uniQure Will Not Renew Marketing Authorization of Glybera

April 20, 2017

On April 20, 2017 UniQure announced that it will not pursue marketing authorization renewal in the European Union for Glybera (alipogene tiparvovec), the first gene-therapy approved by EMA. Glybera’s marketing authorization is set to expire on Oct. 25, 2017.

On April 20, 2017 UniQure announced that it will not pursue marketing authorization renewal in the European Union for Glybera (alipogene tiparvovec), the first gene-therapy approved by EMA. Glybera’s marketing authorization is set to expire on Oct. 25, 2017.

"The decision to not pursue marketing authorization renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern," said Matthew Kapusta, CEO of uniQure in a statement. "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."

Glybera was approved as an orphan medicine by the EMA in 2012 as a treatment for lipoprotein lipase (LPL). According to EMA, LPL is an extremely rare disorder that the agency estimates only affects about “one or two people per million.” In addition to treating a limited number of patients, Glybera is priced at close to $1 million per treatment, Nature Biotechnology reported in 2015.

Although the drug has been on the market in Europe for several years, Glybera had a rocky path to approval. The EMA’s Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) both adopted negative opinions of the therapy in June and October 2011. After reviewing the results from the 27-person trial in October 2011, the CHMP cited insufficient reduction in blood fats and pancreatitis, and a lack of long-term data as reasons for the negative opinion. After re-evaluating studies in 2012, both committees adopted positive opinions of Glybera and the drug was later approved by EMA.

Glybera may represent a cautionary tale for companies looking to bring new gene-therapies to market. Gene-therapies can be expensive to manufacture and some orphan therapies, like Glybera, serve small patient pools.

Dan Soland, former CEO of uniQure told MIT Technology Review in 2016, “I think we learned a tremendous amount about what to do and what not to do, but commercially it has not been a success. It still drains a lot from the company.”

Source: uniQure, EMA, MIT Technology Review, Nature