BRIEF—RNA therapy for childhood blindness disease receives Rare Pediatric Disease designation

Netherlands-based RNA focused drug developer ProQR Therapeutics (Nasdaq:PRQR) has received Rare Pediatric Disease (RPD) designation from the US Food and Drug Administration (FDA) for sepofarsen for the treatment of Leber’s congenital amaurosis 10 (LCA10), the most common cause of blindness due to genetic disease in children.

Sepofarsen (QR-110) is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of LCA10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 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 of a subsequent marketing application for a different product.