On Friday, July 20th, the European Medicines Agency (EMA) for the first time recommended approval of a gene therapy product in the European Union. The landmark ruling is a big win for Dutch company uniQure, as well as potentially the entire field of gene therapy, which features a number of companies in North America and Europe pursuing this treatment method across a wide range of indications.
uniQure is the maker of Glybera, a gene therapy for lipoprotein lipase deficiency (LPLD). The therapy, administered once, provides the gene to produce the protein missing in patients with the LPLD disorder. The EMA decision is an exciting development and holds promise, though it won’t necessarily provide a perfect roadmap to approval for other gene therapy companies. The approved indication population for Glybera is quite small and the therapy has been tested in only 27 patients without extensive clinical trials. Further, the EMA is requiring the company to continue collecting data on the outcomes of all patients treated and to provide that data to them on an ongoing basis.
uniQure is pursuing gene therapy research in other indications and last month signed a collaboration agreement with University of California, San Francisco (UCSF) Professor Krystof Bankiewicz to explore the use of uniQure’s technology in Parkinson’s Disease.
A gene therapy product has yet to be approved in the United States, but many are in the pipeline. This complex and fascinating technology typically uses viruses as vectors to introduce the desired genes (DNA) into the cells of an afflicted patient in order to produce a desirable therapeutic outcome. We expect to hear much more about gene therapy as results from currently in-progress clinical trials are delivered.