09:30am Friday 18 October 2019

The Inside Story Behind the Approval of the Gene Therapy Drug Glybera—Politics and Intrigue Led to Major Milestone

In addition, an in-depth Review revealing the inside story of the European regulatory review and approval of Glybera—chock full of twists and turns, politics, and intrigue—and critiquing the groundbreaking drug’s path to the marketplace appears in Human Gene Therapy Clinical Development. Both articles are available free on the Human Gene Therapy website.

John Kastelein, University of Amsterdam (the Netherlands), and Colin Ross and Michael Hayden, University of British Columbia Vancouver, BC, Canada), describe the long path to the discovery of the genetic mutation responsible for lipoprotein lipase deficiency (LPLD), a rare, inherited disease, and the subsequent work in Dr. Hayden’s laboratory to develop a gene replacement therapy. In the article ” From Mutation Identification to Therapy: Discovery and Origins of the First Approved Gene Therapy in the Western World,” the authors recount the many failures and successes and the significant delays that finally ended on November 2, 2012, when the European Medicines Agency granted marketing approval for Glybera. The therapy will be submitted for review by the U.S. and Canadian regulatory authorities.

James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, and coauthors give a behind-the-scenes view of the protracted clinical development, review, and approval process for Glybera in the Commentary “Lessons Learned from the Clinical Development and Market Authorization of Glybera. ” Although Glybera’s safety was not an issue, its efficacy in the relatively small number of patients who received the therapy was questionable. The authors chronicle a bumpy road to market approval, fraught with rejections, reanalysis of study data, and appeals that led to commercialization of the product with the caveat of an ongoing patient registry to allow for continued review of the effectiveness of the therapy as it is used in more patients.

About the Journal
Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, publishes data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher
Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Nucleic Acid Therapeutics, Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry’s most widely read publication worldwide. A complete list of the firm’s 70 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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