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ProQR’s Usher syndrome treatment gets fast track designation

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The FDA has granted a fast track designation for QR-421a, an investigational treatment for vision loss associated with Usher syndrome type 2 and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene, ProQR announced in a press release.

“We are very pleased with the fast track designation the FDA granted us for QR-421a. Patients with Usher syndrome, the leading cause of combined deafness and blindness, currently have no available therapies for their vision loss and this designation emphasizes the high unmet need in this disease,” ProQR CEO Daniel de Boer said. “We are also looking forward to begin enrollment in the phase 1/2 STELLAR clinical trial in the coming months with preliminary data expected in mid-2019.”

QR-421a, an RNA-based oligonucleotide, previously received orphan drug designation in both the United States and the European Union.