细胞周期蛋白依赖性激酶样5缺乏症相关癫痫治疗药物——加奈索酮

A drug for the treatment of seizures associated with CDKL5 defciency disorder:ganaxolone

加奈索酮被FDA批准其用于治疗与细胞周期蛋白依赖性激酶样5(CDKL5)缺乏症(CDD)相关的癫痫发作。其作用机制通过对突触和突触外γ-氨基丁酸(GABA)A受体的作用,发挥出抗癫痫活性。临床试验结果表明,与安慰剂相比,加奈索酮显著降低了CDD患者相关癫痫的发作频率,且该药物在患者中具有比较好的安全性和耐受性。本文对其药物代谢动力学、药物有效性、药物安全性及药物相互作用等内容进行总结,旨在为临床用药提供基本参考。

The Food and Drug Administration of the United States approved ganaxolone for the treatment of seizures asso?ciated with cyclin-dependent kinase-like 5(CDKL5)deficiency(CDD).Its mechanism of action is to exert anti-epileptic ac?tivity by acting on synaptic and extrasynapticγ-aminobutyric acid(GABA)A receptors.The results of the clinical trials have shown that ganaxolone significantly reduces the frequency of seizures in patients with CDD compared with placebo,more?over,the drug shows good safety and tolerance in patients.This review summarizes the effectiveness,safety,pharmacokinet?ics and drug interactions ganaxolone,in order to provide a basic reference for clinical medication.