ProQR’s Drug Candidate QRX-421 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA
September 05 2017 - 7:00AM
Key Updates
ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that
investigational drug QRX-421 for Usher syndrome has received orphan
drug designation (ODD) from the U.S. Food and Drug Administration
(FDA) and European Medicines Agency (EMA). This marks the third
drug candidate in the company’s ophthalmology pipeline and the
fourth drug in the broader pipeline to receive ODD from the FDA and
EMA. QR-421 is a first-in-class investigational RNA-based
oligonucleotide designed to address the underlying cause of Usher
syndrome due to mutations in exon 13 of the USH2A gene. Usher
syndrome is the leading cause of combined deafness and
blindness.
ODD in the U.S. and European Union provides a special
status for investigational drugs being developed for rare
diseases. The ODD programs offer development program tax
benefits and a waiver of the NDA application user fee, as well as
market exclusivity for up to seven years in the U.S. and ten years
in the European Union following market approval.
“We are pleased to have ODD designation for both our programs
targeting Usher syndrome in the U.S. and EU, representing yet
another important milestone for our company and highlighting the
unmet need for patients in this disease,” said David M. Rodman, MD,
Chief Development Strategy Officer of ProQR. “At ProQR, we are
focused on designing accelerated development strategies that
capitalize on our oligonucleotide approach to potentially bring our
novel medicines to patients quicker and receiving ODD designations
for these is an important step towards this goal.”
ProQR’s ophthalmology pipeline includes the following:
- QR-110 for Leber’s congenital amaurosis 10 (LCA 10) due to the
p.Cys998X mutation, which received IND and CTA clearance and is in
clinical development (PQ-110-001 Phase 1/2 safety and efficacy
study). QR-110 was also granted Fast Track designation by the FDA
and ODD designation by the FDA and EMA.
- QRX-421 for Usher syndrome type 2 due to exon 13 mutations in
the USH2A gene, for which a clinical candidate has been selected
and is ready for IND enabling development studies.
- QRX-411 for Usher syndrome type 2 due to the PE-40 mutation in
the USH2A gene, for which a clinical candidate has been selected
and is ready for IND enabling development studies. QRX-411 also
received ODD designation by the FDA and EMA.
- QRX-1011 for Stargardt’s disease due to c.5461-10T>C
mutations in the ABCA4 gene, which is in optimization phase.
- QRX-504 for Fuchs endothelial corneal dystrophy (FECD), for
which a clinical candidate has been selected and is ready for IND
enabling development studies.
About QRX-421
QRX-421 is a first-in-class investigational RNA-based
oligonucleotide designed to address the underlying cause of Usher
syndrome due to mutations in exon 13 of the USH2A gene. Mutations
in this exon can cause loss of functional USH2A protein that causes
the disease. QRX-421 is designed to exclude exon 13 from the mRNA
(exon skipping) and produce truncated but functional USH2A protein,
thereby modifying the underlying disease.
About Usher Syndrome
Usher syndrome is the leading cause of combined deafness and
blindness. Patients with this syndrome generally progress to a
stage in which they have very limited central vision and moderate
to severe deafness. To date, there are no treatments approved or
products in clinical development that treat the vision loss
associated with Usher syndrome type 2. Usher syndrome type 2 is one
of the most common forms of Usher syndrome and is caused by
mutations in the USH2A gene.
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 QRX-421,
QRX-411, QRX-504, QRX-1011 and QR-110 and the clinical development
and therapeutic potential thereof, statements regarding orphan drug
designation, including the intended benefits of such status,
statements regarding our ongoing and planned discovery and
development of product candidates and the timing thereof, including
those in our opthalmology portfolio, and statements regarding our
oligonucleotide drug discovery platform. 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, 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.
ProQR Therapeutics N.V.:Investor
Contact:Bonnie OrtegaT: +1 858 245 3983ir@proqr.com
Media Contact:Sariette WitteT: +31 6 2970 4513
(NL)T: +1 213 261 8891 (US)pr@proqr.com
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