ProQR Receives Orphan Drug Designation from FDA for QR-1123 for Autosomal Dominant Retinitis Pigmentosa
ProQR Therapeutics announced that it received orphan drug designation (ODD) from the FDA for QR-1123, a first-in-class investigational antisense oligonucleotide designed to address the underlying cause of vision loss associated with autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO) gene.
ODD provides a special status for investigational drugs being developed for rare diseases. The ODD program offers development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to 7 years in the U.S. following market approval.
“We are pleased to have orphan drug designation for our QR-1123 program targeting autosomal dominant retinitis pigmentosa, or adRP,” Daniel de Boer, Chief Executive Officer of ProQR, said in a company news release. “It highlights the unmet need for patients with this progressive disease causing blindness. Our goal is to develop and actively advance a pipeline of programs that can treat inherited retinal diseases like adRP in a targeted manner.”
About QR-1123
QR-1123 is a first-in-class investigational RNA-based oligonucleotide designed to treat adRP due to the P23H mutation in the RHO gene. QR-1123 was discovered and developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutant mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye. QR-1123 was in-licensed from Ionis Pharmaceuticals in 2018, and subsequently received IND clearance in August 2019. QR-1123 has received Fast Track designation from the FDA.
About the Phase 1/2 Aurora trial
Aurora, or PQ-1123-001 trial, is a first-in-human study that will initially include up to 35 adults with adRP due to the P23H mutation in the rhodopsin (RHO) gene. The trial will include a single-dose escalation (open label) arm and a multiple-dose (double-masked) arm in which intravitreal injections of QR-1123 or sham procedure will be given in one eye. The objectives of the trial will include evaluation of safety, tolerability, pharmacokinetics and efficacy, as measured by restoration or improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity (BCVA), visual field (VF) and optical coherence tomography (OCT). The trial will be conducted at expert sites in North America and is expected to start in 2019.