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Hongwei ZhangHongwei Zhang, Ph.D., is assistant professor in the Department of Pharmaceutical Sciences at South Dakota State University.

 
As expected, uniQure’s Glybera (alipogene tiparvovec) was finally granted a commercial authorization by the European Commission on November 2, 2012, after the recommendation by the European Medicines Agency this summer. That means the first gene therapy drug in the Western world will be commercially available on market in all 27 member states of the European Union (EU). In the middle of 2013, the first patient to be treated by the commercial gene therapy product will receive this drug in Germany.

Glybera is a recombinant adeno-associated virus (rAAV)-based gene medicine to treat a rare inherited metabolism disorder, called lipoprotein lipase deficiency (LPLD). Prior to the approval of this innovative drug, there was no effective treatment for LPLD, which leads to frequent bouts of life-threatening pancreatitis and early-onset of diabetes and cardiovascular complications. Currently, the only strategy to address the disease is to severely restrict the level of dietary fat for the patients. Now, for the first time, LPLD patients have a medical treatment option to manage the disease and improve the life quality. They definitely deserve access to the new therapeutic modality, although the small market (1-2 patients per million population) may not make the drug very profitable.

Without question, this approval is another milestone in the history of gene therapy. To a large extent, it helped smooth the way for other gene therapies in development, such as those for hemophilia, retinal degeneration, and Parkinson’s disease, to get approved in the EU or United States. Considering the big market for these more common diseases, it’s very possible that gene therapy will become a disruptive technology in medicine field, although it will not replace the traditional small molecular weight drugs and protein/peptide drugs. It is also expected that the research/development mode and the regulatory process of gene therapy, as well as the health insurance system will experience necessary adjustment to get prepared for the new era of commercial development of gene therapy.

Until now, the U.S. Food and Drug Administration (FDA) has not yet approved any gene therapies. uniQure, the Amsterdam-based company, is planning to apply for regulatory approval to the FDA and sell Glybera in the U.S. market in 2014.

Do you think the FDA will approve the usage of Glybera in the United States?

Read Hongwei's original blog post on gene therapy here.