近日，美国FDA批准其新药Epidiolex口服溶液治疗两岁及以上患者的两种罕见和严重癫痫，Lennox-Gastaut综合征和Dravet综合征相关的癫痫发作。这是FDA批准的第一种含有从大麻中提取的纯化药物的药物。这也是FDA首次批准用于治疗Dravet综合征患者的药物。 批准日期：2018年7月2日 公司：GW RESEARCH LTD EPIDIOLEX(大麻酚[cannabidiol])溶液， 供口服使用 首次美国批准：2018 作用机理 EpdioLeX在人体内发挥抗惊厥作用的确切机制尚不清楚。大麻酚不能发挥其抗惊厥作用。通过与大麻素受体相互作用的效果。 适应症及用法 EPIDIOLEX用于治疗2岁及以上的Lennox-Gastautsyndrome或Dravet综合征患者的癫痫发作。 剂量与给药 在开始治疗前，所有患者都获得血清转氨酶（ALT和AST）和总胆红素水平。 EpdioLeX是口服给药。 ·建议的起始剂量是2.5mg/kg，每天服用两次(5mg/kg/天)。每日两次（10mg/kg/天）。 ·根据个体临床反应和耐受性，EPIDIOLEX可以增加到最大推荐维持剂量10mg/kg，每天两次（20mg/kg/天）。 请参阅滴定的完整处方信息。 建议对中度或重度肝损害患者进行剂量调整。 剂型和强度 口服溶液：100mg／ml。 禁忌症 对大麻酚或任何成分的过敏。 警告和注意事项 肝细胞损伤：EpdioLeX可导致转氨酶升高。同时服用丙戊酸和较高剂量的EpdioLeX增加转氨酶的风险。 海拔高度。见完整的处方信息血清转氨酶和胆红素监测建议。 •嗜睡和镇静：监测嗜睡和镇静，并建议病人在获得足够的睡眠前不要驾驶或操作机器 EpdioLeX的经验。 自杀行为和思维：监测病人自杀行为和想法。 过敏反应：建议患者立即就医。如果发生超敏反应，停止和不重新启动EpdioLeX。 ·撤回抗癫痫药物：应逐步撤回EPIDIOLEX，以尽量减少癫痫发作频率增加和癫痫持续状态的风险。 不良反应 最常见的不良反应（EPIDIOLEX为10%以上，大于安慰剂）是：嗜睡；食欲减退；腹泻；转氨酶升高； 疲劳、乏力和虚弱；皮疹；失眠、睡眠障碍和睡眠质量差；以及感染。 要报告预期的不良反应，请联系格林威治生物科学公司1-833-424-6724（1-833-GBIOSCI）或FDA 1-800-FDA-1088或www.fda.gov/med.。 药物相互作用 CYP3A4或CYP2C19的中度或强抑制剂：考虑EpdioLeX的剂量减少。 CYP3A4或CYP2C19强诱导剂：考虑EpdioLeX的剂量增加。考虑减少UGT1A9、UGT2B7、CYP2C8、CYP2C9和CYP2C19（例如，氯巴扎）的底物的剂量。 CYP1A2和CY2B6的底物也可能需要剂量调节。 在特定人群中的使用 怀孕：基于动物数据，可能会造成胎儿伤害。 包装供应/储存和搬运 供应 EPIDIOLEX是一种草莓口味清澈，无色到黄色的溶液，在105毫升琥珀色玻璃瓶中供应，瓶盖内装有100毫升的口服溶液（NDC 70127—100-01）。每毫升含有100毫克的大麻酚。EpdioLeX包装在纸箱中，两个5毫升校准口服注射器和瓶适配器（NDC 70127至100-10）。药房将提供1毫升，当需要少于1毫升的剂量时，校准口服剂量注射器。 储运 将EPIDIOLEX原装瓶直立存放在20°至25°C(68°F至77°F)之间，允许在15°C至30°C(59°F至86°F)之间移动。 参见美国药典控制的室温。不要冷藏或冷冻。保持关闭。在12周内首次打开瓶子，然后丢弃，任何余数。 完整资料附件： 1）：https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/210365lbl.pdf 2）：https://www.epidiolex.com/sites/default/files/EPIDIOLEX_Full_Prescribing_ Information.pdf EPIDIOLEX(cannabidiol) oral solution, CX [pending DEA scheduling IMPORTANT SAFETY INFORMATION & INDICATIONS CONTRAINDICATION: HYPERSENSITIVITY EPIDIOLEX(cannabidiol) oral solution is contraindicated in patients with a history of hypersensitivity to cannabidiol or any ingredients in the product. WARNINGS & PRECAUTIONS Hepatocellular Injury: EPIDIOLEX can cause dose-related transaminase elevations. Concomitant use of valproate and elevated transaminase levels at baseline increase this risk. Transaminase and bilirubin levels should be obtained prior to starting treatment, at one, three, and six months after initiation of treatment, and periodically thereafter, or as clinically indicated. Resolution of transaminase elevations occurred with discontinuation of EPIDIOLEX, reduction of EPIDIOLEX and/or concomitant valproate, or without dose reduction. For patients with elevated transaminase levels, consider dose reduction or discontinuation of EPIDIOLEX or concomitant medications known to affect the liver (e.g., valproate or clobazam). Dose adjustment and slower dose titration is recommended in patients with moderate or severe hepatic impairment. Consider not initiating EPIDIOLEX in patients with evidence of significant liver injury. Somnolence and Sedation: EPIDIOLEX can cause somnolence and sedation that generally occurs early in treatment and may diminish over time; these effects occur more commonly in patients using clobazam and may be potentiated by other CNS depressants. Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including EPIDIOLEX, increase the risk of suicidal thoughts or behavior. Inform patients, caregivers, and families of the risk and advise to monitor and report any signs of depression, suicidal thoughts or behavior, or unusual changes in mood or behavior. If these symptoms occur, consider if they are related to the AED or the underlying illness. Withdrawal of Antiepileptic Drugs: As with most AEDs, EPIDIOLEX should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. Adverse Reactions: The most common adverse reactions in patients receiving EPIDIOLEX (≥10% and greater than placebo) include somnolence; decreased appetite; diarrhea; transaminase elevations; fatigue, malaise, and asthenia; rash; insomnia, sleep disorder and poor-quality sleep; and infections. Hematologic abnormalities were also observed. Pregnancy: EPIDIOLEX should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Encourage women who are taking EPIDIOLEX during pregnancy to enroll in the North American Antiepileptic Drug (NAAED) Pregnancy Registry. Drug Interactions: Moderate or strong inhibitors or inducers of CYP3A4 and CYP2C19 may affect EPIDIOLEX exposure. EPIDIOLEX may affect exposure to CYP2C19 substrates (e.g., clobazam, diazepam) or others. Concomitant use of EPIDIOLEX and valproate increases the incidence of liver enzyme elevations. Dosage adjustment of EPIDIOLEX or other concomitant medications may be necessary. Drug Abuse: EPIDIOLEX is a Schedule V controlled substance and has a low potential for abuse. Indications: EPIDIOLEX(cannabidiol) oral solution is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) in patients 2 years of age and older. Please refer to the EPIDIOLEX full Prescribing Information for additional important information.