预约演示
更新于：2025-05-16
Stiripentol
司替戊醇
更新于：2025-05-16
概要
基本信息
药物类型 小分子化药 |
别名 1-(1,3-Benzodioxol-5-yl)-4,4-dimethyl-1-penten-3-ol、4,4-Dimethyl-1-((3,4-methylenedioxy)phenyl)-1-penten-3-ol、Diacomit Drysyrup + [8] |
作用方式 激动剂、抑制剂 |
作用机制 GABAA receptor激动剂(γ-氨基丁酸 A 受体激动剂)、L-lactate dehydrogenase抑制剂(L-乳酸脱氢酶抑制剂) |
非在研适应症- |
原研机构 |
非在研机构- |
最高研发阶段批准上市 |
最高研发阶段(中国)申请上市 |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、优先审评 (中国)、孤儿药 (日本) |
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结构/序列
分子式C14H18O3 |
InChIKeyIBLNKMRFIPWSOY-UHFFFAOYSA-N |
CAS号49763-96-4 |
关联
28
项与 司替戊醇 相关的临床试验CTR20250115
评价司替戊醇干混悬剂与氯巴占和丙戊酸联合使用治疗Dravet综合征的安全性和有效性的IV期临床研究
NCT06465472
Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3
CTIS2024-516655-40-00
- STP237
100 项与 司替戊醇 相关的临床结果
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100 项与 司替戊醇 相关的转化医学
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100 项与 司替戊醇 相关的专利（医药）
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504
项与 司替戊醇 相关的文献（医药）2025-04-01PHARMACOLOGY & THERAPEUTICS
From barbiturates to ganaxolone: The importance of chirality in drug development and in understanding the actions of old and new antiseizure medications
Review
作者: Perucca, Emilio ; Bialer, Meir
Out of 37 antiseizure medications (ASMs) currently in the market, 17 are chiral molecules and an additional one (oxcarbazepine) is a prodrug of the chiral compound licarbazepine. Of the 17 chiral ASMs, six (ethosuximide, fenfluramine, methsuximide, mephobarbital, stiripentol and vigabatrin) are marketed as racemates, and the remainder are licensed as enantiomerically pure medicines. Of note, all chiral ASMs introduced prior to 1990 were marketed as racemates. Stiripentol, fenfluramine and vigabatrin are the only racemic ASMs approved by the FDA >10 years after the release of regulatory guidelines on the development of chiral medicines. Despite the fact that pharmacokinetic and pharmacodynamic differences between enantiomers have been recognized for decades, the importance of chirality in understanding the biological actions of ASMs is not widely appreciated, and many recent publications on racemic ASMs refer to these medications as if they were a single molecular entity. In the present article, we provide a critical review of chiral ASMs developed between the 1920s, when mephobarbital was introduced, and 2022, when the last chiral ASM (ganaxolone) was approved. We summarize available data on stereoselective differences in pharmacokinetics and pharmacodynamics of ASMs marketed as racemates. We also discuss regulatory aspects related to the introduction of racemic medicines within the current regulatory scenario in Europe and the U.S., focusing on stiripentol, vigabatrin and fenfluramine as examples of different approaches. We identified a number of critical knowledge gaps that are relevant to the use of chiral drugs in epilepsy, including a remarkable lack of published information on the comparative pharmacokinetics, toxicity and antiseizure activity of the enantiomers of most racemic ASMs. The importance of chirality aspects in understanding the clinical actions of racemic ASMs is discussed, together with the rationale for the development of enantiomerically pure follow-up compounds with potentially improved efficacy, safety and commercial viability.
2025-03-17CHEMICAL RESEARCH IN TOXICOLOGY
Metabolic Activation of Stiripentol Correlates with Cytotoxicity
Article
作者: Jiang, Ziying ; Li, Weiwei ; Shen, Yan ; Wang, Yang ; Zheng, Jiang ; Zhao, Guode ; Luo, Xinyu ; Peng, Ying
Stiripentol (SRP) is an antiepileptic agent utilized in managing seizures related to Dravet syndrome. Long-term safety studies have highlighted significant adverse effects in patients including drowsiness, reduced appetite, ataxia, and elevated levels of serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST). The present study aimed at identifying the reactive metabolite of SRP and defining the potential correlation between its cytotoxicity and metabolic activation. Rat liver microsome incubation of SRP fortified with GSH as a trapping agent produced an α,β-unsaturated ketone metabolite (M1) and a related GSH conjugate (M2). Moreover, both the phase I metabolite and the GSH conjugate were detected in the bile of SRP-treated rats, indicating that both in vivo and in vitro metabolic activation of SRP took place. Notably, SRP exhibited significant cytotoxicity toward rat primary hepatocytes. Pretreatment with ketoconazole, a selective CYP3A enzyme inhibitor, mitigated the susceptibility of hepatocytes to SRP-induced cytotoxicity. These findings suggest that SRP may undergo metabolism to the α,β-unsaturated ketone metabolite, potentially contributing to the cytotoxic effects associated with SRP.
