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.

本新药株式会社(Nippon Shinyaku) 的子公司 NS Pharma, Inc. (NS Pharma) 今天宣布，美国食品药品监督管理局 (FDA) 已接受 REGENXBIO Inc. (REGENXBIO；总部：美国马里兰州罗克维尔；首席执行官： Curran M. Simpson，纳斯达克股票代码：RGNX）的RGX-121（clemidsogene lanparvovec）疗法，是一款潜在的首创、在研基因疗法，用于治疗II型粘多糖贮积症（MPS II）。FDA授予REGENXBIO的生物制品许可申请（BLA）优先审查资格，处方药使用者付费法案（PDUFA）的目标审批日期为2025年11月9日。

, 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.

日本新药株式会社与REGENXBIO就RGX-121以及用于治疗MPS I的RGX-111的开发和商业化达成了战略合作伙伴关系。在RGX-121获得潜在批准后，NS Pharma将全权负责该药物在美国的商业化。

'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.'.

“这一FDA决定代表了为MPS群体患者带来一种新的、可能改变生命的治疗选择的重要里程碑，”NS Pharma总裁杉山幸辉博士表示。“我们对与REGENXBIO的合作感到兴奋，并相信双方专业能力的结合将为MPS家庭带来新的希望。”

About RGX-121 (clemidsogene lanparvovec)

关于RGX-121（clemidsogene lanparvovec）

RGX-121 is a potential one-time AAV therapeutic for the treatment of boys with MPS II, designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS). Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted iduronate-2-sulfatase (I2S) protein beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS.

RGX-121 是一种潜在的单次AAV基因疗法，用于治疗患有MPS II的男孩，旨在将艾杜糖醛酸-2-硫酸酯酶 (IDS) 基因递送到中枢神经系统 (CNS)。在中枢神经系统的细胞内递送IDS基因，可以提供一个永久的分泌型艾杜糖醛酸-2-硫酸酯酶 (I2S) 蛋白来源，超越血脑屏障，从而实现中枢神经系统细胞的长期交叉纠正。

RGX-121 expressed protein is structurally identical to normal I2S..

RGX-121表达的蛋白质在结构上与正常的I2S相同。

RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA.

RGX-121 已获得 FDA 授予的孤儿药、罕见儿科疾病、快速通道和再生医学高级疗法资格认定。

About MPS II

关于MPS II

MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme I2S leading to an accumulation of glycosaminoglycans (GAGs), including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the CNS.

MPS II，或称亨特综合征，是一种罕见的X连锁隐性遗传病，由溶酶体酶I2S缺乏引起，导致糖胺聚糖（GAGs），包括肝素硫酸（HS）在组织中积累，最终导致细胞、组织和器官功能障碍，包括中枢神经系统。

In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate heparan sulfate (HS) D2S6, which has been shown to correlate with neurocognitive manifestations of the disorder..

在该病的严重形式中，早期发育里程碑可能达到，但发育迟缓在18至24个月时已明显可见。针对MPS II神经学表现的具体治疗方法仍然是一个重要的未满足医疗需求。MPS II患者中I2S酶活性的关键生物标志物包括其底物肝素硫酸（HS）D2S6，已被证明与该疾病的神经认知表现相关。

About REGENXBIO Inc.

关于REGENXBIO公司

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. For more information, please visit .

REGENXBIO是一家领先的临床阶段生物技术公司，致力于通过基因治疗的治愈潜力改善生活。自2009年成立以来，REGENXBIO率先开发了AAV疗法，这是一类创新的基因治疗药物。欲了解更多信息，请访问。

www.regenxbio.com

www.regenxbio.com

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About NS Pharma, Inc.

关于NS Pharma公司

NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit

NS Pharma, Inc. 是 Nippon Shinyaku Co., Ltd. 的全资子公司。NS Pharma 是 Nippon Shinyaku Co., Ltd. 的注册商标。欲了解更多信息，请访问