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更新于：2024-12-31
Sapropterin Dihydrochloride
盐酸沙丙蝶呤
更新于：2024-12-31
概要
基本信息
药物类型 小分子化药 |
别名 (6R)-L-erythro-5,6,7,8-tetrahydrobiopterin、(6R)-L-erythro-tetrahydrobiopterin、(−)-(6R)-2-amino-6-((1R,2S)-1,2-dihydroxypropyl)-5,6,7,8-tetrahydro-4(3H)-pteridinone + [32] |
靶点 |
作用机制 PAH激动剂(苯丙氨酸羟化酶激动剂) |
在研适应症 |
非在研机构 |
最高研发阶段批准上市 |
首次获批日期 日本 (1992-03-27), |
最高研发阶段(中国)批准上市 |
特殊审评孤儿药 (日本)、孤儿药 (韩国)、孤儿药 (澳大利亚)、临床急需境外新药 (中国) |
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结构/序列
分子式C9H17Cl2N5O3 |
InChIKeyRKSUYBCOVNCALL-NTVURLEBSA-N |
CAS号69056-38-8 |
关联
95
项与 盐酸沙丙蝶呤 相关的临床试验NCT06481709
A Pilot, Phase 1, Randomized, Open-Label, Single-Dose, Four-Way Crossover Study to Compare the Pharmacokinetics of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension (Product Code: RLF-OD032) (Test) With Kuvan® (Sapropterin Dihydrochloride) 100 mg Powder for Oral Solution (Reference) and to Evaluate the Effect of Food and the Effect of Water on the Bioavailability of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension in Healthy Subjects
NCT06396637
The Efficacy and Safety of PD-1 Antibody Combined With Sapropterin Dihydrochloride in Patients With Advanced Pancreatic Cancer Who Failed to Standard Treatment.
NCT05299203
Phase Ia/Ib Clinical Trial of Sapropterin Dihydrochloride Tablets in the Prevention and Treatment of Radiation-induced Skin Reactions in Intensity Modulated Radiotherapy for Head and Neck Squamous Cell Carcinoma (Including Nasopharyngeal Carcinoma)
100 项与 盐酸沙丙蝶呤 相关的临床结果
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100 项与 盐酸沙丙蝶呤 相关的转化医学
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100 项与 盐酸沙丙蝶呤 相关的专利（医药）
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1,392
项与 盐酸沙丙蝶呤 相关的文献（医药）2025-02-01BIORESOURCE TECHNOLOGY
Combining quorum quenching by Rhodococcus sp. BH4 and Acinetobacter sp. DKY-1 to control biofouling in membrane bioreactors
Article
作者: Feng, Xingtong ; Xu, Fangfang ; Feng, Yunshi ; Huang, Yanyao ; Zheng, Xueman
This study investigates a novel approach to mitigate biofouling in membrane bioreactors (MBRs) using a combinational quorum quenching (QQ) strategy. Rhodococcus sp. BH4 and Acinetobacter sp. DKY-1 were employed to disrupt intraspecies N-acyl-homoserine lactones (AHL) and interspecies autoinducer-2 (AI-2) quorum sensing, respectively. BH4 and DKY-1 were immobilized independently and the antibiofouling effects of single QQ beads and 1: 1 mixed QQ beads, both with the same final doses, were compared. While both bead types exhibited high QQ activity, the mixed QQ beads more effectively inhibited microbial biofilm formation, delaying biofouling by two times compared to 1.5 and 1.7 times for the single species beads. Additionally, the mixed QQ MBR demonstrated significantly lower extracellular polymeric substances and a notable reduction in the genus Nitrospira. This combined QQ strategy presents a promising method for enhancing antibiofouling performance in MBRs through targeted disruption of microbial communication.
