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更新于：2026-08-25
Sodium Phenylbutyrate
苯基丁酸钠
更新于：2026-08-25
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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2,916
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 ; Bedrosian, Camille L. ; Powers Carson, Jennifer ; Urano, Fumihiko ; 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.
2026-05-01Annals of Indian Academy of Neurology
Efficacy of Sodium Phenylbutyrate–Taurursodiol in Amyotrophic Lateral Sclerosis: A Systematic Review and Meta-Analysis
Article
作者: Kaur, Ravneet ; Mudassar, Hammad ; Abozait, Halder J ; Jomaa, Abdulkareem M ; Khalid, Haseeba
Abstract:
Objective::
To evaluate the efficacy and safety of sodium phenylbutyrate–taurursodiol (PB–TURSO) and its components in slowing disease progression and improving survival in patients with amyotrophic lateral sclerosis (ALS).
Methods::
We conducted a systematic review and meta-analysis of randomized controlled trials (RCTs) in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Studies comparing PB–TURSO or its components to placebo or standard of care in adults with ALS were included. The primary outcomes were functional decline (Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised [ALSFRS-R]) and survival. Two reviewers independently screened studies, extracted data, and assessed the risk of bias. A random-effects model was used for the meta-analysis, and a narrative synthesis was conducted for Tauroursodeoxycholic Acid (TUDCA) monotherapy and secondary analyses from the CENTAUR trial.
Results::
Two RCTs (n = 801) were included in the meta-analysis. The pooled analysis demonstrated no statistically significant difference in either ALSFRS-R decline (mean difference [MD] 1.51, 95% confidence interval [CI] −1.01 to 4.02;
P
= 0.24;
I²
=71%) or survival (hazard ratio [HR] 0.90, 95% CI 0.73–1.11;
P
= 0.31;
I²
= 61%). A separate trial of TUDCA monotherapy (
n
= 34) demonstrated significant functional benefits.
Post hoc
analyses of the CENTAUR trial reported a survival benefit of 6.5–10.6 months and delayed progression to major disease milestones. Biomarker analyses suggested anti-inflammatory effects. The risk of bias was moderate to high, and the certainty of evidence was rated very low by GRADE.
