← Al het nieuws

ProQR Receives Fast Track Designation from FDA

ProQR Therapeutics, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that it received Fast Track designation from the Food and Drug Administration (FDA) for QR-1123.

QR-1123 is 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,” said Daniel de Boer, Chief Executive Officer of ProQR. “We look forward to beginning enrollment in the Phase 1/2 (Aurora) clinical trial for QR-1123 in the coming months.”

Source: ProQR