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Lack of demand forces uniQure to withdraw Glybera gene therapy in Europe

PBR Staff Writer Published 24 April 2017

uniQure 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).

According to the Dutch gene therapy company, the usage of Glybera has been quite narrow and it would rather prefer to sustain its focus on important programs in hemophilia B, congestive heart failure and Huntington’s disease.

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.

uniQure CEO Matthew Kapusta said that the decision not to go for its renewal in Europe was not due to any risk-benefit concern but had come after a thoughtful and careful assessment of patient requirements and its clinical use.

Kapusta added: "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead.

"In line with our previously announced strategy, we will focus our resources on advancing our hemophilia B program into a pivotal trial, moving our Huntington's disease program into a clinical proof-of-concept trial, and progressing our research and development collaboration with Bristol-Myers Squibb."  

uniQure stated that it had been in talks with the European Medicines Agency (EMA) to discuss measures to cease activities related to Glybera and review plans for existing patient monitoring.

The Dutch company will continue to provide Glybera to Chiesi Group, its commercialization rights holder in Europe, to treat any patients approved for treatment before October 25. It will also handle the termination of the Phase IV post-approval trial of the gene therapy.