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JR-141治疗亨特综合征：已获日本MHLW孤儿药称号

2020-09-19 Allan MedSci原创

亨特综合征JR-141
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JCR Pharmaceuticals近日宣布，日本厚生劳动省（MHLW）已授予JR-141（Pabinafusp Alfa）治疗粘多糖贮积症II型（亨特综合症）孤儿药称号。

JCR Pharmaceuticals近日宣布，日本厚生劳动省（MHLW）已授予JR-141（Pabinafusp Alfa）治疗粘多糖贮积症II型（亨特综合症）孤儿药称号。JR-141是一种能够渗透血脑屏障（BBB）的重组异氰酸酯2-硫酸酯酶，用于治疗粘多糖贮积症II型（Hunter综合征）。JCR的专有BBB技术J-BrainCargo®已应用于该产品。JR-141已于2018年10月在美国和2019年2月在欧洲获得孤儿药称号，此外，JCR计划于2020年9月在日本提交JR-141的销售授权申请。

粘多糖贮积症II型（亨特综合征）是X连锁的隐性溶酶体贮积病（LSD），是由缺乏异丁二酸2-硫酸酯酶引起的，该酶可分解人体内的糖胺聚糖（粘多糖）。亨特综合征会引起广泛的症状。

由于JR-141（Pabinafusp Alfa）具有穿越血脑屏障（BBB）的能力，JR-141有望抑制中枢神经系统症状的发作或发展。在日本进行的III期临床试验中，所有参与者的脑脊液中粘多糖积累浓度均下降。关于安全性，未报告与JR-141相关的临床不良事件。

 

原始出处：

https://www.firstwordpharma.com/node/1758814?tsid=4

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    2020-09-21 chentianping

    #日本#

    0

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    2020-09-21 huiwelcome

    #孤儿药称号#

    0

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    2020-09-21 zhouqu_8

    #综合征#

    0

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    2020-09-20 1209e435m98(暂无昵称)

    学习了，谢谢分享

    0

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    2020-09-19 DXM123

    学习

    0

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