EU watchdog disagrees with negative view on UniQure gene therapy

06 May, 2015 6:18 pm

LONDON – European regulators do not agree with the negative view expressed by one adviser about the efficacy of UniQure’s drug Glybera, the Western world’s first gene therapy.

The Dutch company said it had received a final assessment report from the European Medicines Agency’s Committee for Advanced Therapies (CAT) following doubts raised by the adviser, a so-called rapporteur.

“The CAT did not agree with the negative view of the rapporteur,” UniQure said in a statement, adding that the views of the CAT were endorsed by the agency’s senior body, the Committee for Human Medicinal Products.

UniQure’s Nasdaq-listed shares fell last month when it emerged that the rapporteur believed Glybera lacked efficacy. A German regulator for new drugs also suspended its assessment of the product.

The drug, which is sold by UniQure’s unlisted Italian marketing partner Chiesi for treating an ultra-rare blood disorder, has had a tortuous journey to market as regulators have struggled to assess its effectiveness because of the very small number of patients available for clinical tests.


The medicine was approved in Europe two years ago but its launch was delayed to allow for the collection of six-year follow-up data on its benefits.

UniQure is also developing other gene therapies that are viewed as more important to its commercial future. In April it signed a major collaboration deal with Bristol-Myers Squibb. – Reuters




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