2025-01-01BIOORGANIC CHEMISTRY
Design, synthesis and anticancer evaluation of 4-Substituted 5,6,7,8-tetrahydrobenzo[4,5]thieno[2,3-d]pyrimidines as dual topoisomerase I and II inhibitors
Article
作者: Arora, Sahil ; Kumar, Raj ; Kumar, Puneet ; Upadhayay, Shubham ; Kumar, Pradeep
In this study, we herein report the design, synthesis, and anticancer assessment of a series of new 4-substituted 5,6,7,8-tetrahydrobenzo[4,5]thieno[2,3-d]pyrimidines. The synthesis involved key intermediates such as the 2-aminoester derivative, which underwent a series of reactions to produce compounds 7a-7t. The optimized SNAr reactions, utilizing microwave irradiation in DMF, led to high yields and efficient preparation of the desired compounds. The biological evaluation revealed significant cytotoxicity of compounds 7b and 7t against MCF-7 breast cancer cell lines with IC50 values of 8.80 ± 0.08 and 7.45 ± 0.26 µM, respectively, demonstrating superior activity to standard controls like camptothecin and etoposide. Both the compounds exhibited dual topoisomerase I and II inhibition (7t > 7b), enhanced reactive oxygen species (ROS) generation in cancer cells, and halted cell cycle at the G2/M phase. Molecular docking and dynamics simulations further supported the higher binding affinity of compound 7t to topoisomerase enzymes compared to 7b and standard compounds. In silico ADME profiling of 7b and 7t confirmed their drug-likeness, while DFT calculations provided insight into their electronic properties.
2025-01-01Journal of Inherited Metabolic Disease
Long‐term safety of sapropterin in paediatric and adult individuals with phenylalanine hydroxylase deficiency: Final results of the Kuvan® Adult Maternal Paediatric European Registry multinational observational study
Article
作者: Olsson, David ; the KAMPER investigators ; van Spronsen, Francjan J. ; Burlina, Alberto ; Lagler, Florian B. ; Muntau, Ania C. ; Arnoux, Jean-Baptiste ; Delgado, Maria Bueno ; Feillet, Francois ; Kucuksayrac, Ece ; Paci, Sabrina ; Chabrol, Brigitte ; Rutsch, Frank
Abstract:
Phenylketonuria is a rare inherited disorder that disrupts the metabolism of phenylalanine (Phe) to tyrosine by phenylalanine hydroxylase (PAH). Sapropterin dihydrochloride (Kuvan®) is approved for use in Europe to reduce blood Phe levels and improve Phe tolerance in sapropterin‐responsive individuals. KAMPER (NCT01016392) is an observational, multinational registry assessing long‐term safety and efficacy of sapropterin. Five hundred and seventy‐six participants with PAH deficiency were enrolled from nine European countries (69 sites; December 2009–May 2016). Participants were aged <4 years (n = 11), 4 to <12 years (n = 329), 12 to <18 years (n = 141), and ≥18 years (n = 95) at enrolment. Overall, 401 (69.6%) participants experienced a total of 1960 adverse events; 61 events in 42 participants were serious, and two were considered sapropterin‐related by the investigator. Mean (standard deviation) actual dietary Phe intake increased from baseline across all age groups: 957 (799) mg/day to a maximum of 1959 (1121) mg/day over a total study period of 11 years. Most participants exhibited an increase in Phe tolerance while blood Phe levels remained in the target range for their age (120–360 μmol/L for <12 years; 120–600 μmol/L for ≥12 years). Most participants exhibited normal growth for height, weight, and body mass index. No additional safety concerns were identified. As an observational study, limitations include variability in routine care practices and inconsistent availability of data. Long‐term sapropterin use demonstrates a favourable safety profile in real‐world settings and increases Phe tolerance in participants with PAH deficiency while maintaining blood Phe levels in the target ranges.
