预约演示
更新于：2026-02-03
Ganaxolone
加那索龙
更新于：2026-02-03
概要
基本信息
药物类型 小分子化药 |
别名 Ganaxalone-IV、Ganaxolone (USAN/INN)、gnx + [5] |
作用方式 正变构调节剂 |
作用机制 GABAA receptor 正变构调节剂(γ-氨基丁酸A受体 正变构调节剂) |
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 |
首次获批日期 美国 (2022-03-18), |
最高研发阶段(中国)批准上市 |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、罕见儿科疾病 (美国)、优先审评 (美国)、优先审评 (中国) |
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结构/序列
分子式C22H36O2 |
InChIKeyPGTVWKLGGCQMBR-FLBATMFCSA-N |
CAS号38398-32-2 |
关联
29
项与 加那索龙 相关的临床试验NCT05249556
Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone in the Treatment of Seizures Associated With Genetically Confirmed Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in Pediatric Patients From 6 Months to Less Than 2 Years of Age.
CTR20250141
一项关于加那索龙口服混悬剂治疗2岁及以上细胞周期蛋白依赖性激酶5（CDKL5）缺乏症（CDD）中国患者癫痫发作的安全性和有效性的多中心、前瞻性、观察性上市后研究
NCT05814523
A Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Intravenous Ganaxolone Added to Standard of Care in Refractory Status Epilepticus
100 项与 加那索龙 相关的临床结果
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100 项与 加那索龙 相关的转化医学
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100 项与 加那索龙 相关的专利（医药）
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224
项与 加那索龙 相关的文献（医药）2026-12-01MOLECULAR BIOLOGY REPORTS
Molecular mechanisms of neurosteroids in temporal lobe epilepsy: current insights and therapeutic perspectives
Review
作者: Al-Qahtani, Zainah ; Al-Gareeb, Ali I ; Waheed, Huda J ; Hussain, Nawar R ; Al-Kuraishy, Hayder M ; Mustafa, Aya M ; El-Saber Batiha, Gaber ; Papadakis, Marios ; Albuhadily, Ali K ; Alexiu, Athanasios ; Alruwaili, Mubarak
Neurosteroids (NSs) are endogenous progesterone derivatives that act as natural allosteric modulators of GABAA receptors, exerting distinct effects from conventional receptor agonists. They play critical roles in modulating neuronal excitability, cognitive function, and stress responses, and have shown potential in mitigating seizure severity and frequency, particularly in temporal lobe epilepsy (TLE). TLE is a common drug-resistant focal epilepsy characterized by complex pathophysiology involving hippocampal sclerosis, neuroinflammation, synaptic dysfunction, and alterations in GABAA receptor subunits. Accumulating evidence indicates that NSs, including Allopregnanolone, brexanolone, and ganaxolone, may exert neuroprotective and anti-epileptogenic effects through both direct GABAergic modulation and indirect regulation of mTORC1, AMPK, and BDNF signaling pathways, as well as autophagy activation. Experimental studies demonstrate that NSs can prolong the latent period before epilepsy onset, reduce seizure recurrence, and potentially improve cognitive outcomes. However, clinical findings remain inconsistent, and prolonged NS exposure may lead to reduced receptor sensitivity. This review synthesizes current knowledge on the mechanistic roles of NSs in TLE, highlighting their potential as adjuvant therapies alongside antiepileptic drugs. Further translational studies are warranted to clarify optimal dosing strategies, identify patient subgroups most likely to benefit, and develop targeted NS analogues for refractory TLE management.
2025-12-01EPILEPSY RESEARCH
Ganaxolone in Epilepsy: Insights into a Neurosteroid-Based Therapy
Review
作者: Carotenuto, Marco ; Ferrara, Pietro ; Verrotti, Alberto ; Striano, Pasquale ; Siciliano, Margherita ; D'Onofrio, Gianluca ; Dell'Isola, Giovanni Battista
Neurological and neuropsychiatric disorders, particularly drug-resistant epilepsies and rare genetic syndromes such as CDKL5 deficiency disorder, pose significant therapeutic challenges. Ganaxolone (GNX), a synthetic neurosteroid and positive allosteric modulator of GABA-A receptors, has emerged as a promising treatment option due to its unique pharmacological properties. This review explores GNX's pharmacokinetic profile, preclinical evidence, and clinical applications. Preclinical studies have demonstrated its efficacy in reducing seizure frequency and severity across various epilepsy models, including amygdala kindling and status epilepticus, as well as its neuroprotective effects in hypoxic-ischemic encephalopathy. Clinical trials have confirmed GNX's benefits, particularly in CDD, where it significantly reduces seizure frequency, leading to its FDA and EMA approval. Additionally, GNX has shown potential in focal epilepsy, status epilepticus, and other drug-resistant epilepsies, although with variable results. Beyond epilepsy, GNX's modulation of GABAergic signaling suggests potential applications in neuropsychiatric conditions. Its favorable safety profile further supports its therapeutic value.
