The Western world’s 1st gene therapy will not return to the European market after its 5-year conditional approval expires on Oct. 25 this year, according to UniQure (NSDQ:QURE).
The company’s Glybera product will only be available for patients who were approved for treatment and reimbursement before Oct. 25, UniQure partner Chiesi said today.
“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,” CEO Matthew Kapusta said in prepared remarks. “Glybera’s usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead.”
“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.”
The European Commission gave UniQure a 5-year marketing authorization for Glybera in October 2012, approving it as a treatment for patients with familial lipoprotein lipase deficiency – a rare genetic disorder.
The conditional approval required UniQure to establish a global registry for the long-term surveillance of patients, conduct a post-approval clinical study, submit for annual regulatory examinations and incorporate additional risk management procedures, the company said.
UniQure spokesman Tom Malone told Xconomy that the company didn’t have the commercial market to make selling Glybera worthwhile – since the gene therapy was approved for reimbursement in 2014, only 1 patient has received Glybera.
