孤独症谱系障碍新药研发进展

Research and Development Progress of New Drugs for Autism Spectrum Disorder

目的了解孤独症谱系障碍(ASD)治疗新药的研发进展。方法检索药智网及ClinicalTrails中ASD治疗新药的相关文献。检索时间为2023年11月30日。总结全球处于临床研发阶段的,治疗ASD核心症状、共患症状及同时治疗两类症状新药的研发进展。结果针对ASD核心症状的新药主要有L1-79胶囊(酪氨酸羟化酶抑制剂)、STP-1胶囊(钠-钾-氯共转运体特异性抑制剂)、SB-121口服溶液(罗伊氏乳酸杆菌),研究分别处于Ⅱ期、Ⅰ期、Ⅰ期(已结束)临床试验阶段;针对共患症状的新药主要有AB-2004粉剂(4-乙基苯磺酸)、他司美琼胶囊/口服混悬液(褪黑素受体激动剂),研究分别处于Ⅱ、Ⅲ期临床试验阶段;而同时针对治疗两类症状的新药主要有CM-AT粉剂(胰酶浓缩粉末制剂)、ML-004片(选择性小分子5-羟色胺受体激动剂)、大麻二酚口服溶液(非精神类大麻素制剂)和JNJ-42165279片(高效能可口服的脂肪酸酰胺水解酶抑制剂),分别处于注册前申报,以及Ⅱ期、Ⅱ期、Ⅱ期临床试验阶段。结论ASD的治疗新药研发大多尚处于临床概念验证阶段,仍待通过大样本的Ⅲ期临床试验进行有效性和安全性验证。

Objective To understand the research and development(R&D)progress of new drugs for autism spectrum disorder(ASD).Methods Literature related to the new drugs for ASD in the Yaozh.com and ClinicalTrails were searched in November 30,2023.The R&D progress of new drugs worldwide in the clinical stage for ASD core symptoms,comorbid symptoms and both of symptoms was summarized.Results The new drugs for the ASD core symptoms mainly included L1-79 capsules(tyrosine hydroxylase inhibitors),STP-1 capsules(sodium-potassium-chloride cotransporter specific inhibitors)and SB-121 oral solution(Lactobacillus reuteri),which were in the clinical phasesⅡ,ⅠandⅠ(finished)trials,respectively.The new drugs for the ASD comorbid symptoms mainly included AB-2004 powder(4-ethylbenzenesulfonic acid)and tasimelteon capsules/oral suspensions(melatonin receptor agonist),which were in the clinical phasesⅡandⅢtrials,respectively.The new drugs for the ASD core and comorbid symptoms mainly included CM-AT powder(concentrated pancreatin powder),ML-004 tablets(selective small-molecule 5-hydroxytryptamine receptor agonist),cannabidiol oral solution(non-psychiatric cannabinoid preparation)and JNJ-42165279 tablets(high-efficiency and oral fatty acid amide hydrolase inhibitor),which were in the pre-registration application and clinical phasesⅡ,ⅡandⅡtrials,respectively.Conclusion The R&D of new drugs for ASD is still in the clinical verification stage,and their effectiveness and safety still need to be verified through large-scale clinical phaseⅢtrials.