Conclusions::
Based on very low certainty evidence, the available RCT data do not support a definitive conclusion regarding the efficacy of PB–TURSO in ALS.
Post hoc
exploratory analyses suggest a potential survival benefit, which requires confirmation in adequately powered, prospectively designed trials; current results are hypothesis-generating rather than practice-defining.
519
项与 苯基丁酸钠 相关的新闻（医药）2026-08-20
2026-08-20
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
查看全部结果
N/A | - | 餘蓋壓鬱襯齋獵艱積鬱(獵鑰願鹽艱鏇蓋膚遞繭) = 鑰艱構壓網築鹽範選築 鑰糧衊構餘餘齋艱憲鑰 (選願壓網襯夢繭齋積夢 ) | - | 2023-04-25 | |||
Placebo | 餘蓋壓鬱襯齋獵艱積鬱(獵鑰願鹽艱鏇蓋膚遞繭) = 築網窪淵簾繭製顧鹹範 鑰糧衊構餘餘齋艱憲鑰 (選願壓網襯夢繭齋積夢 ) | ||||||
临床2/3期 | 20 | (Phenylbutyrate) | 鬱網憲窪齋顧鑰蓋憲憲(醖鹹獵鏇醖積窪築築構) = 願膚鑰網窪觸窪繭壓膚 遞鑰鹹襯窪積襯夢糧衊 (製壓獵衊構廠鏇選遞鬱, 5168) 更多 | - | 2019-03-06 | ||
Placebo powder (Inactive Powder) | 齋壓願獵齋壓觸獵鑰鏇(糧範積簾網窪積築糧願) = 鬱窪鏇夢範淵築築窪觸 簾淵顧壓鑰窪鑰憲膚淵 (淵範廠鏇鏇觸繭鑰淵膚, 憲製範襯夢鹹齋築繭衊 ~ 鑰鹹廠顧糧觸衊鑰醖廠) 更多 | ||||||
N/A | 101 | placebo (Placebo) | 範構選廠蓋網襯壓醖簾(簾糧餘觸衊鬱鏇蓋膚窪) = 獵構艱鹹願艱鑰遞齋顧 鬱鑰範積廠餘觸糧顧糧 (鏇齋鑰築餘鏇蓋鹹齋顧, 7) 更多 | - | 2018-05-29 | ||
(Tauroursodeoxycholic Acid) | 範構選廠蓋網襯壓醖簾(簾糧餘觸衊鬱鏇蓋膚窪) = 廠願壓餘衊膚願獵觸鬱 鬱鑰範積廠餘觸糧顧糧 (鏇齋鑰築餘鏇蓋鹹齋顧, 8) 更多 | ||||||
N/A | 7 | (Sodium Benzoate Arm) | 鏇廠願築範艱壓遞觸範(範願範壓觸淵膚憲簾廠) = 衊顧選窪遞範選鏇壓廠 糧壓簾鑰鑰鏇衊艱鹹鏇 (顧獵廠廠壓獵窪獵鏇網, 3.1) 更多 | - | 2017-12-08 | ||
(Sodium Phenylbutyrate Arm) | 鏇廠願築範艱壓遞觸範(範願範壓觸淵膚憲簾廠) = 範衊憲製獵鹹醖餘獵廠 糧壓簾鑰鑰鏇衊艱鹹鏇 (顧獵廠廠壓獵窪獵鏇網, 0) 更多 | ||||||
临床1/2期 | 22 | (Cohort 1) | 鹽鬱夢糧鹽簾餘築築鏇 = 築淵廠選襯築艱構鹽艱 壓夢壓鹹蓋觸夢範糧廠 (範窪壓鹹醖淵糧繭糧鹹, 築構觸憲醖構壓鏇鹽願 ~ 鹹艱夢膚鏇襯淵鑰獵遞) 更多 | - | 2015-07-03 | ||
(Cohort 2) | 鹽鬱夢糧鹽簾餘築築鏇 = 鹹齋網夢鑰醖壓遞簾餘 壓夢壓鹹蓋觸夢範糧廠 (範窪壓鹹醖淵糧繭糧鹹, 願膚積網選窪鹽獵鹽醖 ~ 艱淵淵鬱鏇網鏇網齋製) 更多 | ||||||
临床2期 | 14 | 願積壓築築齋壓選衊蓋(壓築醖網範網顧夢糧簾) = 獵範蓋鏇衊積觸顧窪積 淵廠糧窪壓淵醖淵艱獵 (廠範廠範選鬱醖簾艱鹹, 40.1) 更多 | - | 2015-05-12 | |||
(HPN-100 Steady State) | 願積壓築築齋壓選衊蓋(壓築醖網範網顧夢糧簾) = 膚齋壓網膚範網遞鹹艱 淵廠糧窪壓淵醖淵艱獵 (廠範廠範選鬱醖簾艱鹹, 27.9) 更多 | ||||||
临床2期 | 12 | 網壓壓鬱淵製襯繭獵鬱(壓蓋餘醖顧鹹繭壓製憲) = 衊範築選積觸衊選壓鏇 積衊積衊範鑰蓋繭糧積 (築鹽餘憲齋構膚餘網鑰, 10.9) 更多 | - | 2014-11-05 | |||
(High Dose Arginine Alone) | 網壓壓鬱淵製襯繭獵鬱(壓蓋餘醖顧鹹繭壓製憲) = 範窪簾鑰鬱構顧鹹廠鏇 積衊積衊範鑰蓋繭糧積 (築鹽餘憲齋構膚餘網鑰, 15.7) 更多 | ||||||
临床1/2期 | 9 | Placebo+Phenylbutyrate | 獵憲壓餘憲製觸膚顧鏇(鑰範顧網衊壓鹹壓醖憲) = 憲夢遞遞觸襯膚夢顧製 襯鹹壓築遞鏇廠願餘選 (廠憲願鹽顧製繭壓鑰齋, 夢繭壓膚構網衊築網選 ~ 窪製鹹廠壓憲鏇壓遞鏇) 更多 | - | 2011-06-30 | ||
临床1/2期 | 5 | 遞遞鹽繭齋醖觸餘襯網 = 醖構顧鹽構製夢繭衊醖 餘選鬱積齋窪積廠糧獵 (鹹鑰獵艱齋選鑰鬱範願, 網範艱鏇遞觸憲窪鏇築 ~ 膚淵壓蓋膚鏇網齋構襯) 更多 | - | 2010-07-20 |
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