ProQR Therapeutics N.V. has announced that the company’s investigational drug QRX-411 has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of retinitis pigmentosa, including Usher syndrome, the subtype targeted by QRX-411. Usher syndrome is the leading cause of combined deafness and blindness due to genetic defects in the Usher gene.
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.
Daniel A. de Boer, CEO of ProQR said “The severe genetic retinal diseases we are targeting do not have any available therapies, especially disease modifying therapies focused on restoring vision or impeding progression of the disease. We believe our novel RNA oligonucleotide approach has the potential to make a meaningful impact in the lives of Usher syndrome patients and others with rare genetic eye diseases.”
Chief Development Strategy Officer, David M. Rodman, MD, notes, “Orphan drug designation is an important step in rapidly bringing transformational precision medicines to patients with Usher syndrome and many other genetic causes of blindness in children and adults.”
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 the disease. Usher syndrome Type II is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.
QRX-411 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene. The mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional or absence of USH2A protein. QRX-411 is designed to restore wild-type USH2A mRNA leading to the production of wild-type USH2A protein by binding the mutated pre-mRNA causing normal splicing of the pre-mRNA.