首页|尿酸盐转运蛋白-1抑制剂治疗痛风临床研发审评考虑

尿酸盐转运蛋白-1抑制剂治疗痛风临床研发审评考虑

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痛风是一种单钠尿酸盐(monosodium urate,MSU)沉积在关节所致的晶体相关性关节病,其与嘌呤代谢紊乱和/或尿酸排泄减少所致的高尿酸血症直接相关.随着人们物质生活水平的不断提高以及越来越多的含嘌呤类食物的摄入,痛风的患病率呈逐年上升的趋势.目前国内常用的降尿酸药物包括抑制尿酸合成(别嘌醇和非布司他)和促进尿酸排泄(苯溴马隆)2 类,虽然现有的药物可以减轻痛风发作和痛风的负担,但其仍然有一定的局限性,需要研发新的有效、安全痛风治疗药物.目前以尿酸盐转运蛋白-1(urate transporter 1,URAT1)为靶向的药物设计是抗痛风药物研发的热点.本文综述了URAT1 抑制剂的临床应用现状以及URAT1 品种临床试验申报日益增多的情况,结合审评中遇到的相关问题,阐述目前URAT1 抑制剂药物治疗痛风临床试验的一般性设计与审评考虑,期望为企业研发人员在痛风临床试验设计方面提供参考,从而提高研发效率,使患者早日获益.
Evaluation consideration for clinical research and development of URAT1 inhibitors for gout
Gout is a crystal-related arthropathy caused by the deposition of monosodium urate(MSU)in the joints,which is directly related to hyperuricemia caused by purine metabolism disorders and/or reduced uric acid excretion.With the continuous improvement of people's material living standard and the intake of more and more purine-containing foods,the prevalence of gout is increasing year by year.Currently,commonly used urate-lowering drugs in China include two categories:drugs inhibiting uric acid synthesis(allopurinol and febuxostat)and that promoting uric acid excretion(benbromarone).Although existing drugs can reduce gout attacks and the burden of gout,it has certain limitations and new effective and safe gout treatment drugs need to be developed.Currently,URAT1-targeted drug design is a hot topic in the research and development of anti-gout drugs.This paper reviewed the current status of clinical application of URAT1 inhibitors.Regarding the increasing number of applications for clinical trials of URAT1 varieties,combined with the relevant problems encountered in the evaluation,we also discussed the design and evaluation consideration for clinical trials of URAT1 inhibitors,so as to improve the research and development and provide references for researchers.

gouturate transporter 1 inhibitorclinical research and developmentevaluation consideration

色日格楞、艾星、赵伯媛、李远红、赵田、张杰

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国家药品监督管理局药品审评中心,北京 100076

痛风 尿酸盐转运蛋白-1抑制剂 临床研发 审评考虑

2024

中国新药杂志
中国医药科技出版社 中国医药集团总公司 中国药学会

中国新药杂志

CSTPCD北大核心
影响因子:1.039
ISSN:1003-3734
年,卷(期):2024.33(8)
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