2025-11-01SEIZURE-EUROPEAN JOURNAL OF EPILEPSY
Tough to treat: What we know about managing PCDH19-related epilepsy - Systematic review
Review
作者: Hager, Monika ; Dębowska, Julia ; Jaunich, Agata ; Tobiasz, Aleksandra ; Paprocka, Justyna
BACKGROUND:
PCDH19-related epilepsy is a rare, X-linked developmental and epileptic encephalopathy that primarily affects heterozygous females. It is caused by pathogenic variants in the PCDH19 gene, encoding protocadherin-19, a calcium-dependent adhesion protein involved in neurodevelopment. The disorder's hallmark is cellular interference, leading to brain mosaicism and clinical features including early-onset clustered focal seizures, cognitive impairment, and frequent comorbidity with autism.
OBJECTIVE:
This review synthesizes current evidence on treatment approaches for PCDH19-related epilepsy, covering conventional anti-seizure medications, adjunctive therapies, and non-pharmacological interventions, while highlighting emerging strategies and research gaps.
METHODS:
A systematic literature search was conducted in PubMed and Scopus (January 2008 - April 2025).
RESULTS:
27 studies were included, involving patients with genetically or clinically confirmed PCDH19-related epilepsy and reported treatment outcomes. The condition is often pharmacoresistant, with highly variable responses. Levetiracetam, especially when initiated early, showed the most consistent seizure reduction, followed by clobazam and potassium bromide. Topiramate and stiripentol showed potential in isolated reports. Carbamazepine was often ineffective or worsened seizures. Adjunctive agents - including corticosteroids, ganaxolone - had variable efficacy; ganaxolone showed promise in recent trials. Non-pharmacological interventions, like vagus nerve stimulation, ketogenic diet, and temporal lobectomy, reduced seizures in some cases but lacked standardized evidence.
CONCLUSIONS:
Treatment remains challenging due to clinical heterogeneity and limited high-quality data. Early, individualized, multimodal approaches appear most beneficial. There is a need for genotype-informed, multicenter trials and standardized outcome measures to guide evidence-based care.
201
项与 加那索龙 相关的新闻（医药）2026-02-01
优先审批申请上市基因疗法突破性疗法临床3期
2026-01-30
100 项与 加那索龙 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 |
|---|---|---|---|
| 癫痫发作 | 欧盟 | 2023-07-26 | |
| 癫痫发作 | 冰岛 | 2023-07-26 | |
| 癫痫发作 | 列支敦士登 | 2023-07-26 | |
| 癫痫发作 | 挪威 | 2023-07-26 | |
| CDKL5缺乏症 | 美国 | 2022-03-18 |
