uniQure Receives Orphan Medicinal Product Designation in Europe for AMT-130 in Huntington’s disease
January 22 2018 - 7:00AM
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
diseaseHuntington’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
StatementsThis 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. CantorDirect:
339-970-7536Mobile:
617-680-9452 m.cantor@uniQure.com
Eva M. Mulder Direct: +31 20 240 6103 Mobile:
+31 6 52 33 15 79e.mulder@uniQure.com
For media:
Tom MaloneDirect: 339-970-7558Mobile:
339-223-8541t.malone@uniQure.com
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