UniQure confirmed that it will not be looking to renew European marketing approval of the familial lipoprotein lipase deficiency (LPLD) gene therapy Glybera® (alipogene tiparvovec) when the existing EC authorization expires later this year. The firm’s CEO, Matthew Kapusta, said the decision wasn't related to any risk–benefit concerns. Rather, UniQure indicated that there wasn’t enough demand for the treatment, given the significant costs associated with maintaining a manufacturing infrastructure and associated regulatory and patient monitoring activities and post-marketing trial.
“Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead,” Kapusta said in a statement.
The EC had granted a 5-year marketing authorization for Glybera under exceptional circumstances as a treatment for the familial LPLD indication in 2012. The approval, which marked the first for a gene therapy in Europe, required UniQuire to establish a global registry for the long-term surveillance of patients, carry out a post-approval clinical study, submit for annual regulatory reassessments, and establish additional risk management procedures.
UniQure says it is in discussions with the European Medicines Agency on how to wind down these activites. The company estimates that taking Glybera off the market will allow it to make product-related savings of about $2 million a year, net of any payments due to Chiesi, which holds exclusive rights for Glybera commercialization in Europe and other selected countries. UniQure will continue to provide Chiesi with the product for patients who have been approved for treatment before the current marketing authorization end date of October 25, 2017. UniQure said it will also terminate the Phase IV post-approval study.
“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,” Mr. Kapusta added.
In November last year, UniQure announced restructuring plans that would involve shedding 20% to 25% of its workforce by the end of 2017, refocusing its pipeline, and consolidating manufacturing. By the end of the year, the firm had confirmed that it wouldn’t pursue approval of Glybera in the U.S.
In January, the FDA granted UniQure’s lead hemophilia B gene therapy candidate AMT-060 Breatkthrough Therapy designation. AMT-060 is undergoing Phase I/II evaluation. Positive data from the ongoing study was reported in December.