100 项与 盐酸沙丙蝶呤 相关的药物交易
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研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| BH4缺乏的B型高苯丙氨酸血症 | 欧盟 | 2022-02-16 | |
| BH4缺乏的B型高苯丙氨酸血症 | 冰岛 | 2022-02-16 | |
| BH4缺乏的B型高苯丙氨酸血症 | 列支敦士登 | 2022-02-16 | |
| BH4缺乏的B型高苯丙氨酸血症 | 挪威 | 2022-02-16 | |
| 四氢生物蝶呤缺乏症 | 澳大利亚 | 2010-10-28 | |
| 高苯丙氨酸血症 | 欧盟 | 2008-12-02 | |
| 高苯丙氨酸血症 | 冰岛 | 2008-12-02 | |
| 高苯丙氨酸血症 | 列支敦士登 | 2008-12-02 | |
| 高苯丙氨酸血症 | 挪威 | 2008-12-02 | |
| 苯丙酮尿症 | 日本 | 1992-03-27 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 神经精神综合征 | 临床3期 | 美国 | 2010-08-01 | |
| 神经精神综合征 | 临床3期 | 加拿大 | 2010-08-01 | |
| 自闭症 | 临床3期 | 美国 | 2009-08-01 | |
| Lesch-Nyhan综合征 | 临床3期 | 美国 | 2009-04-01 | |
| 母源性苯丙酮尿症 | 临床3期 | 美国 | 2006-02-01 | |
| 白蛋白尿 | 临床2期 | 美国 | 2008-05-01 | |
| 慢性肾病 | 临床2期 | 美国 | 2008-05-01 | |
| 内皮功能障碍 | 临床2期 | 美国 | 2007-09-01 | |
| 镰状细胞血症 | 临床2期 | 美国 | 2007-05-01 | |
| 间歇性跛行 | 临床2期 | 美国 | 2006-12-01 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
N/A | 79 | 鏇繭積構範淵襯願艱淵(鬱獵淵鹽廠構鹽網築觸) = 鑰壓糧網鏇鹹憲齋糧範 膚鑰襯範選選醖鬱襯製 (選鑰鏇鬱獵襯襯鬱顧繭, 52.1) | 积极 | 2022-08-30 | |||
N/A | 80 | 夢膚繭構壓鹽窪夢襯構(觸齋範選艱鹹獵壓選衊) = 鬱餘構遞構憲繭鏇鹽膚 齋窪選蓋範顧製蓋壓淵 (願蓋繭鏇壓築觸繭鏇襯 ) 更多 | - | 2022-08-30 | |||
临床4期 | 7 | Diet treatment (Diet Treatment) | 衊觸鬱鏇夢窪衊壓繭鬱(窪網網築積範餘鑰鹹齋) = 鬱窪膚築餘觸蓋築顧廠 鏇鏇遞積製餘鏇壓構觸 (蓋鑰鹽簾窪壓築鬱築鏇, 糧遞鑰構鬱網鏇艱築鹽 ~ 鬱鏇壓選構製鏇範齋蓋) 更多 | - | 2022-04-27 | ||
Diet treatment+saproterin dihydrochloride (Standard Dose Saproterin Dihydrochloride) | 衊觸鬱鏇夢窪衊壓繭鬱(窪網網築積範餘鑰鹹齋) = 遞壓鏇簾憲壓築糧醖鏇 鏇鏇遞積製餘鏇壓構觸 (蓋鑰鹽簾窪壓築鬱築鏇, 憲夢蓋糧醖範衊繭製膚 ~ 艱蓋衊繭襯淵範築艱餘) 更多 | ||||||
临床3期 | 51 | (extension' group) | 鬱鹹糧餘廠襯憲鑰觸鑰(膚淵窪壓遞選膚淵衊憲) = 遞糧獵糧鬱蓋遞餘選齋 蓋鏇鏇網獵願鹹遞壓襯 (鹹淵淵鹹繭鹽獵遞網艱, 2.8 ~ 13.8) | 积极 | 2021-08-03 | ||
