Risdiplam: First Approval
Risdiplam: First Approval
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DOI：
10.1007/s40265-020-01410-z
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发表时间：
2020-10-12
期刊：
影响因子：
11.5
通讯作者：
Dhillon, Sohita
中科院分区：
文献类型：
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作者：
Dhillon, Sohita
Risdiplam（Evrysdi（TM））是一种口服给药的运动神经元2（SMN 2）-定向RNA剪接修饰剂，由Roche、PTC Therapeutics Inc和SMA Foundation开发，用于治疗脊髓性肌萎缩症。该小分子被设计用于治疗由染色体5 q突变导致SMN蛋白缺乏引起的脊髓性肌萎缩症。该药物增强了替代基因SMN 2产生全长和功能性SMN蛋白的能力。2020年8月，Evrysdi（TM）（risdiplam）在美国首次获得批准，用于治疗2个月及以上的脊髓性肌萎缩症患者。Risdiplam正在全球许多国家进行该适应症的预注册，包括欧盟、巴西、智利、中国、印度尼西亚、俄罗斯、韩国和台湾。本文总结了导致首次批准用于脊髓性肌萎缩症的risdiplam开发的里程碑。
Risdiplam (Evrysdi (TM)) is an orally administered, survival motor neuron 2 (SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The small molecule is designed to treat spinal muscular atrophy caused by mutations in chromosome 5q leading to SMN protein deficiency. The drug boosts the ability of an alternative geneSMN2to produce full-length and functional SMN protein. In August 2020, Evrysdi (TM) (risdiplam) received its first approval in the USA for the treatment of spinal muscular atrophy in patients 2 months of age and older. Risdiplam is in pre-registration for this indication in numerous countries worldwide, including the European Union, Brazil, Chile, China, Indonesia, Russia, South Korea and Taiwan. This article summarizes the milestones in the development of risdiplam leading to this first approval for spinal muscular atrophy.