Lack of demand forces uniQure to withdraw Glybera gene therapy in Europe stat : pharmaceutical-business-review

Lack of demand forces uniQure to withdraw Glybera gene therapy in EuropeuniQure said that due to low patient demand it will not pursue a renewal of its European marketing authorization for Glybera (alipogene tiparvovec), which is due to expire on 25 October. The marketing authorization of the gene therapy was approved in 2012 for the treatment of patients with familial lipoprotein lipase deficiency (LPLD). It will also handle the termination of the Phase IV post-approval trial of the gene therapy. To be administered as a one-time gene therapy to patients, Glybera introduces copies of natural LPL gene to form functional LPL enzyme which in turn has been shown to provide a long-term therapeutic effect. Kapusta added: "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead.




Kulraj Singh BhangraA million-dollar gene therapy treatment for a rare blood disease will be withdrawn from the European market due to lack of demand. The drug received authorisation in 2012 for the treatment of an ultra-rare blood disorder known as familial lipoprotein lipase deficiency (LPLD) and was the first ever gene therapy treatment licensed in Europe. Gene therapy company UniQure announced on 20 April that it will not pursue the European marketing renewal for its drug Glybera (alipogene tiparvovec) which is due to expire in October 2017. Very small numbers of potential patients, combined with the very high cost have resulted in demand being low. It launched two years later with a US$1 million price-tag, but has only been used on one patient in Germany since its approval.


This doesn't mean that commercial gene therapy drugs are dead... far from it. Until gene therapy drugs are relatively commonplace, pharma brands may have to accept that they won't get as much of return on investment as their patients. However, the failure suggests that how gene therapy is sold may be as important as how well it works. While the approach is still relatively exotic, it's not so exotic that pharmaceutical companies can charge a premium or expect to turn a serious profit. However, the rarity of the disease Glybera targets meant that UniQure didn't exactly have an abundance of medical data to show that the treatment worked as promised.


collected by :Lucy William

To follow all the new news about Health care