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ProQR Receives Rare Pediatric Disease Designation from FDA for QR-421a

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Designation is for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene

ProQR Therapeutics N.V., a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, announced that it received Rare Pediatric Disease (RPD) designation from the United States (US) Food and Drug Administration (FDA) for QR-421a for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene.

QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene.

The RPD designation provides priority review by the FDA to encourage treatments for rare pediatric diseases. Under the RPD program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may qualify for a voucher that can be redeemed to receive a priority review by the FDA for any subsequent marketing application for a different product. Such a voucher is transferrable and may be sold.

“We are pleased to have received the second rare pediatric designation for our clinical stage development programs,” said Daniel A. de Boer, chief executive officer of ProQR. “Both sepofarsen for LCA10, the most common cause of blindness due to genetic disease in children, and QR-421a have received rare pediatric designation, underscoring the high unmet need for patients. We aim to make a difference for these patients as we work to advance our pipeline of programs to treat inherited retinal diseases.”

Read the full press release here.

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