Netherlands-based gene therapy company uniQure has announced that it will not pursue to renew the marketing authorization of Glybera (alipogene tiparvovec) in Europe.
Glybera, the Western world’s first approved gene therapy was developed by uniQure for the treatment of lipoprotein lipase deficiency, an ultra-rare genetic disorder. In October 2012, it was a granted a five-year marketing authorization by the European Commission (EC). This conditional approval is set to expire on October 25, 2017 after which uniQure will pull the drug from the market and wind down all the related infrastructure.
As part of approval by the EC, uniQure was required to establish a global registry for the long-term surveillance of patients, conduct a post-approval clinical study, submit for regulatory reassessments and implement additional risk management procedures. These activities required a significant infrastructure and uniQure had to bear the full costs related to this. The drug did not have a large commercial market to make it worth continuing to sell and as of 2016 only one person was treated with this gene therapy.
Privately-held Italian firm Chiesi Farmaceutici held exclusive rights for the commercialization of Glybera in Europe and other selected countries, as well as for the co-development and commercialization of a gene therapy for hemophilia B. Under the terms of the agreement between uniQure and Chiesi, uniQure will continue to make product available to Chiesi to treat any patients that are approved for treatment prior to October 25, 2017.
According to Matthew Kapusta, CEO of uniQure, the decision was made after a “careful and thoughtful evaluation of patient needs and clinical use of the therapy and is not related to any risk-benefit concern. Glybera’s usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead.”
Glybera’s withdrawal is expected to reduce uniQure’s future expenses related to the product by approximately $2 million annually. The company now intends to focus the resources on advancing the hemophilia B and Huntington’s disease gene therapy programs.
Source: uniQure announces it will not seek marketing authorization renewal for Glybera in Europe. Press Release