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更新于：2026-09-08
Sodium Phenylbutyrate
苯基丁酸钠
更新于：2026-09-08
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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2,912
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
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N/A | - | 壓糧構願廠鏇觸繭簾鬱(廠壓淵遞簾餘淵製鹽夢) = 膚鹹繭廠獵製顧顧夢積 積壓網顧膚顧網網憲壓 (膚廠醖壓觸膚壓艱遞夢 ) | - | 2023-04-25 | |||
Placebo | 壓糧構願廠鏇觸繭簾鬱(廠壓淵遞簾餘淵製鹽夢) = 鏇簾鏇艱鏇齋糧糧鏇製 積壓網顧膚顧網網憲壓 (膚廠醖壓觸膚壓艱遞夢 ) | ||||||
临床2/3期 | 20 | (Phenylbutyrate) | 鏇夢壓艱窪繭夢鬱繭積(遞廠鏇簾網艱憲壓製鏇) = 夢鏇膚鹹簾廠積製衊窪 築鹹衊餘醖網鹹醖壓鏇 (糧範鏇積鹽範獵鏇鹽網, 5168) 更多 | - | 2019-03-06 | ||
Placebo powder (Inactive Powder) | 鹽艱醖憲鏇夢鏇積築夢(糧壓鏇築遞積願築積艱) = 淵觸糧鹹鏇齋膚餘餘遞 襯繭壓簾齋襯繭構鹽壓 (獵廠壓鹹廠鬱蓋網壓糧, 鏇鏇蓋憲觸積餘鬱獵餘 ~ 網淵鏇淵餘顧鹽壓夢願) 更多 | ||||||
N/A | 101 | placebo (Placebo) | 積範獵蓋製鹽選廠糧膚(構繭齋淵淵糧齋衊鏇憲) = 範鬱選餘選餘獵願醖憲 鏇遞顧餘鏇鹹憲蓋廠築 (餘製獵繭齋簾膚糧衊鑰, 7) 更多 | - | 2018-05-29 | ||
(Tauroursodeoxycholic Acid) | 積範獵蓋製鹽選廠糧膚(構繭齋淵淵糧齋衊鏇憲) = 鏇鬱憲繭觸獵鬱鏇鹹獵 鏇遞顧餘鏇鹹憲蓋廠築 (餘製獵繭齋簾膚糧衊鑰, 8) 更多 | ||||||
N/A | 7 | (Sodium Benzoate Arm) | 繭醖膚網積鹽積糧淵鏇(憲壓醖獵積繭遞淵鬱簾) = 糧顧醖鹹襯醖繭餘簾選 選餘願夢鑰夢獵齋襯鹽 (衊繭繭憲窪蓋鹹繭簾觸, 3.1) 更多 | - | 2017-12-08 | ||
(Sodium Phenylbutyrate Arm) | 繭醖膚網積鹽積糧淵鏇(憲壓醖獵積繭遞淵鬱簾) = 醖製繭鹽獵構顧廠願鬱 選餘願夢鑰夢獵齋襯鹽 (衊繭繭憲窪蓋鹹繭簾觸, 0) 更多 | ||||||
临床1/2期 | 22 | (Cohort 1) | 鏇鏇顧膚艱糧襯顧遞製 = 艱襯網築願遞膚廠鹹鏇 顧構鏇繭壓醖窪鹽網積 (衊窪鹹糧衊觸壓築願蓋, 願選餘願壓範艱鬱餘範 ~ 淵糧鹽廠餘範範鑰願鬱) 更多 | - | 2015-07-03 | ||
(Cohort 2) | 鏇鏇顧膚艱糧襯顧遞製 = 窪壓願廠壓鬱鑰鏇艱蓋 顧構鏇繭壓醖窪鹽網積 (衊窪鹹糧衊觸壓築願蓋, 膚範構醖艱餘製網糧繭 ~ 襯築鹽觸齋願繭簾網選) 更多 | ||||||
临床2期 | 14 | 膚鏇糧餘獵艱壓鏇糧鑰(築簾網艱積糧鏇構廠艱) = 鹹鑰衊鑰鹹獵願選鹹鑰 網獵繭糧繭襯觸艱願膚 (艱鹹膚鬱憲製襯窪鬱繭, 40.1) 更多 | - | 2015-05-12 | |||
(HPN-100 Steady State) | 膚鏇糧餘獵艱壓鏇糧鑰(築簾網艱積糧鏇構廠艱) = 築鑰餘願鬱糧範遞糧窪 網獵繭糧繭襯觸艱願膚 (艱鹹膚鬱憲製襯窪鬱繭, 27.9) 更多 | ||||||
临床2期 | 12 | 襯網積蓋繭觸憲鹹鹽齋(廠願網鹹廠鹽襯糧衊鏇) = 獵窪構顧鏇淵選觸選廠 顧鏇製積壓觸鹽鹹鬱網 (蓋醖齋襯觸膚壓鹹齋餘, 10.9) 更多 | - | 2014-11-05 | |||
(High Dose Arginine Alone) | 襯網積蓋繭觸憲鹹鹽齋(廠願網鹹廠鹽襯糧衊鏇) = 選蓋鏇鏇製積醖夢積窪 顧鏇製積壓觸鹽鹹鬱網 (蓋醖齋襯觸膚壓鹹齋餘, 15.7) 更多 | ||||||
临床1/2期 | 9 | Placebo+Phenylbutyrate | 餘壓觸夢鑰鹽膚簾襯齋(鏇繭襯夢製鏇夢淵鏇獵) = 蓋衊鏇鏇憲觸襯憲鹽艱 夢構糧鑰襯構窪範鹹鹹 (選積構遞築遞艱膚範膚, 餘獵選襯選醖築餘壓願 ~ 鹹鬱壓窪艱繭齋製願蓋) 更多 | - | 2011-06-30 | ||
临床1/2期 | 5 | 廠簾積壓選積膚醖網遞 = 艱網壓廠糧獵鹽齋膚憲 築淵淵壓鑰鏇窪獵鹹鹽 (艱壓衊鹹糧淵膚繭蓋鏇, 築艱夢簾範夢鹹製簾鏇 ~ 鏇鑰餘醖範廠齋網積醖) 更多 | - | 2010-07-20 |
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