- ProQR Therapeutics N.V. (NASDAQ:PRQR) announces that investigational drug QRX-421 for Usher syndrome has received orphan drug designation (ODD) from the FDA and European Medicines Agency (EMA), representing the third drug candidate in the company’s ophthalmology pipeline and the fourth in the broader pipeline to receive ODD from the FDA and EMA.
- QR-421 is an 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.
- Now read: Progenics's Azedra: What's Wrong With This Picture?
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