Rare Disease Report

First Gene Therapy Ever Approved Calling it Quits

APRIL 21, 2017
James Radke
uniQure, the first and only company to have a gene therapy approved, will not seek to renew its gene therapy approval in Europe.
In October 2012, the European Commission gave uniQure a 5-year marketing authorization for Glybera to treat patients with familial lipoprotein lipase deficiency, also known as familial chylomicronemia syndrome (FCS).
In 2014, the gene therapy was finally launched with a price tag of $1 million per treatment. What ultimately deterred uniQure from wanting to renew the approval is that the demand is low.
Really low. Since 2014, only 1 patient has received Glybera.
It is not clear if the low demand is due to insurance companies saying no to the price tag or the fact that many of the FCS patients that can get the gene therapy already got it in the clinical trials. FCS is an extremely rare condition – affecting only 1 in 1 million people.
In a news release,  Matthew Kapusta, chief executive officer of uniQure said,  “Glybera’s usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead.”
Kapusta added, “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.”

Not a Money Maker

By withdrawing the gene therapy from the market, uniQure expects to reduce future expenses related to the product by approximately $2 million annually.
Most of the savings will come from the company terminating the additional studies required of them by the EU following the 5-year conditional approval of the therapy.
While Glybera was not a success, the company will hopefully learn from their mistakes as they continue to pursue gene therapies for hemophilia B and Huntington’s disease.

What is Familial Chylomicronemia Syndrome (FCS)

FCS is a rare genetic disease characterized by extremely high triglyceride levels and risk of pancreatitis. Currently, there are several drugs in development to treat FCS but at present, there no approved drugs for this condition. Patients must adhere to a strict low fat diet to reduce the risk of pancreatitis. For more information about FCS, visit fcs.raredr.com
In the clips below, James Underberg, MD, of NYU Langone Medical Center describes FCS and how it is currently managed in the U.S.



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