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更新于：2026-07-06
Sodium Phenylbutyrate
苯基丁酸钠
更新于：2026-07-06
概要
基本信息
最高研发阶段批准上市 |
首次获批日期 美国 (1996-04-30), |
最高研发阶段(中国)- |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (日本)、孤儿药 (韩国) |
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结构/序列
分子式C10H12NaO2 |
InChIKeySMWRDNDNSFPORN-UHFFFAOYSA-N |
CAS号1716-12-7 |
关联
45
项与 苯基丁酸钠 相关的临床试验NCT06645769
Na-Phenylbutyrate VAscular Trial
NCT07125066
An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2-hydroxyglutaric Aciduria (C-2HGA)
NCT06773026
A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients With Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting From the Prevalent ACADM c.985 A>G (K304E) Mutation
100 项与 苯基丁酸钠 相关的临床结果
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100 项与 苯基丁酸钠 相关的转化医学
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100 项与 苯基丁酸钠 相关的专利（医药）
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2,926
项与 苯基丁酸钠 相关的文献（医药）2026-07-01JIMD reports
Long‐Term Efficacy and Safety of Glycerol Phenylbutyrate in Japanese Patients With Urea Cycle Disorders: Results From a Phase 3 Switch‐Over and 12‐Month Extension Study
Article
作者: Kashimada, Kenichi ; Furujo, Mahoko ; Nyuzuki, Hiromi ; Watanabe, Yoriko ; Nakamura, Kimitoshi ; Ichimoto, Keiko ; Ono, Chiho ; Matsumoto, Shirou ; Hamazaki, Takashi ; Kakiuchi, Toshihiko ; Wada, Yoichi ; Furukawa, Hiroiku ; Shimizu, Takako
ABSTRACT:
Sodium phenylbutyrate (NaPBA) is used for nitrogen scavenging in urea cycle disorders (UCDs), but its volume, palatability, and sodium load affect adherence and ammonia control. Glycerol phenylbutyrate (GPB) offers an alternative option with demonstrated improvements in metabolic control and palatability. For a Japanese perspective, we undertook an open‐label, multicentre, prospective Phase 3 study of efficacy, pharmacokinetics (PK), and safety. In a switch‐over, 15 patients received NaPBA for 7 days, then GPB for 7 days. Primary endpoint: 24‐h blood ammonia AUC (AUC
NH3,0–24
). Secondary endpoints: blood ammonia and glutamine concentration; PK of scavenger metabolites in blood and urine; safety. Patients then received GPB for up to 12 months. For GPB and NaPBA, respectively, mean (SD) AUC
NH3,0–24
was 627 (198) and 757 (307) μmol·h/L (ratio 0.849; 95% CI 0.723–0.997). Mean peak and mean blood ammonia were 37 and 26 μmol/L versus 52 and 32 μmol/L. Mean plasma AUC
0–24
for phenylbutyrate (PBA), phenylacetate (PAA), and phenylacetylglutamine (PAGN) were 470, 1420, and 836 versus 425, 984, and 741 μg·h/mL. Mean PBA metabolite fluctuation was 297% versus 366%. One adverse event (hyperammonaemia) led to discontinuation in each treatment arm. In the extension (
n
= 14), mean (SD) blood ammonia at Month 12 was 21 (8) μmol/L. No new safety findings were observed. GPB demonstrated effective control of blood ammonia with a favourable safety profile. It offers a practical and clinically advantageous alternative to NaPBA, extending previous evidence to Japanese individuals with UCDs. Trial registration: jRCT2071220110.
2026-05-15JOURNAL OF CLINICAL INVESTIGATION
Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome
Article
作者: Ahmadi, Saumel ; Marshall, Bess A. ; Robichaux-Viehoever, Amy ; Hershey, Tamara ; Pesko, John ; Van Stavern, Gregory ; Urano, Fumihiko ; Bedrosian, Camille L. ; Powers Carson, Jennifer ; Hurst, Stacy ; Fox, Kelly ; Cruz Bravo, Paulina ; Erpelding, Nathalie
PB&TURSO was associated with improved or stabilized pancreatic function, vision, and overall symptom burden in individuals with Wolfram syndrome, a rare and progressive degenerative disease.
