uniQure Announces FDA Orphan Drug Designation for AMT-130 in Huntington's disease
(Thomson Reuters ONE) -
~ First Investigational Gene Therapy in Huntington's Disease to Receive
Designation ~
LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Oct. 06, 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 AMT-130, its proprietary gene therapy candidate for Huntington's disease,
has received Orphan Drug Designation (ODD) by the U.S. Food and Drug
Administration (FDA). 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.
ODD in the United States provides a special status for investigational drugs
being developed for rare diseases considered to affect only up to 200,000 people
in the U.S. The ODD programs offer product market exclusivity for up to seven
years in the U.S. and ten years in the European Union following regulatory
approval, along with tax and financial incentives for companies developing
medicines for such orphan indications.
"Attaining orphan designation recognizes the potential that AMT-130 holds in
delivering meaningful therapeutic benefit to patients suffering from this
devastating disease," said Matthew Kapusta, chief executive officer of uniQure.
"It supports our ongoing development in Huntington's as we seek to bring the
first gene therapy approach to this disease into the clinic next year."
AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA which
silences 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.
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
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
August 8, 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(at)uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder(at)uniQure.com
For media:
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone(at)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
Datum: 06.10.2017 - 13:00 Uhr
Sprache: Deutsch
News-ID 562745
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Business News
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"uniQure Announces FDA Orphan Drug Designation for AMT-130 in Huntington's disease"
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