英文药名：CERDELGA capsules(Eliglustat Tartrate) 中文药名：依利格鲁司特胶囊 生产厂家：赛诺菲公司 サデルガカプセル100mg 治疗类别名称 葡糖苷合酶抑制剂 批准上市日期：2015年9月 商標名 CERDELGA 一般名 エリグルスタット酒石酸塩　Eliglustat Tartrate 化学名 N-［（1R,2R）-1-（2,3-Dihydrobenzo［b］［1,4］dioxin-6-yl）-1-hydroxy-3-（pyrrolidin-1-yl）propan-2-yl］octanamide hemi-（2R,3R）-tartrate 分子式 （C23H36N2O4）2・C4H6O6 分子量 959.17 化学構造式 性状 白细黄棕色粉末。 熔点 168℃ 条件批准 和药品风险管理计划在发展，要正确实施。 临床试验的病人都在国内，上市后极其有限，通过在复试期间实施的所有管理的情况下使用效果调查，并了解这种药物的使用患者的背景资料，早期的数据收集这种药物的安全性和有效性，采取必要的措施，正确使用此药。 药效药理 1. 作用机序 戈谢病是由葡糖脑苷脂酶活性溶酶体酶滴剂，葡糖主要积累在巨噬细胞的溶酶体，肝和脾的大小，贫血和血小板减少，异常骨疼痛和骨的肿瘤和带来的变形。药物被选择性抑制葡萄糖神经酰胺合酶，抑制葡糖的形成。 2. 药理作用 (1) 葡糖苷酶抑制活性 体外：在来自人黑素瘤细胞系制备的微粒，一个葡萄糖神经酰胺合酶的浓度依赖性（19.6±0.68nmol/ L的IC 50值）抑制。 (2) 减少的浓度的葡糖效果 1）本剂对健康成人（外国人，男性和女性）时，剂量为50，200和350毫克的每日两次重复给药，血浆葡糖浓度以剂量依赖性方式降低 2）该药物减少在动物实验和犬末梢组织正常大鼠血浆的葡糖浓度。 3）该药物是D409V/空戈谢病I型模型小鼠抑制了葡糖苷时间积累的组织。该代理是D409V/空戈谢病的I型小鼠年老的模型，以减少外周组织和血浆中累积的葡糖。 3. 细胞的数量戈谢降血脂作用 本剂是D409V/空戈谢病的I型小鼠模型，减少了戈谢病（戈谢细胞）典型活化溶胀的巨噬细胞的数量。 适应病症 戈谢病（贫血，血小板减少症，肝脾肿大和骨症状）的改善的各种症状。 用法与用量 成人口服，每日2次，每次100毫克/粒。此外，要根据患者的状况病情适当使用。 包装规格 胶囊剂 100毫克 PTP 14胶囊 制造和销售 赛诺菲公司 完整处方资料附件：http://www.info.pmda.go.jp/go/pack/3999037M1023_2_01/ CERDELGA capsules(Eliglustat Tartrate)サデルガカプセル100mg 近日，口服戈谢病药物Cerdelga（Eliglustat Tartrate サデルガカプセル100mg）获得日本批准，用于特定1型戈谢病（Gaucher disease）成人患者的一线治疗. CERDELGA (eliglustat) capsules is a glucosylceramide synthase inhibitor indicated for the long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. CYP2D6 ultra-rapid metabolizers may not achieve adequate concentrations of CERDELGA to achieve a therapeutic effect. A specific dose cannot be recommended for CYP2D6 indeterminate metabolizers. Important Safety InformationCERDELGA (eliglustat) capsules are a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in certain adults. Your doctor will perform a test to help determine if CERDELGA is right for you. It is not known if CERDELGA is safe and effective in children. Before taking CERDELGA, tell your doctor about all of your medical conditions, including kidney or liver problems, history of heart attacks, or heart rhythm problems (including long QT syndrome). If you are pregnant or plan to become pregnant or breastfeed, talk to your physician. It is not known if CERDELGA will harm your unborn baby. CERDELGA can affect the way other medicines work and other medicines can affect how CERDELGA works. Using CERDELGA with other medicines or herbal supplements (including St. John’s Wort) may cause an increased risk of side effects, including ECG changes and irregular heart beat. Especially tell your doctor if you take medicines for fungal infections, tuberculosis, seizures, heart rhythm and rate problems, high blood pressure, or depression or other mental health problems. Your doctor may need to prescribe a different medicine, change your dose of other medicines, or change your dose of CERDELGA. Tell your doctor about any new medicines before you start taking them. Swallow the capsule whole. If you miss a dose of CERDELGA, take the next dose at the usual time. Do not take two doses of CERDELGA at the same time. Avoid eating or drinking grapefruit products while taking CERDELGA. Grapefruit products can increase the amount of CERDELGA in your body. The most common adverse reactions (≥10%) for CERDELGA are: fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain. Call your doctor for medical advice about adverse effects.