Firm Pulls World’s First Gene Therapy Treatment: No One Wants It – NBCNews.com

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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.

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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.

"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.

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.

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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.

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.

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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.

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.

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.

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Firm Pulls World's First Gene Therapy Treatment: No One Wants It - NBCNews.com