ProQR Receives Orphan Drug Designation from EMA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa
November 29 2017 - 7:00AM
Key Updates
ProQR Therapeutics N.V.(Nasdaq:PRQR), a company dedicated to
changing lives through the creation of transformative RNA medicines
for the treatment of severe genetic rare diseases including cystic
fibrosis, Leber's congenital amaurosis 10 and dystrophic
epidermolysis bullosa, today announced that investigational drug
QR-313 for dystrophic epidermolysis bullosa (DEB) has received
orphan drug designation (ODD) from the European Medicines Agency
(EMA). QR-313 is a first-in-class RNA-based oligonucleotide
designed to address the underlying cause in dystrophic
epidermolysis bullosa (DEB) due to mutations in exon 73 of the
COL7A1 gene. DEB is a rare genetic disease that can lead to severe
blistering of the skin resulting in high treatment burden and poor
quality of life for patients.
In September 2017, QR-313 also received ODD from the FDA.
This marks the fifth drug candidate in the company’s pipeline to
receive ODD from the FDA and EMA. A first-in-human clinical
trial of QR-313 is expected to be initiated in 2018, with interim
data readout also expected in 2018.
“We are pleased to have orphan drug designation for our QR-313
program targeting dystrophic epidermolysis bullosa in both the U.S.
and Europe,” said David M. Rodman, MD, Chief Development Strategy
Officer of ProQR. “This represents another milestone for our
company and highlights the unmet need for patients with this
devastating disease. Our goal is to develop and actively advance a
pipeline of programs that can treat DEB mutations in a targeted
manner.”
About EMA Orphan Drug Designation (ODD)
In Europe, to qualify for orphan drug designation, a
medicine must be intended for the treatment, prevention or
diagnosis of a disease that
is life-threatening or chronically debilitating,
when the prevalence in the EU is not more than 5 in 10,000 (or
it is unlikely that marketing of the medicine would generate
sufficient returns to justify the investment needed for its
development), and when no satisfactory method of diagnosis,
prevention or treatment of the condition exist, or, if such method
exists, the medicine will be of significant benefit to
those affected by the condition. As incentives to encourage
the development of orphan medicines, the EU offers protocol
assistance specific for designated orphan medicines, 10 years
of market exclusivity once the medicine is on the market, and fee
reductions.
About QR-313
QR-313 is a first-in-class RNA-based oligonucleotide designed to
address the underlying cause of dystrophic epidermolysis bullosa
(DEB) due to mutations in exon 73 of the COL7A1 gene.
Mutations in this exon can cause loss of functional collagen type
VII (C7) protein. Absence of C7 results in the loss of anchoring
fibrils that normally link the dermal and epidermal layers of the
skin together. QR-313 is designed to exclude exon 73 from the mRNA
(exon skipping) and produce a functional C7 protein, thereby
restoring functionality of the anchoring fibrils.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the
creation of transformative RNA medicines for the treatment of
severe genetic rare diseases such as cystic fibrosis, Leber’s
congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based
on our unique proprietary RNA repair platform technologies we are
growing our pipeline with patients and loved ones in mind. *Since
2012*
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 QR-313 and
the clinical development and the therapeutic potential thereof,
statements regarding ODD and the potential benefits thereof, and
statements regarding our pipeline of programs targeting DEB. Our
actual results could differ materially from those anticipated in
these forward-looking statements for many reasons, including,
without limitation, risks associated with our clinical development
activities, including that we may not realize the intended benefits
afforded by orphan drug designation for our QR-313 program
targeting DEB, positive results observed in our prior and ongoing
studies may not be replicated in later trials or guarantee approval
of any product candidate by regulatory authorities, manufacturing
processes and facilities, regulatory oversight, product
commercialization, intellectual property claims, and the risks,
uncertainties and other factors in our filings made with the
Securities and Exchange Commission, including certain sections of
our annual report filed on Form 20-F. 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, except as required by
law.
ProQR Therapeutics N.V.:
Investor and Media Contact:Bonnie OrtegaT: +1
858 245 3983ir@proqr.com
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