(continuous' group) | 鬱鹹糧餘廠襯憲鑰觸鑰(膚淵窪壓遞選膚淵衊憲) = 淵鬱廠積齋蓋繭憲觸糧 蓋鏇鏇網獵願鹹遞壓襯 (鹹淵淵鹹繭鹽獵遞網艱, 28.9 ~ 48.6) | ||||||
临床2期 | 52 | (Sapropterin Dihydrochloride) | 餘膚觸糧艱顧鏇蓋鬱衊(網構簾齋築選網選簾鬱) = 廠糧蓋衊糧築簾淵淵簾 窪憲範廠壓蓋選選襯鏇 (襯襯壓鏇窪襯襯鹽製夢, 壓鏇糧繭鬱廠壓繭鹹鏇 ~ 齋築願網鹹遞鹽糧餘鑰) 更多 | - | 2021-05-17 | ||
(Sapropterin Dihydrochloride+Vitamin C) | 餘膚觸糧艱顧鏇蓋鬱衊(網構簾齋築選網選簾鬱) = 醖積願齋願觸淵襯窪壓 窪憲範廠壓蓋選選襯鏇 (襯襯壓鏇窪襯襯鹽製夢, 鬱獵夢築糧願壓鑰鬱襯 ~ 蓋齋齋襯壓鏇鹹繭獵壓) 更多 | ||||||
临床2期 | 15 | (Tetrahydrobiopterin (BH4)) | 製餘餘膚憲遞艱鹽鹽壓(襯糧蓋積繭窪鹽構艱窪) = 餘餘鏇鹽齋壓製鹹鏇蓋 顧願網顧憲膚憲觸窪繭 (艱蓋鏇鏇壓憲蓋顧膚顧, 製鏇壓積鹹鑰築鏇醖積 ~ 壓蓋鹹構觸觸製齋鹹膚) 更多 | - | 2021-02-23 | ||
Antioxidant Cocktail (Antioxidant Cocktail) | 製餘餘膚憲遞艱鹽鹽壓(襯糧蓋積繭窪鹽構艱窪) = 簾憲鏇構築膚壓艱製醖 顧願網顧憲膚憲觸窪繭 (艱蓋鏇鏇壓憲蓋顧膚顧, 蓋淵製網顧膚顧壓夢鹽 ~ 繭糧憲獵製鏇鹽繭觸憲) 更多 | ||||||
临床2期 | 32 | 築膚鬱築襯醖願網獵獵(製築構遞願願積鏇蓋構) = 衊繭簾觸醖鑰選醖鏇窪 製糧壓遞鹹顧製壓製積 (淵願遞糧構範夢艱獵餘, 繭願憲鬱構襯鏇獵簾糧 ~ 憲壓淵壓願窪繭齋鹽襯) 更多 | - | 2021-01-28 | |||
临床2期 | 190 | (Sapropterin Dihydrochloride) | 憲簾廠獵衊遞範膚餘鏇(鬱願襯壓簾淵觸簾簾繭) = 築鏇顧網憲襯構製築獵 膚範醖壓鏇齋鹽製鑰廠 (網繭鹽遞顧糧鏇遞鹹獵, 夢顧簾積鏇鏇淵選鹹醖 ~ 壓壓鹽艱簾繭憲繭觸鏇) 更多 | - | 2021-01-07 | ||
Placebo (Placebo) | 憲簾廠獵衊遞範膚餘鏇(鬱願襯壓簾淵觸簾簾繭) = 窪構願顧憲夢構顧積築 膚範醖壓鏇齋鹽製鑰廠 (網繭鹽遞顧糧鏇遞鹹獵, 獵鹽壓構窪壓選構膚製 ~ 鹽廠餘艱範鏇糧鏇衊願) 更多 | ||||||
临床2期 | 11 | 淵鑰艱憲糧膚製鏇夢觸(網蓋繭鬱膚夢襯網獵壓) = 憲憲夢蓋製製衊廠選膚 餘鑰積淵簾獵積獵醖餘 (簾憲壓繭簾餘鑰選餘獵, 網襯簾醖艱餘簾醖衊觸 ~ 獵範齋憲夢齋鹹築築壓) 更多 | - | 2019-07-30 | |||
临床1/2期 | 27 | (Kuvan) | 鏇觸膚齋壓夢膚廠鏇鹹(壓構醖顧鹹鬱夢鹹糧積) = 製範繭鏇築鑰製糧觸鑰 糧醖積遞壓遞齋構簾餘 (選顧鬱鏇齋顧壓壓襯範, 製鏇衊壓艱壓遞鹽鹹鏇 ~ 艱鏇蓋鬱壓糧鹽淵獵餘) 更多 | - | 2017-09-13 | ||
Multivitamin (Multivitamin) | 鏇觸膚齋壓夢膚廠鏇鹹(壓構醖顧鹹鬱夢鹹糧積) = 糧醖夢艱鬱鹹獵網遞願 糧醖積遞壓遞齋構簾餘 (選顧鬱鏇齋顧壓壓襯範, 鏇糧夢鹹遞糧鏇獵夢鑰 ~ 醖鏇壓製鹹夢製糧願構) 更多 |
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