction in acute pancreatitis in lipoprotein lipase deficient patients.

Recurrent acute pancreatitis is the most debilitating complication of lipoprotein lipase deficiency (LPLD) and is associated with significant morbidity and mortality. These data were presented at the International Symposium on Atherosclerosis in Boston, one on the most prestigious conferences on arterial disease well-attended by expert physicians.

The three year follow-up data for the eight patients from the first clinical trial show a statistically significant, tenfold decrease in the incidence of acute pancreatitis. From the second trial in fourteen patients one year data are available showing similar results. The data from both trials also confirm that the treatment is well-tolerated and safe.

AMT has developed Glybera as a treatment for patients with the genetic disorder lipoprotein lipase deficiency. 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. In the second trial it was observed that the white colouring of blood vessels in het eyes of patients (lipemia retinalis) disappeared after administration of Glybera showing that this gene therapy lowers the chylomicron levels in the blood. The disease can result in difficult-to-treat diabetes and is associated with significant morbidity and mortality.

AMT has a unique production platform that circumvents the obstacles that have hindered development of gene therapy technologies into drugs.