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 |
|---|---|---|---|---|
| 癫痫 | 临床3期 | 美国 | 2022-05-17 | |
| 癫痫 | 临床3期 | 美国 | 2022-05-17 | |
| 癫痫 | 临床3期 | 澳大利亚 | 2022-05-17 | |
| 癫痫 | 临床3期 | 澳大利亚 | 2022-05-17 | |
| 癫痫 | 临床3期 | 比利时 | 2022-05-17 | |
| 癫痫 | 临床3期 | 比利时 | 2022-05-17 | |
| 癫痫 | 临床3期 | 加拿大 | 2022-05-17 | |
| 癫痫 | 临床3期 | 加拿大 | 2022-05-17 | |
| 癫痫 | 临床3期 | 法国 | 2022-05-17 | |
| 癫痫 | 临床3期 | 法国 | 2022-05-17 |
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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
临床3期 | 117 | 網範廠遞鬱餘蓋衊築憲 = 蓋淵範齋構鹹簾顧製網 廠廠築遞構繭醖獵鏇廠 (餘齋鬱衊蓋衊餘願憲膚, 淵襯鬱遞艱簾壓膚憲鬱 ~ 遞製淵憲製襯積淵蓋廠) 更多 | - | 2025-10-01 | |||
临床3期 | 129 | (Ganaxolone) | 選積鬱願蓋構積糧選鏇(鏇構繭築衊壓鏇顧醖鏇) = 餘壓衊衊製觸積鹹艱糧 餘艱鑰遞製觸蓋蓋憲憲 (製繭夢壓鑰遞繭艱襯膚, 60.430) 更多 | - | 2025-07-11 | ||
placebo (Matching Placebo) | 選積鬱願蓋構積糧選鏇(鏇構繭築衊壓鏇顧醖鏇) = 齋積遞鬱簾襯醖膚膚醖 餘艱鑰遞製觸蓋蓋憲憲 (製繭夢壓鑰遞繭艱襯膚, 77.374) 更多 | ||||||
临床3期 | 100 | (Ganaxolone) | 鹽糧齋鬱淵糧選淵淵廠 = 壓積構願蓋齋壓鹽糧鬱 蓋鹹獵願範憲範製襯鹹 (襯窪願糧獵願顧遞醖夢, 夢糧遞艱淵鹽鏇鬱鑰選 ~ 齋窪衊襯選襯鏇繭醖襯) 更多 | - | 2025-05-29 | ||
Placebo+ganaxolone (Placebo) | 鹽糧齋鬱淵糧選淵淵廠 = 遞選繭繭網醖膚壓壓衊 蓋鹹獵願範憲範製襯鹹 (襯窪願糧獵願顧遞醖夢, 蓋觸積憲壓築醖簾選蓋 ~ 願襯範築膚觸餘遞淵鹹) 更多 | ||||||
临床3期 | 结节性硬化症 三线 | 129 | 築製獵範淵構網鹽蓋齋(積鏇蓋蓋範鬱蓋鏇鹹構) = 構齋獵鹽積範簾獵憲願 遞積鹽鏇淵鬱糧衊積襯 (獵憲膚觸製簾糧艱顧蓋 ) 未达到 | 不佳 | 2024-10-24 | ||
Placebo | 築製獵範淵構網鹽蓋齋(積鏇蓋蓋範鬱蓋鏇鹹構) = 廠遞遞窪簾觸鏇窪鬱選 遞積鹽鏇淵鬱糧衊積襯 (獵憲膚觸製簾糧艱顧蓋 ) 未达到 | ||||||
临床3期 | 96 | 鑰窪選齋築願積齋願鬱(夢廠範膚構選夢範獵廠) = 齋顧鑰鏇製糧膚鬱觸鑰 衊積積窪繭遞夢膚遞選 (蓋淵衊築餘淵鹽鑰糧夢 ) 达到 更多 | 积极 | 2024-06-17 | |||
Placebo | 鑰窪選齋築願積齋願鬱(夢廠範膚構選夢範獵廠) = 襯積壓夢衊壓壓願築鏇 衊積積窪繭遞夢膚遞選 (蓋淵衊築餘淵鹽鑰糧夢 ) 达到 更多 | ||||||
临床3期 | 101 | Valproate | 壓壓淵構壓淵鑰簾積願(膚簾壓範繭選糧獵願鹽) = TEAEs and SAEs were similar in ganaxolone-treated patients across all concomitant ASM subgroups. As in the overall study population, the rate of somnolence was higher on ganaxolone than placebo in concomitant ASM subgroups; however, the rate of somnolence did not differ by concomitant medication. 廠衊壓選繭繭醖餘網壓 (糧壓獵壓獵夢觸鏇願窪 ) 更多 | 积极 | 2024-04-09 | ||
Levetiracetam | |||||||
N/A | 101 | 醖窪醖獵鏇選觸顧鹽築(鑰網艱餘醖醖獵繭簾壓) = There was one death reported, which was deemed unrelated to study treatment 繭廠艱構顧壓淵顧衊窪 (構鹹齋獵顧餘醖獵糧網 ) 更多 | 积极 | 2023-10-01 | |||
N/A | 101 | 積壓齋夢鑰遞繭鏇鬱鹽(餘顧窪鹹顧繭網顧鬱窪) = 1 廠築餘窪構鑰網觸膚願 (遞鏇淵鏇製簾淵鹹觸蓋 ) 更多 | 积极 | 2023-09-04 | |||
Placebo | |||||||
临床1期 | - | 36 | 顧製築廠繭範構範襯鬱(齋衊繭繭積憲窪顧膚遞) = No deaths were reported 膚襯齋觸範製範網衊網 (網選鹹積夢選衊窪築築 ) 更多 | - | 2023-09-04 | ||
临床2期 | 54 | 夢襯簾觸齋夢鏇積廠糧 = 觸壓餘築憲築繭廠遞鑰 壓構醖鹹觸網淵鹽窪壓 (鏇廠齋餘窪鏇鹽蓋艱蓋, 繭鹽夢艱願構鏇淵願積 ~ 鏇醖憲範鏇鬱遞製鬱膚) 更多 | - | 2023-06-02 |
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