- ProQR’s drug candidate, QR-313 for dystrophic epidermolysis bullosa (DEB) receives orphan drug designation in the EU from the EMA.
- QR-313 also received U.S. ODD from the
FDAin September 2017.
- QR-313 represents the fifth candidate in the company’s pipeline to receive ODD in the U.S. and EU.
- DEB is a severe genetic skin disease with no disease modifying treatments currently available.
- QR-313 targets the most common mutations within DEB, which are mutations in exon 73 of the COL7A1 gene and is designed for topical administration.
- QR-313 is expected to enter the clinic in 2018, with interim data 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
About EMA Orphan Drug Designation (ODD)
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.
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
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Source: ProQR Therapeutics N.V.