uniQure has announced that it will not pursue the renewal of the Glybera ( Alipogene tiparvovec ) marketing authorization in Europe when it is scheduled to expire on October 25, 2017.
According to uniQure the decision to not pursue marketing authorization renewal of Glybera in European Union 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.
Glybera’s usage has been extremely limited and uniQure does not envision patient demand increasing materially in the years ahead.
In October 2012, the European Commission granted a five-year marketing authorization for Glybera under exceptional circumstances as a treatment for a small subset of patients with familial lipoprotein lipase deficiency ( LPLD ), an ultra-rare genetic disorder.
As part of Glybera’s approval, uniQure was required to establish a global registry for the long-term surveillance of patients, conduct a post-approval clinical study, submit for annual regulatory reassessments and implement additional risk management procedures.
All of these activities required a significant infrastructure for uniQure that included the Company bearing the full costs of maintaining commercial manufacturing capabilities, managing development and validation of numerous assays and supporting regulatory interactions and inspections.
uniQure has initiated discussions with the European Medicines Agency ( EMA ) to discuss steps to wind down these various activities and review plans for ongoing patient monitoring.
Under the terms of the agreement between uniQure and Chiesi Group, which has exclusive rights for the commercialization of Glybera in Europe and other selected countries, uniQure will continue to make product available to Chiesi to treat any patients that are approved for treatment prior to October 25, 2017, and will also be responsible for terminating the phase IV post-approval study.
As a result of the withdrawal of Glybera, uniQure expects to reduce future expenses related to the product by approximately $2 million annually, beginning in 2018 and net of any payments to Chiesi.
These cost savings will be in addition to those previously announced by the Company related to the consolidation of manufacturing into the Company’s Lexington facility.
uniQure continues to expect its existing cash resources will be sufficient to fund operations into 2019.
Glybera is a one time, single-administration gene therapy, which introduces copies of natural LPL gene to produce functional LPL enzyme, providing a long-term therapeutic effect, currently documented up to six years from administration. ( Xagena )
Source: uniQure, 2017