治疗CKLD5缺乏症相关癫痫发作药物:加奈索酮

Drug for treating CKLD5 deficiency related epileptic seizures:ganaxolone

加奈索酮(ganaxolone)是美国Marinas制药公司研发的一种口服γ-氨基丁酸A型(GABAA)受体正向变构调节剂,能特异性地调节中枢神经系统中的GABAA受体,加强时相性和紧张性抑制,以控制癫痫发作。2022年3月美国食品和药物管理局基于一项重要的Ⅲ期临床试验结果,批准其作为首个用于治疗细胞周期蛋白依赖性蛋白激酶5缺乏症的药物。临床试验表明,加奈索酮耐受性良好,以该药为基础的治疗方案可有效控制患者癫痫发作频率,提高生活质量。

Ganaxolone is an oral GABAA receptor positive variant modulator developed by Marinas Pharmaceuticals in the United States,and it can specifically modulate GABAA receptors in the central nervous system to enhance temporal and tonic inhibition for seizure control.In March 2022,the U.S.Food and Drug Administration approved it as the first drug to treat cyclin-dependent kinase-like 5 deficiency based on the results of a major phaseⅢclinical trial.Clinical trials have shown that ganaxolone is well tolerated,and the treatment regimen based on this drug is effective in controlling seizure frequency of patients and improving their quality of life.