欧盟批准罕见的肌肉骨骼疾病药物Crysvita

2018-02-27 MedSci MedSci原创

医生现在可以利用Kyowa Kirin / Ultragenyx的Crysvita治疗在欧盟获得条件性批准的地区的患有罕见的慢性进行性肌肉骨骼疾病X连锁低磷酸盐血症的儿童。在儿童中,这种情况会引起骨骼疾病,导致下肢畸形和身高下降。传统的治疗方法包括每天多剂量的磷酸盐和活性维生素D,以抵消FGF23的过量作用,但不能纠正潜在的疾病。Crysvita(burosumab)是一种抗FGF23全人源单克隆

医生现在可以利用Kyowa Kirin / Ultragenyx的Crysvita治疗在欧盟获得条件性批准的地区的患有罕见的慢性进行性肌肉骨骼疾病X连锁低磷酸盐血症的儿童。在儿童中,这种情况会引起骨骼疾病,导致下肢畸形和身高下降。传统的治疗方法包括每天多剂量的磷酸盐和活性维生素D,以抵消FGF23的过量作用,但不能纠正潜在的疾病。Crysvita(burosumab)是一种抗FGF23全人源单克隆抗体,是第一种针对XLH潜在病理生理学的治疗方法。有条件的批准允许其用于治疗XLH,在一岁以上的儿童中有骨病的影像学证据,以及骨骼发育不良的青少年。"该药物的放行"给受XLH影响的欧洲人带来了希望,令人兴奋的是,欧洲是第一个全球监管机构批准的Crysvita。"Kyowa Kirin国际总裁兼首席执行官Tom Stratford说到。"我们现在将重点放在与卫生部门合作,确保欧洲国家的患者获得服务。"该公司还表示,它打算继续与医疗保健供应商和患者合作,以进一步了解受XLH影响的人群的真实体验。"这将有助于确保患者的识别和诊断,并改善这种罕见疾病的护理标准,"Stratford博士指出。原文出处:

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    2019-02-08 fyxzlh
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    2018-03-01 syscxl
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