DNA double helix Laguna Design \/ Getty Images <\/p>\n
UniQure went into the record books when its gene therapy Glybera was approved by European regulators for an ultra-rare blood disorder in 2012, and the drug was finally launched two years later with a price tag of around $1 million per treatment. <\/p>\n
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But the number of patients eligible for treatment was always tiny and, with no sign of demand improving, the company said it had decided not to renew Glybera's five-year European marketing authorization, which is due to expire on Oct. 25. <\/p>\n
\"Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead,\" said UniQure Chief Executive Matthew Kapusta. <\/p>\n
The group, which had already decided not to pursue a U.S. approval for the drug, said the decision was not related to any safety concerns. <\/p>\n
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Glybera is given as a series of injections to fight lipoprotein lipase deficiency (LPLD), a disabling condition that clogs the blood with fat. The drug is sold in Europe on UniQure's behalf by Italian drugmaker Chiesi Farmaceutici. <\/p>\n
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. <\/p>\n
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However, the setback is unlikely to derail rising investor interest in gene therapy, which has been triggered recently by a number of advances in treating a range of genetic diseases, most of which affect far more patients than LPLD. <\/p>\n
Industry analysts said the decision to pull the plug on Glybera would make little difference to UniQure's financial outlook. In fact, the move will save some $2 million in annual costs and help UniQure focus on other gene medicines. <\/p>\n
Scientists have been working on gene therapies for more than a quarter of a century but it is only recently that the approach has started to become a commercial reality, although the U.S. Food and Drug Administration has yet to approve any. <\/p>\n
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Visit link: DNA double helix Laguna Design \/ Getty Images UniQure went into the record books when its gene therapy Glybera was approved by European regulators for an ultra-rare blood disorder in 2012, and the drug was finally launched two years later with a price tag of around $1 million per treatment. Related: But the number of patients eligible for treatment was always tiny and, with no sign of demand improving, the company said it had decided not to renew Glybera's five-year European marketing authorization, which is due to expire on Oct. 25 Continue reading
\nFirm Pulls World's First Gene Therapy Treatment: No One Wants It - NBCNews.com<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"