-- Marketing Authorization for
Glybera® to Expire on October 25, 2017 --
-- Company Maintains Focus on Core
Programs in Hemophilia B, Huntington's Disease and Congestive Heart
Failure --
LEXINGTON, Mass. and AMSTERDAM, the Netherlands,
April 20, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a
leading gene therapy company advancing transformative therapies for
patients with severe medical needs, today 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.
"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," stated
Matthew Kapusta, chief executive officer of uniQure.
"Glybera's usage has been extremely limited and we do not envision
patient demand increasing materially in the years ahead."
Mr. Kapusta added, "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."
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.
About Glybera:
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.
Chiesi Group has exclusive rights
for the commercialization of Glybera in Europe and other selected
countries, as well as for the co-development and commercialization
of a gene therapy for hemophilia B.
About
uniQure
uniQure is delivering on the
promise of gene therapy - single treatments with potentially
curative results. We are leveraging our modular and validated
technology platform to rapidly advance a pipeline of proprietary
and partnered gene therapies to treat patients with hemophilia,
Huntington's disease and cardiovascular
diseases. www.uniQure.com
uniQure
Forward-Looking Statements
This press
release contains forward-looking statements. All statements other
than statements of historical fact are forward-looking statements,
which are often indicated by terms such as "anticipate," "believe,"
"could," "estimate," "expect," "goal," "intend," "look forward to",
"may," "plan," "potential," "predict," "project," "should," "will,"
"would" and similar expressions. Forward-looking statements are
based on management's beliefs and assumptions and on information
available to management only as of the date of this press release.
These forward-looking statements include, but are not limited to,
statements regarding the winding down of our Glybera program, the
development of our other gene therapy product candidates, the
success of our collaborations and the risk of cessation, delay or
lack of success of any of our ongoing or planned clinical studies
and/or development of our product candidates. Our actual results
could differ materially from those anticipated in these
forward-looking statements for many reasons, including, without
limitation, risks associated with corporate reorganizations and
strategic shifts, collaboration arrangements, our and our
collaborators' clinical development activities, regulatory
oversight, product commercialization and intellectual property
claims, as well as the risks, uncertainties and other factors
described under the heading "Risk Factors" in uniQure's 2016 Annual
Report on Form 10-K filed on March 15, 2017. Given these risks,
uncertainties and other factors, you should not place undue
reliance on these forward-looking statements, and we assume no
obligation to update these forward-looking statements, even if new
information becomes available in the future.