北海康成-B(01228.HK):注射用維拉苷酶β(戈芮寧,CAN103)在中國獲批上市
格隆匯5月20日丨北海康成-B(01228.HK)公佈,注射用維拉苷酶β(戈芮寧,CAN103)已於2025年5月15日獲中國國家藥品監督管理局授予上市批準,用於治療I型及III型戈謝病。公司持有開發及商業化產品的全球專有權利。
戈謝病是一種最常見的溶酶體貯積症之一,爲位於1q22號染色體長臂上的葡萄糖腦苷脂酶基因突變引起的常染色體隱性遺傳病。該病對男性和女性的影響是同等的。臨牀上戈謝病包括0型(圍產期致死型)、I型(慢性非神經病變型)、II型(急性神經病變型)和III型(慢性神經病變型),其中I型和III型戈謝病患者多能活到成年。戈謝病是由葡萄糖腦苷脂酶(酸性b-葡萄糖苷酶)缺乏引起的,這種酶有助於分解溶酶體內一種被稱作葡萄糖腦苷脂(葡糖神經醯胺)的細胞膜鞘脂。葡萄糖腦苷脂主要積聚在某些器官內的單核巨噬細胞系細胞中(Gaucher細胞),導致脾腫大,肝腫大,貧血,血小板減少症,骨痛和骨折,嚴重的戈謝病類型(圍產期致死型,II型和III型),早期可出現神經系統症狀。
戈芮寧是國內首個自主研發適用於12歲及以上青少年和成人I型及III型戈謝病患者的長期酶替代療法。作爲一類創新藥,其可完全替代同類進口產品,將大大提升國內患者用藥的可及性。
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