亨特综合征新药「艾度硫酸酯酶β注射液」在中国获批

2020-09-04 医药魔方 医药魔方

9月3日,诺爱药业/北海康成在中国提交的艾度硫酸酯酶β注射液上市申请获得国家药监局批准,用于治疗亨特综合征。

9月3日,诺爱药业/北海康成在中国提交的艾度硫酸酯酶β注射液上市申请获得国家药监局批准,用于治疗亨特综合征。

亨特综合征又称II型黏多糖贮积症,是一种罕见的、可致残的、并且致命的遗传性疾病,该疾病由2-硫酸酯酶缺乏引起,这是分解体内糖胺聚糖(GAGs)所必需的一种酶。2-硫酸酯酶缺乏会导致患者GAG累积并引发严重的骨骼、组织、神经和多器官并发症,并最终导致死亡。

亨特综合征在东亚较为高发,目前尚不能治愈,标准疗法为酶替代治疗或姑息疗法。艾度硫酸酯酶β(英文商品名:Hunterase)是重组人艾杜糖醛酸-2-硫酸酯酶(IDS)酶替代疗法,由GC Pharma开发,北海康成拥有该药中国权益。

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    2020-09-05 1381abdab4m

    #罕见病##亨特综合征#,即#II型黏多糖贮积症#药物艾度硫酸酯酶β注射液上市,祝贺!

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