FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment

Core Insights - The U.S. FDA has accepted the Biologics License Application (BLA) for RGX-121, a gene therapy for Mucopolysaccharidosis II (MPS II), with a target action date of November 9, 2025 [1][5] - NS Pharma will exclusively commercialize RGX-121 in the U.S. upon potential approval, following a strategic partnership with REGENXBIO [2][3] Company Overview - NS Pharma, a subsidiary of Nippon Shinyaku Co., Ltd., focuses on developing and commercializing innovative therapies [8] - REGENXBIO is a clinical-stage biotechnology company specializing in gene therapy, particularly AAV Therapeutics [7] Product Details - RGX-121 is designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system, potentially providing a long-term solution for MPS II [4] - The therapy has received multiple FDA designations, including Orphan Drug Product and Rare Pediatric Disease [5] Disease Context - MPS II, also known as Hunter Syndrome, is a rare genetic disorder caused by a deficiency in the I2S enzyme, leading to significant health challenges, particularly neurological [6]