Gene therapy with Alipogene Tiparvovec (Glybera) for Lipoprotein Lipase Deficiency shows promise

Over a 6-year period, patients with the genetic disease lipoprotein lipase deficiency (LPLD) who received a single gene therapy treatment of alipogene tiparvovec had a marked reduction in the severity and frequency of pancreatitis. No cases of severe pancreatitis and only one admission to the intensive care unit for an lipoprotein lipase deficiency related abdominal event were reported in the study published in Journal Human Gene Therapy.

The researchers assessed lipoprotein lipase deficiency related acute abdominal events that required hospital care in a small group of patients treated with a single dose of the gene therapy product Glybera®. The results support an association between gene therapy for this rare genetic disease and overall reductions in healthcare costs and resource utilization.

“As gene therapy moves forward into the mainstream of medicine, it will be critical to define the evidence of its benefit to patients with specific diseases,” says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. “This work with alipogene tiparvovec provides an evidence-based guideline for its use in patients with lipoprotein lipase deficiency going forward.”

Citation: Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis.
Gaudet Daniel, Stroes Erik S., Méthot Julie, Brisson Diane, Tremblay Karine, Bernelot Moens Sophie J., Iotti Giorgio, Rastelletti Irene, Ardigo Diego, Corzo Deyanira, Meyer Christian, Andersen Marc, Ruszniewski Philippe, Deakin Mark, and Bruno Marco J. Human Gene Therapy 2016 pp: hum.2015.158
DOI: http://dx.doi.org/10.1089/hum.2015.158
Adapted from press release by Mary Ann Liebert, Inc., publishers

Pancreatitis Reduced by Nearly 50% After Gene Therapy to Treat Lipoprotein Lipase Deficiency