Firm Pulls World's First Gene Therapy Treatment: No One Wants It stat : NBC News

The biotech company behind the Western world's first gene Therapy and the most expensive prescription medicine in history is giving up on the product because of lack of demand. Related: Gene Therapy Offers Hope to Cancer PatientsGlybera is given as a series of injections to fight lipoprotein lipase deficiency (LPLD), a disabling condition that clogs the blood with fat. In fact, the move will save some $2 million in annual costs and help UniQure focus on other gene medicines. Only one patient has been treated commercially since the $1 million treatment was first approved in Europe nearly five years ago, a spokeswoman for Dutch-based UniQure said on Thursday. The commercial flop is a reminder of the economic challenges facing the emerging field of gene therapy, which seeks to cure rare genetic diseases by offering a one-time fix of a faulty DNA but inevitably comes at a very high price.




First Gene Therapy Ever Approved Calling it QuitsJames RadkeNot a Money MakerWhat is Familial Chylomicronemia Syndrome (FCS)uniQure, the first and only company to have a gene therapy approved, will not seek to renew its gene therapy approval in Europe.In October 2012, the European Commission gave uniQure a 5-year marketing authorization for Glybera to treat patients with familial lipoprotein lipase deficiency, also known as familial chylomicronemia syndrome (FCS).In 2014, the gene therapy was finally launched with a price facebook/" target="_blank">tagof $1 million per treatment. Currently, there are several drugs in development to treat FCS but at present, there no approved drugs for this condition. What ultimately deterred uniQure from wanting to renew the approval is that the demand is low.Really low. Patients must adhere to a strict low fat diet to reduce the risk of pancreatitis. Since 2014, only 1 patient has received Glybera.It is not clear if the low demand is due to insurance companies saying no to the price tag or the fact that many of the FCS patients that can get the gene therapy already got it in the clinical trials.


UniQure to Yank Pioneering Gene Therapy From Market in EuropeXconomy Boston —[Updated, 9:33 a.m. ET, see below] It took decades to get the first gene therapy in the Western world to market. Said Van Deventer in 2016, many of the gene therapy companies "I'm pretty sure wouldn't have been there if there were not Glybera." UniQure (NASDAQ: QURE), with operations in Amsterdam and Lexington, MA, said this morning that it will not seek to renew marketing authorization of alipogene tiparvovec (Glybera), the Western world's first approved gene therapy, in Europe. Gene therapy has since pushed forward more rapidly.