2025-02-01Neurology and Therapy
Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain
Review
作者: Sánchez-Carpintero, Rocío ; Villanueva, Vicente ; García-Ron, Adrián ; Calvo-Medina, Rocío ; Gil-Nagel, Antonio ; García-Peñas, Juan José
BACKGROUND:
Despite considerable evidence for the efficacy and safety of stiripentol in Dravet syndrome (DS), some aspects of stiripentol use remain challenging in clinical practice, such as dose titration and the adjustment of concomitant antiseizure medications (ASMs) to prevent potential adverse effects.
AIM:
To (1) provide practical recommendations on the initiation of stiripentol treatment in patients with DS, (2) evaluate its effectiveness in the patient, and (3) guide the management of drug interactions and other aspects of treatment monitoring.
METHODS:
Six Spanish neurologists (the authors) with expertise in the management of pediatric and adult patients with DS held a meeting in early 2024 to develop expert recommendations regarding the use of stiripentol in DS, based on a review of the literature and their common clinical experience.
RESULTS:
According to these recommendations, stiripentol can be administered to patients with DS of any age, although its initiation and titration vary according to age group. Individualized adjustment of concomitant ASMs, such as valproic acid and clobazam or drugs specifically for DS (i.e., fenfluramine), at initiation and during stiripentol treatment, can mitigate drug interactions, thereby increasing the long-term tolerability of stiripentol treatment. In specific cases, stiripentol doses of > 50 mg/kg/day may be contemplated, and acute stiripentol administration may be considered to control refractory status epilepticus. Blood tests should be performed before starting stiripentol, at 3, 6, and 12 months after starting treatment, and then annually, except in the event of adverse effects, when additional testing may be necessary. Most adverse effects can be adequately managed by adjusting concomitant medications.
CONCLUSION:
These practical recommendations may be easily adapted for use in different countries, and should increase physicians' confidence in the initiation and monitoring of stiripentol treatment, thus facilitating effective management of patients with DS and improving clinical outcomes.
100 项与 司替戊醇 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 |
|---|---|---|---|
| CDKL5缺乏症 | 美国 | 2018-08-20 | |
| 癫痫发作 | 美国 | 2018-08-20 | |
| Dravet综合征 | 欧盟 | 2007-01-03 | |
| Dravet综合征 | 冰岛 | 2007-01-03 | |
| Dravet综合征 | 列支敦士登 | 2007-01-03 | |
| Dravet综合征 | 挪威 | 2007-01-03 | |
| 强直阵挛性癫痫 | 欧盟 | 2007-01-03 | |
| 强直阵挛性癫痫 | 冰岛 | 2007-01-03 | |
| 强直阵挛性癫痫 | 列支敦士登 | 2007-01-03 | |
| 强直阵挛性癫痫 | 挪威 | 2007-01-03 |
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 |
|---|---|---|---|---|
| 原发性高草酸尿症1型 | 临床3期 | - | 2024-08-01 | |
| 原发性高草酸尿症2型 | 临床3期 | - | 2024-08-01 | |
| 原发性高草酸尿症III型 | 临床3期 | - | 2024-08-01 | |
| 原发性高草酸尿症 | 临床2期 | 法国 | 2019-05-21 | |
| 慢性肾病 | 临床1期 | 保加利亚 | 2023-07-24 | |
| 多形性胶质母细胞瘤 | 临床前 | 美国 | 2025-04-29 |
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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 | 25 | 鬱觸鹹鑰壓簾夢繭憲觸(醖顧獵遞獵憲繭糧遞鑰) = 積築壓淵夢醖窪壓襯憲 範製窪鹹襯鬱糧構淵築 (齋獵夢蓋夢蓋廠獵夢廠 ) 更多 | 积极 | 2024-10-21 | |||
N/A | - | - | 願願鑰鬱簾構顧鬱積觸(鏇簾顧構鑰窪範襯醖廠) = STP administration led to a median decrease in GTCS frequency of 84.4%, compared to 5.8% in the placebo group 齋餘選淵鹽鹹糧構壓願 (襯鏇淵鹽鑰淵構獵構壓 ) | 积极 | 2023-09-04 | ||
Placebo | |||||||
N/A | - | 22 | (Dravet syndrome) | 構齋獵積製鬱網繭糧淵(築齋壓鑰蓋壓願網襯鑰) = 範憲築淵餘鹽願夢選膚 鏇膚積鏇淵鹽鹹餘憲窪 (襯夢遞築夢夢鏇遞築鹹 ) | 不佳 | 2009-07-02 |
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