Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics.
Sepofarsen, an RNA-based oligonucleotide, is designed to address the underlying cause of LCA10 in the CEP290 gene.
With the designation, the drug will receive priority review by the FDA, and it could qualify ProQR for a voucher redeemable for priority FDA review of a subsequent marketing application for a different product, the release said.
“This designation for sepofarsen underscores the significant unmet
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