ProQR Therapeutics N.V. (Nasdaq:PRQR)—a Leiden, Netherlands-based biotech company that develops drugs to treat genetic disorders like Usher syndrome—announced that investigational drug QRX-421 to treat Usher syndrome has received orphan drug designation (ODD) from the US 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, according to ProQR. 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.

ODD in the United States 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 United States and ten years in the European Union following market approval, according to the announcement.

“We are pleased to have ODD designation for both our programs targeting Usher syndrome in the United States and EU, representing yet another important milestone for our company,” said David M. Rodman, MD, chief development strategy officer at 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:

  • 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.

Source: ProQR Therapeutics 

Image: ProQR Therapeutics