uniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe
April 20 2017 - 7:00AM
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.
uniQure Contacts:
For Media:
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com
For Investors:
Eva M. Mulder
Direct: +31 20 240 6103
Mobile:+31 6 52 33 15 79
e.mulder@uniQure.com
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Jun 2024 to Jul 2024
uniQure NV (NASDAQ:QURE)
Historical Stock Chart
From Jul 2023 to Jul 2024