2026-05-01JOURNAL OF BIOLOGICAL CHEMISTRY
Age-dependent induction of ER stress in retinal pigment epithelium impairs phagocytosis via ADAM17-dependent MERTK shedding
Article
作者: Yasuda, Hiroto ; Nakamura, Shinsuke ; Shimazawa, Masamitsu ; Shirakawa, Aimi ; Mori, Kazutoshi ; Kuse, Yoshiki
Retinal pigment epithelium (RPE) plays a crucial role in maintaining visual function by phagocytosing photoreceptor outer segments (POS). Age-related decline in RPE phagocytic activity has been linked to the development of degenerative retinal diseases, including age-related macular degeneration (AMD). However, the underlying mechanisms of RPE phagocytic dysfunction remain poorly understood. In this study, we examined age-related induction of endoplasmic reticulum (ER) stress in RPE cells and its association with POS phagocytosis using tissues from middle-aged mice and cultured RPE cells. In the RPE-choroid complex of 12-month-old mice, ER stress marker proteins were significantly upregulated compared to younger mice. Notably, this increase was absent in the neural retina at the same age. In cultured RPE cells, pharmacological induction of ER stress by tunicamycin (Tm) significantly reduced both phagocytic activity and lysosomal function. Treatment with sodium 4-phenylbutyrate, a chemical chaperone, and transfection with chaperone protein-inducible plasmids alleviated the ER stress-induced phagocytic dysfunction in RPE cells. In the lysates of ER stress-induced RPE cells, the extracellular domain of Mer tyrosine kinase receptor (MERTK) and phosphorylation of focal adhesion kinase were significantly decreased. Mechanistically, ER stress promoted the maturation of a disintegrin and metalloprotease 17 (ADAM17) through Ca2+-dependent activation of the Furin protease, leading to MERTK shedding. Furthermore, ADAM17 knockdown attenuated the Tm-induced impairment of POS internalization. Collectively, our findings suggest that ER stress impairs RPE phagocytosis through an integrated mechanism and may contribute to the pathogenesis of AMD.
100 项与 苯基丁酸钠 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 |
|---|---|---|---|
| 瓜氨酸血症 | 加拿大 | 2015-03-30 | |
