Nippon Shinyaku Co. Ltd. has announced a significant development in the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. The U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application for RGX-121 (clemidsogene lanparvovec), an investigational gene therapy developed by REGENXBIO. This potential first-in-class therapy aims to address the underlying genetic cause of MPS II by introducing the IDS gene to inhibit long-term disease progression. Nippon Shinyaku, through its subsidiary NS Pharma, Inc., will hold exclusive commercialization rights in the U.S. and Asia, including Japan, upon approval. The BLA submission is supported by promising results from the Phase I/II/III CAMPSIITE trial. Nippon Shinyaku continues to focus on rare disorders, aligning with its mission to help people lead healthier, happier lives.