uniQure Receives Orphan Medicinal Product Designation in Europe for AMT-130 in Huntington's disease
January 22 2018 - 7:00AM
YASTEST
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, Jan. 22, 2018 (GLOBE NEWSWIRE) -- uniQure
N.V. (NASDAQ:QURE), a leading gene therapy company advancing
transformative therapies for patients with severe medical needs,
today announced that AMT-130, its proprietary gene therapy
candidate for Huntington's disease, has received an Orphan
Medicinal Product Designation (OMPD) from the European Medicines
Agency. AMT-130 represents the first investigational AAV-gene
therapy in Huntington's disease to receive such designation. In
October 2017 the company announced that the U.S. Food and Drug
Administration granted orphan drug designation for the same
indication. There are currently no approved medical treatments
aimed at addressing the underlying cause of Huntington's disease,
and AMT-130 has the potential to play a role in this area of high
unmet medical need.
AMT-130 consists of an AAV5
vector carrying an engineered micro-RNA specifically designed to
silence the huntingtin gene. The therapeutic goal is to inhibit the
production of the mutant protein. Using AAV vectors to deliver
micro-RNAs directly into the brain represents a highly innovative
approach to treating Huntington's disease.
"The granting of orphan drug
designation in Europe represents another important milestone for
our AMT-130 program," said Matthew Kapusta, chief executive officer
of uniQure. "Huntington's disease affects approximately 70,000
people in the U.S. and Europe1, making this
one of the largest clinical unmet needs in the rare disease field.
We expect to file an Investigational New Drug application later
this year and be the first AAV-gene therapy to enter clinical
development for Huntington's disease."
To qualify for OMPD in Europe a
therapy must be intended for the treatment of a disease that is
life-threatening or chronically debilitating and have a patient
prevalence in the European Union of no more than 5 in 10,000.
OMPD offers product market exclusivity for ten years in the
European Union following regulatory approval, along with tax and
financial incentives for companies developing medicines for such
orphan indications.
About
Huntington's disease
Huntington's disease is a rare, inherited neurodegenerative
disorder that leads to loss of muscle coordination, behavioral
abnormalities and cognitive decline, resulting in complete physical
and mental deterioration over a 12- to 15-year period of time. The
disease is caused by the expansion of CAG trinucleotide in exon 1
of a multifunctional gene coding for protein called huntingtin.
Despite the clear etiology, there are no therapies available to
treat the disease, delay onset or slow progression of a patient's
decline.
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
- http://www.ema.europa.eu
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, the development of our
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, and the scope of protection provided by our
patent portfolio. Our actual results could differ materially from
those anticipated in these forward-looking statements for many
reasons, including, without limitation, risks associated with our
and our collaborators' clinical development activities,
collaboration arrangements, corporate reorganizations and strategic
shifts, 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 Quarterly Report on Form 10-Q filed on November 1, 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
Investors:
Maria E.
Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor@uniQure.com
Eva M.
Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com
For media:
Tom
Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: uniQure N.V. via Globenewswire
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