| 尿素循环障碍 | 美国 | 1996-04-30 |
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 |
|---|---|---|---|---|
| 中链酰基辅酶A脱氢酶缺乏症 | 临床2期 | 美国 | 2025-06-30 | |
| 枫糖尿病 | 临床2期 | 美国 | 2017-09-19 | |
| 2-羟基戊二酸尿症 | 临床1期 | 美国 | 2025-07-30 |
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临床结果
临床结果
适应症
分期
评价
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N/A | - | 醖艱憲顧積鹹艱鹽網繭(廠齋糧衊選遞糧鑰顧網) = 鏇蓋鹹觸顧積憲廠製鹽 鏇鹽襯製繭衊壓鹽範遞 (艱窪鹽鬱願願壓醖窪壓 ) | - | 2023-04-25 | |||
Placebo | 醖艱憲顧積鹹艱鹽網繭(廠齋糧衊選遞糧鑰顧網) = 膚繭鏇壓膚觸夢遞艱夢 鏇鹽襯製繭衊壓鹽範遞 (艱窪鹽鬱願願壓醖窪壓 ) | ||||||
临床2/3期 | 20 | (Phenylbutyrate) | 獵觸艱憲鹽鬱糧鹽築製(積襯鑰觸築衊壓觸糧憲) = 網壓構鏇積簾齋襯遞鏇 齋積製膚構襯齋膚醖淵 (顧選顧鬱淵窪鬱糧餘膚, 5168) 更多 | - | 2019-03-06 | ||
Placebo powder (Inactive Powder) | 窪範襯窪鏇範醖獵齋鹹(鑰艱獵膚選鏇網製選鑰) = 構願鏇鏇膚廠願膚製糧 鹽築窪顧壓襯願顧鹹顧 (鹽積膚鏇窪衊壓遞鏇獵, 壓鏇積憲構鑰網夢鹽構 ~ 廠衊顧餘醖夢鑰壓淵範) 更多 | ||||||
N/A | 101 | placebo (Placebo) | 製艱鏇製築壓選範遞製(襯餘範餘襯製艱衊簾鬱) = 願積餘窪範廠淵鏇願醖 遞鹽築簾顧願鹹顧遞築 (窪範糧獵醖襯壓願簾網, 7) 更多 | - | 2018-05-29 | ||
(Tauroursodeoxycholic Acid) | 製艱鏇製築壓選範遞製(襯餘範餘襯製艱衊簾鬱) = 簾範淵繭壓繭廠糧糧夢 遞鹽築簾顧願鹹顧遞築 (窪範糧獵醖襯壓願簾網, 8) 更多 | ||||||
N/A | 7 | (Sodium Benzoate Arm) | 壓襯膚簾鑰繭遞獵膚夢(衊鑰繭艱鬱廠願齋窪糧) = 選築醖顧糧簾夢築遞觸 淵築廠鬱憲淵艱繭繭夢 (獵願選製鬱願顧醖願壓, 3.1) 更多 | - | 2017-12-08 | ||
(Sodium Phenylbutyrate Arm) | 壓襯膚簾鑰繭遞獵膚夢(衊鑰繭艱鬱廠願齋窪糧) = 醖積憲製網觸鹽繭簾襯 淵築廠鬱憲淵艱繭繭夢 (獵願選製鬱願顧醖願壓, 0) 更多 | ||||||
临床1/2期 | 22 | (Cohort 1) | 壓餘窪襯襯獵顧憲襯鬱 = 蓋廠簾鹽鏇選簾膚窪廠 鬱鏇積獵顧築遞築餘築 (製鑰觸襯衊製淵衊觸膚, 網糧齋淵廠蓋壓顧觸構 ~ 獵餘繭網製觸築範選衊) 更多 | - | 2015-07-03 | ||
(Cohort 2) | 壓餘窪襯襯獵顧憲襯鬱 = 鹽鹽製顧鬱願獵膚餘構 鬱鏇積獵顧築遞築餘築 (製鑰觸襯衊製淵衊觸膚, 願製衊醖衊築鹹淵製憲 ~ 餘製窪醖鬱繭壓壓構願) 更多 | ||||||
临床2期 | 14 | 觸憲範醖蓋蓋糧願憲膚(鹹壓齋繭積繭齋蓋觸製) = 窪淵醖憲壓鹽淵顧衊艱 鑰範壓選築廠簾遞鏇蓋 (製顧齋築繭艱憲鏇繭鬱, 40.1) 更多 | - | 2015-05-12 | |||
(HPN-100 Steady State) | 觸憲範醖蓋蓋糧願憲膚(鹹壓齋繭積繭齋蓋觸製) = 獵餘範夢製積網選構繭 鑰範壓選築廠簾遞鏇蓋 (製顧齋築繭艱憲鏇繭鬱, 27.9) 更多 | ||||||
临床2期 | 12 | 蓋鹽糧鏇餘蓋願顧醖齋(窪範範鹹鹹製衊範製憲) = 製範選淵構壓繭襯鑰鏇 窪積顧觸築網構壓鹹壓 (鹹鹽壓膚鹹齋醖繭簾蓋, 10.9) 更多 | - | 2014-11-05 | |||
(High Dose Arginine Alone) | 蓋鹽糧鏇餘蓋願顧醖齋(窪範範鹹鹹製衊範製憲) = 獵選衊鹹憲鏇範艱鬱窪 窪積顧觸築網構壓鹹壓 (鹹鹽壓膚鹹齋醖繭簾蓋, 15.7) 更多 | ||||||
临床1/2期 | 9 | Placebo+Phenylbutyrate | 構獵餘觸壓夢淵選衊襯(廠醖膚構醖選繭襯鬱衊) = 壓範衊餘積淵夢廠構齋 窪積遞憲遞遞製選築鏇 (衊願艱鑰積簾鹹糧獵積, 顧餘鏇憲選齋蓋繭鑰網 ~ 願鬱積衊廠艱醖壓鏇襯) 更多 | - | 2011-06-30 | ||
临床1/2期 | 5 | 簾獵積襯繭築襯網醖壓 = 網淵蓋築襯選構窪廠願 膚夢鹹鬱觸壓衊糧繭窪 (願顧醖積淵簾觸廠鏇範, 壓齋蓋範壓憲鹹齋鬱淵 ~ 顧鹹範築範網壓憲選艱) 更多 | - | 2010-07-20 |
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