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FDA Accepts Biologics License Application for Mucopolysaccharidosis II Treatment
NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced today that the U.S. Food and Drug Administration has accepted for review the Biologics License Application (BLA) submission by REGENXBIO Inc. (REGENXBIO; Headquarters: Rockville, Maryland, USA; CEO: Curran M. Simpson, NASDAQ: RGNX) for RGX-121 (clemidsogene lanparvovec), a potential first-in-class, investigational gene therapy for the treatment of Mucopolysaccharidosis II (MPS II). The FDA granted
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[{"type":"text","content":"PARAMUS, N.J., May 14, 2025 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced today that the U.S. Food and Drug Administration has accepted for review the Biologics License Application (BLA) submission by REGENXBIO Inc. (REGENXBIO; Headquarters: Rockville, Maryland, USA; CEO: Curran M. Simpson, NASDAQ: RGNX) for RGX-121 (clemidsogene lanparvovec), a potential first-in-class, investigational gene therapy for the treatment of Mucopolysaccharidosis II (MPS II). The FDA granted REGENXBIO's BLA Priority Review with a Prescription Drug User Fee Act ("PDUFA") target action date of November 9, 2025.","length":682,"tagName":"p"},{"type":"image","alt":"Logo for NS Pharma, Inc. (PRNewsfoto/NS Pharma, Inc.)","displaySize":"","headline":null,"caption":"Logo for NS Pharma, Inc. (PRNewsfoto/NS Pharma, Inc.)","className":"","disableSlideshowImg":false,"size":{"original":{"width":400,"height":67,"url":"https://media.zenfs.com/en/prnewswire.com/354d640585c26a59af279124b4668afd"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/Y8ytKgxFPFBHoMHMWa_Pbg--/YXBwaWQ9aGlnaGxhbmRlcjt3PTcwNTtoPTExODtjZj13ZWJw/https://media.zenfs.com/en/prnewswire.com/354d640585c26a59af279124b4668afd","width":400,"height":67}},"href":"https://mma.prnewswire.com/media/2377806/NS_Pharma_Inc__Logo.html","hrefExternal":true,"rel":"nofollow"},{"type":"text","content":"In January 2025, Nippon Shinyaku and REGENXBIO entered into a strategic partnership for the development and commercialization of RGX-121, as well as RGX-111, which is for the treatment of MPS I. Upon potential approval of RGX-121, NS Pharma will be exclusively responsible for commercializing RGX-121 in the U.S.","length":312,"tagName":"p"},{"type":"text","content":""This FDA decision represents a significant milestone in bringing a new, potentially life-changing treatment option to patients in the MPS community," said NS Pharma President, Yukiteru Sugiyama, Ph.D. "We are excited about our partnership with REGENXBIO and the value of our combined expertise in generating renewed hope for MPS families."","length":360,"tagName":"p"},{"type":"text","content":"For more details, please see the press release from REGENXBIO: https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-fda-acceptance-a...