uniQure on Thursday announced that it would not pursue renewal of its European marketing authorisation for Glybera (alipogene tiparvovec) when it expires on October 25, due to a lack of demand for the gene therapy. CEO Matthew Kapusta cautioned "the decision…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." He added that "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."
The European Commission granted Glybera a five-year marketing authorisation in 2012 for the treatment of patients with lipoprotein lipase deficiency who experience recurring acute pancreatitis. Glybera, the first gene therapy to be approved in the Western world, was later launched at a cost of 1.1 million euros ($1.2 million). According to uniQure spokesman Tom Malone, only one patient has received Glybera since it was approved for reimbursement in 2014.
Under the European authorisation, uniQure said it had to create a global registry for long-term patient surveillance, conduct a post-approval clinical study, apply for annual regulatory reassessments and implement additional risk-management procedures, "all of [which] required a significant infrastructure…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 indicated that it has started discussions with the European Medicines Agency concerning steps to end these activities and review plans for ongoing patient monitoring. The drugmaker stated that it will continue to make Glybera available for patients approved for treatment prior to October 25, under the terms of its agreement with Chiesi Farmaceutici, which holds exclusive rights to market the gene therapy in Europe and other selected countries.
Meanwhile, uniQure estimated that its decision will reduce future expenses related to Glybera by $2 million annually beginning next year. Kapusta said "in line with our previously announced strategy, we will focus our resources on advancing our haemophilia B programme into a pivotal trial, moving our Huntington's disease programme into a clinical proof-of-concept trial, and progressing our R&D collaboration with Bristol-Myers Squibb."
uniQure decided against seeking US regulatory approval for Glybera in 2015 after the FDA said the company would have to provide data from another clinical trial before it could make a decision.
For some previous analysis regarding Glybera, read ViewPoints: Glybera broke down some barriers for gene therapy, but plenty more to go.
To read more Top Story articles, click here.