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Drugs. 2018 Apr;78(6):707-714. doi: 10.1007/s40265-018-0905-7.
Burosumab (Crysvita; Kyowa Hakko Kirin Co., Ltd. and Ultragenyx Pharmaceutical Inc.) is a fully human monoclonal antibody directed at fibroblast growth factor 23 (FGF23). Excessive FGF23 production has been implicated in various hypophosphataemic diseases. Inhibition of FGF23 by burosumab results in increased renal phosphate reabsorption and increased serum levels of phosphorus and active vitamin D. In February 2018, the EMA granted subcutaneous burosumab conditional marketing authorization for the treatment of X-linked hypophosphataemia (XLH) with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons. In April 2018, the US FDA approved burosumab for the treatment of XLH in adults and children one year of age and older. Multinational phase III trials of burosumab are currently underway in adult and paediatric patients with XLH. Burosumab is also being evaluated in the phase II setting in adults with tumour-induced osteomalacia and epidermal nevus syndrome in the USA, as well as in Japan and Korea. This article summarizes the milestones in the development of burosumab leading to its first global approval in the EU for XLH in paediatric patients.
布罗索尤单抗（Crysvita；协和发酵麒麟株式会社和 Ultragenyx 制药公司）是一种针对成纤维细胞生长因子 23（FGF23）的全人源单克隆抗体。过量的 FGF23 产生与各种低磷血症疾病有关。布罗索尤单抗抑制 FGF23 可导致肾脏磷酸盐重吸收增加和血清磷和活性维生素 D 水平升高。2018 年 2 月，EMA 授予皮下注射布罗索尤单抗有条件上市许可，用于治疗 X 连锁低磷血症（XLH），有骨骼疾病的放射学证据的 1 岁及以上儿童和骨骼生长中的青少年。2018 年 4 月，美国 FDA 批准布罗索尤单抗用于治疗 1 岁及以上成人和儿童的 XLH。目前正在对 XLH 成人和儿科患者进行布罗索尤单抗的多中心 III 期试验。布罗索尤单抗也正在美国、日本和韩国的肿瘤诱导性骨软化症和表皮痣综合征成人中进行 II 期评估。本文总结了布罗索尤单抗的发展里程碑，最终该药在欧盟获得批准，用于治疗儿科患者的 XLH。
