预约演示
更新于：2025-05-31
Stiripentol
司替戊醇
更新于：2025-05-31
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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503
项与 司替戊醇 相关的文献（医药）2025-06-01Neurology and Therapy
Efficacy of Stiripentol Beyond Dravet Syndrome: A Retrospective Medical Record Review of Patients with Drug-Resistant Epilepsies
Article
作者: Duat-Rodríguez, Anna ; González-Alguacil, Elena ; García-Peñas, Juan José ; Ballarà-Petitbò, María ; Lamagrande-Casanova, Nuria ; Soto-Insuga, Víctor ; Cardenal-Muñoz, Elena ; Benítez-Provedo, Cristina
INTRODUCTION:
Stiripentol is approved as an add-on therapy with clobazam and/or valproate for seizures associated with Dravet syndrome (DS). Evidence of stiripentol efficacy in other drug-resistant epilepsies is limited.
METHODS:
This was a single-centre, retrospective, observational study of patients aged ≤ 15 years with non-Dravet epilepsy or DS who initiated stiripentol treatment in Spain.
RESULTS:
The study included 18 patients with DS and 17 with non-Dravet epilepsy; 76.5% of the latter had a developmental and epileptic encephalopathy. Median (range) age at stiripentol initiation was 52 (4-180) months. Three months of add-on stiripentol provided overall improvement in seizures (number, duration and/or intensity) for 76.5% of the non-Dravet and 61.1% of the DS patients (p = 0.30), all of whom were drug-resistant to prior antiseizure medications (ASMs). Stiripentol reduced seizure frequency by ≥ 50% in 58.8% of the non-Dravet patients and 44.4% of the DS cohort (p = 0.40); 20% of all patients became seizure-free. Stiripentol reduced all seizure types in both cohorts. Kaplan-Meier survival analysis found a higher probability of sustained stiripentol efficacy in the DS cohort (120 months) than the non-Dravet cohort (16 months; p = 0.012). Stiripentol improved cognition and Clinical Global Impression scale scores in approximately 60% of all patients; sleep improved for 19.2%. Acute stiripentol treatment (maximum dose 6.7-100 mg/kg/day) initiated in five patients (four with non-Dravet epilepsy and one with DS) during refractory status epilepticus (SE) successfully resolved SE over a median 0.5 days. Adverse events, mainly mild-to-moderate, occurred in 47.1% and 41.2% of patients in the non-Dravet and DS cohorts, respectively. Six patients (35.3%) with non-Dravet epilepsy discontinued ≥ 1 other ASMs after stiripentol initiation.
CONCLUSION:
Add-on stiripentol provides overall improvement in different seizure types and non-seizure manifestations for paediatric patients with drug-resistant epilepsy, including epileptic syndromes besides DS, and appeared effective in acute treatment of SE. Stiripentol was generally well tolerated.
2025-05-01EPILEPSIA
Pharmacokinetic variability and use of therapeutic drug monitoring of cannabidiol in patients with refractory epilepsy
Article
作者: Svendsen, Torleiv ; Burns, Margrete Larsen ; McQuade, Tao Angell‐Petersen ; Sætre, Johan ; Wolden, Martha ; Johannessen Landmark, Cecilie ; Sætre, Erik ; Øiestad, Elisabeth Leere ; Gottås, André ; Våtevik, Anne ; Johannessen, Svein I. ; Kjeldsen, Signe Flood
Abstract:
Objective:
Cannabidiol (CBD) (Epidyolex) is a new antiseizure medication (ASM) for rare and severe epileptic syndromes. We aimed to investigate the pharmacokinetic variability of CBD to elucidate relationships between doses, serum concentrations, metabolites, and biochemical markers of toxicity by using therapeutic drug monitoring (TDM) data.
Methods:
Data on serum concentrations of all ASMs, CBD, and the active metabolite 7‐hydroxy‐cannabidiol (7‐OH‐CBD) were collected (January 2022 to June 2023) at the Section for Clinical Pharmacology, National Centre for Epilepsy, Oslo University Hospital.
Results:
Data from 52 patients were included: 122 serum concentration measurements (1–7 per patient); 48% female, mean age 23 (range 3–55) years. At maintenance (n = 34), the mean daily dose was 535 (SD 224) mg, that is, 10.03 (standard deviation [SD] .49) mg/kg/day, serum concentration .26 (SD .14) for CBD and .13 (SD .10) μmol/L for 7‐OH‐CBD. Reference ranges of .15–.50 μmol/L for CBD and .04–.25 μmol/L for 7‐OH‐CBD are proposed, which included 80% of measurements. There was a linear correlation between CBD dose to concentration and CBD to CBD‐7‐OH concentrations (r2 = .39 and .38) (p < .05). The hepatic marker alanine aminotransferase (ALT) increased on average 37%, demonstrating a moderate effect on liver function. Intra‐individual coefficients of variation (CVs) were 32% (SD 17) for CBD and 48% (SD 24) for 7‐OH‐CBD (n = 15, ≥3 measurements). Twenty different ASMs were used: clobazam (n = 24), valproate (n = 17), and stiripentol (n = 8) were most common. The mean concentration ratio of desmethyl‐clobazam/clobazam increased by 140% (7.29–17.5; p < .05) but was variable, pointing to enzyme inhibition by CBD.
Significance:
This observational study with TDM data revealed extensive pharmacokinetic variability of CBD in patients with refractory epilepsy. The results demonstrate the need for close follow‐up and use of TDM, including biochemical markers of toxicity, for individualized treatment with CBD.
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.
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
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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
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N/A | 25 | 簾醖遞醖觸糧蓋獵醖製(鑰製艱鑰齋繭積選憲積) = 淵繭廠遞廠鏇襯窪觸膚 積鏇糧艱襯餘獵齋襯夢 (廠遞構餘構製選獵鬱構 ) 更多 | 积极 | 2024-10-21 | |||
N/A | - | - | 選築鏇衊夢願獵願夢膚(憲鏇蓋遞醖顧夢糧淵憲) = STP administration led to a median decrease in GTCS frequency of 84.4%, compared to 5.8% in the placebo group 醖遞選鏇鹽構顧襯觸夢 (鬱糧憲繭獵鏇糧製壓網 ) | 积极 | 2023-09-04 | ||
Placebo | |||||||
N/A | - | 22 | (Dravet syndrome) | 衊鏇鏇鹽膚鹹築鹽獵膚(襯範齋範鹽網淵糧鏇範) = 網構鹹鑰範夢構醖簾製 鑰蓋觸艱膚獵鬱憲壓艱 (夢鬱鬱淵鑰鑰觸夢糧構 ) | 不佳 | 2009-07-02 |
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