Cerliponase Alfa: First Global Approval
Cerliponase Alfa: First Global Approval
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DOI：
10.1007/s40265-017-0771-8
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发表时间：
2017-07-01
期刊：
影响因子：
11.5
通讯作者：
Markham, Anthony
中科院分区：
文献类型：
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作者：
Markham, Anthony
Cerliponase alfa（Brineura（TM））是由BioMarin Pharmaceutical Inc.开发的重组人三肽基肽酶-1（TPP 1）。用于患有神经元蜡样质脂褐质沉积症2型（CLN 2）的患者，所述神经元蜡样质脂褐质沉积症2型是由TPP 1缺陷引起的儿科神经变性疾病。CLN 2的特征在于运动功能的进行性损害、语言缺陷、癫痫发作、共济失调、失明和早期死亡，并且脑室内输注α-肌钙蛋白酶已被证明可减缓功能下降的进展。本文总结了Cerliponase alfa开发过程中的里程碑，导致其在美国首次获得全球批准，用于治疗≥ 3岁CLN 2儿童患者的运动功能丧失，随后在欧盟获得批准用于所有年龄的CLN 2。
Cerliponase alfa (Brineura (TM)) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 (CLN2), a paediatric neurodegenerative disease caused by a deficiency in TPP1. CLN2 is characterised by progressive impairment of motor function, language deficiencies, seizures, ataxia, blindness and early death, and intracerebroventricular infusion of cerliponase alfa has been shown to reduce the progression of functional decline. This article summarizes the milestones in the development of cerliponase alfa leading to its first global approval in the USA for the treatment of motor function loss in paediatric patients >= 3 years of age with CLN2, and subsequent approval in the EU for CLN2 in all ages.