ProQR Therapeutics announced that it received fast track designation 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.
Fast track designation is granted by the FDA to drugs in development for serious conditions with the potential to fulfill an unmet medical need. It was established with the intention to bring promising drugs to patients sooner by facilitating development with more frequent FDA interactions and expediting the review process.
“We are very pleased to receive fast track designation by the FDA for QR-1123. There are no current treatment options available for patients to improve vision or prevent vision loss due to adRP. Further, this designation emphasizes the high unmet need in this disease,” Daniel de Boer, Chief Executive Officer of ProQR, said in a company news release. “We look forward to beginning enrollment in the phase 1/2 (Aurora) clinical trial for QR-1123 in the coming months.”
QR-1123 is a first-in-class investigational antisense oligonucleotide that was discovered and developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. 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.