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STAT+: Ionis wins FDA approval for first drug for Alexander disease, a rare and deadly neurological disorder
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关键摘要
Ionis获FDA批准首款Alexander病治疗药Zanvastro，显著延缓运动功能衰退
- Zanvastro是全球首个获批的Alexander病疾病修饰疗法
- 临床试验显示用药组步行速度稳定，对照组下降33%
- 药物在儿童中或可改善而非仅稳定运动功能
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正文提要
Ionis Pharmaceuticals on Thursday received Food and Drug Administration approval for the first disease modifying therapy for Alexander disease, a rare and deadly neurological condition.
The FDA approved the drug, Zanvastro, for use in children and adults after a pivotal trial showed that walking speed, a common measure of motor function in neurologic disease, stayed stable in treated patients while those in the control group saw a 33% decline.
The study also contained hints that treating young children can improve motor function, not just stabilize it. Researchers found that the drug was generally safe, with serious adverse events more common in the control group than among those treated.