2月28日，GW制药宣布美国FDA授予该公司用于治疗儿童Lennox-Gastaut综合征(LGS)的药物Epidiolex孤儿资格，LGS是一种罕见的严重形式的儿童发作性癫痫，这款药物以植物提取成分大麻二酚(CBD)为主要活性成分。Epidiolex是一种口服的、高纯度CBD提取物液体制剂，CBD是一种来自大麻植物的非精神类成分。
2013年11月，GW宣布FDA授予Epidiolex用于治疗Dravet综合症孤儿药资格。今年1月份，GW打算推进Epidiolex用于LGS和Dravet综合症的一项临床开发项目。今年下半年，GW打算与美国主要儿科癫痫专家一起努力开始一项Epidiolex的初步2期临床试验。这项试验如果获得成功，该公司预期会开展这款药物用于LGS和Dravet综合症的3期关键临床试验，以支持向FDA提交新药申请。
LGS是一种严重形式的儿童发作性癫痫。癫痫发作类型因患者而异，包括强直性发作、失张力发作、非典型失神发作和肌阵挛发作。大多数LGS儿童会经历某些程度的智力功能或信息处理功能的损伤，以及发育迟缓和行为障碍。LGS可由大脑畸形、围产期窒息、严重头部外伤、中枢神经系统感染和遗传退行性或代谢症状引起。据估计，美国大约有1.4万-1.85万LGS患者，欧洲约有2.3万-3.1万患者。
“这次在Epidiolex用于LGS被授予孤儿药资格不久之前，其用于Dravet综合征也获得FDA孤儿药资格。我们现在正积极与FDA讨论有关Epidiolex的监管途径，我们相信这款药物有潜力满足明显未满足的儿童严重癫痫发作的需要，这一领域所有用来控制这些发作的选择已经用完，” GW制药董事长Justin Gover阐述说。GW正在以提供一种FDA批准的药物为目标来满足这一需求。
New Drugs Online Report for cannabidiol
Information
Generic Name: cannabidiol
Trade Name: Epidiolex
Synonym: GW42003
Entry Type: New molecular entity
Development and Regulatory status
UK: None
EU: None
US: Phase III Clinical Trials
UK launch Plans: Available only to registered users
Actual UK launch date:
Comments
Oct 14: Granted orphan drug status in the EU for treatment of Dravet syndrome [5].
27/10/2014 10:34:45
Jun 14: GW anticipates starting anaother PIII trial in Dravet syndrome in 1Q 2015. GW expects to hold a pre-IND meeting with the FDA for Epidiolex in the treatment of LGS in mid-2014, and expects to conduct two PIII trials in LGS during 2015 [1].
18/06/2014 13:06:09
Jun 14: Epidiolex has been granted fast-track status and orphan drug status in the US [1].
18/06/2014 13:03:59
Jun 14: GW Pharmaceuticals plan to start a Phase 2/3 clinical trial of Epidiolex in Dravet syndrome in 2H 2014 [1].
18/06/2014 12:56:21
Jun 14: Pre-clinical for Dravet syndrome and Lennox-Gastaut syndrome in the UK [2].
18/06/2014 12:51:16
Trial or other data
Mar 16: Results of PIII study (NCT02091375) of cannabidiol (CBD) for the treatment of Dravet syndrome, a resistant form of epilepsy announced. Patients (n=120) were randomised to either CBD 20mg/kg/day (n=61) or placebo (n=59). The drug was added to existing treatment of an average of 3 other antiepileptic drugs (AEDs), patients having previously tried and failed an average of more than 4 other AEDs. The average age of trial participants was 10 years and 30% of patients were aged below 6 years. The median baseline convulsive seizure frequency per month was 13. Primary efficacy endpoint was the percentage change in the monthly frequency of convulsive seizures during the 14-week treatment period compared with the 4-week baseline observation period. Median reduction in monthly convulsive seizures on CBD was 39% compared 13 percent reduction on placebo, which was statistically significant (p=0.01) [12].
30/03/2016 12:14:02
Mar 16: GW Pharmaceuticals announces positive results of the first pivotal PIII study of cannabidiol (Epidiolex) for treatment of Dravet syndrome. Patients taking Epidiolex achieved a median reduction in monthly convulsive seizures of 39% vs. a reduction on placebo of 13% (p=0.01). A series of sensitivity analyses of the primary endpoint confirmed the robustness of this result. The difference between Epidiolex and placebo emerged dur
