Velaglucerase alfa商品名为Vpriv,属于生物药物,是美国Shire公司开发的一种水解性溶酶体特异性葡萄糖脑苷脂酶。Vpriv于2010年2月26日获美国FDA批准上市,注射用于儿童和成人的1型戈谢病(Gaucher disease,GD)的长期酶替代治疗(ERT)。 1型儿童及成人戈谢病为常染色体隐性遗传性疾病。是由于β-葡糖苷酶-葡糖脑苷酯酶缺乏致葡糖脑苷脂在肝、脾、骨骼和中枢神经系统的单核巨噬细胞内蓄积。 Vpri属于人细胞源性酶替代疗法（ERT）药物，可作长期用药治疗1型儿童及成人戈谢病。Shire公司表示，之前已获准治疗戈谢病的ERT药物在供应上没有保证，在样的情况下Vpriv的上市可为这类患者提供新的用药选择。 更令人欣喜的是，Vpriv的售价将比其他同类ERT药物价格低15%。 FDA药物评价第III办公室主任Julie Beitz医学博士说：“VPRIV的批准将为高歇氏病患者提供一种安全和有效替代治疗”，“既往接受Cerezyme酶替代治疗1型高歇氏病的患者可安全转用。” VPRIV®（velaglucerase阿尔法供注射用），静脉使用 美国首次批准：2010年2月  公司： 目前的主要变化 剂量和给药方法 警告和注意事项 二千零十五​​分之四 二千零十五​​分之四 适应症和用法 VPRIV为长期酶替代疗法（ERT）表示的水解溶酶体葡糖脑特异性酶为患者的1型戈谢病。 用法用量 建议在成人和儿童患者4岁以上的起始剂量为： 患者天真的酶替代疗法：60单位/千克。 患者被稳定伊米苷酶剂量的戈谢病治疗：两个星期后，去年伊米苷酶剂量，可切换到VPRIV以往伊米苷酶剂量。 根据确定的小瓶的数量来重建病人的实际重量和处方剂量。 提供的VPRIV冻干粉末必须配制用注射用无菌水。 重构VPRIV溶液必须在100毫升之前静脉内输注0.9％氯化钠溶液稀释。 通过直列低蛋白结合0.2μm的过滤器管理该稀释VPRIV溶液 剂型和规格 冻干粉末进行重构和稀释输液。 可提供400套单次使用小瓶。 禁忌 无 警告和注意事项 超敏性反应包括过敏性休克： 过敏反应，包括过敏性休克发生。 确保人员管理产品的心肺复苏措施得到充分的培训，并随时实施紧急医疗服务 考虑减慢输注速率，治疗用抗组胺剂，退烧药和/或皮质类固醇，和/或如果一个超敏反应的输注期间发生在停止治疗。考虑预治疗的患者之前反应抗组胺药和/或皮质类固醇。 不良反应 在患者≥10％临床研究中最常见的不良反应有：过敏反应，头痛，头晕，腹痛，恶心，背痛，关节痛，长期激活PTT，疲劳/虚弱，和发热。 修订：4/2015 包装规格 1 NDC:54092-701-041小瓶，玻璃（瓶）1箱包含一小瓶，玻璃 14毫升1瓶，玻璃包内包含的BOX（54092-701-04） 2 NDC:54092-701-021小瓶，玻璃1盒（瓶），包含一小瓶，玻璃 2毫升1瓶，玻璃这个包内包含的BOX（54092-701-02） VPRIV (Shire Human Genetics Therapies, Inc.) VPRIV- velaglucerase alfa injection, powder, lyophilized, for solution Shire Human Genetics Therapies, Inc. Velaglucerase alfa Pronunciation: VEL-a-GLOO-ser-ase AL-fa Class: Enzyme replacement Trade Names AdvertisementVPRIV - Injection, lyophilized powder for solution 200 units - Injection, lyophilized powder for solution 400 units Pharmacology Velaglucerase alfa catalyzes the hydrolysis of glucocerebroside to glucose and ceramide. Slideshow: The ABC's of Hepatitis: Get to Know This Viral Disease Pharmacokinetics Distribution Vd is 82 to 108 mL/kg. Elimination The half-life is 11 to 12 min. Plasma Cl ranges from 6.72 to 7.56 mL/min/kg. Indications and Usage Long-term enzyme replacement therapy for children and adults with type I Gaucher disease. Contraindications None well documented. Dosage and Administration Adults and Children older than 4 yr of age IV 60 units/kg every other week. Dosage adjustments are based on achievement and maintenance of each patient's therapeutic goals. Clinical studies have evaluated doses ranging from 15 units/kg to 60 units/kg every other week. General Advice •For IV infusion only. Not for intradermal, subcutaneous, IM, IV bolus, or intra-arterial administration. •Reconstitute powder with sterile water for injection: 2.2 mL for 200 unit vial; 4.3 mL for 400 unit vial. •Upon reconstitution, mix vial gently. Do not shake. •Do not use if reconstituted solution is cloudy or discolored, or contains opaque particles. •Withdraw prescribed dose from vial(s) and dilute in 100 mL of sodium chloride 0.9% injection. •Administer diluted solution by 60-min IV infusion. •Do not infuse with other products in the same infusion tubing because the compatibility in solution with other products has not been evaluated. •Diluted solution may be filtered through an in-line, low protein-binding 0.2 mcm filter during administration. •Consider pretreatment with antihistamines and/or corticosteroids if hypersensitivity symptoms developed during previous infusion. Storage/Stability Store vials at 36° to 46°F. Reconstituted or diluted solution is stable for up to 24 h when stored at 36° to 46°F. The infusion should be completed within 24 h of reconstitution. Do not freeze. Protect from light. Discard any unused reconstituted solution. Do not save for future use. Drug Interactions None well documented. Adverse Reactions Cardiovascular Hypertension, hypotension, tachycardia (more than 3%). CNS Headache (36%); dizziness (22%); asthenia/fatigue (13%). Dermatologic Flushing, rash, urticaria (more than 3%). GI Abdominal pain (19%); nausea (10%). Musculoskeletal Back pain (18%); joint (knee) pain (15%); bone pain (more than 3%). Miscellaneous Infusion-related reaction (52%); upper respiratory tract infection (32%); pyrexia (22%); APTT prolonged (11%). Precautions Monitor Periodically monitor for immunoglobulin G (IgG) antibody formation during first year of treatment. Pregnancy Category B . Lactation Undetermined. Children Safety and efficacy in patients younger than 4 yr of age are not established. Elderly Approach dose selection for an elderly patient cautiously; consider potential comorbid conditions. Hypersensitivity Has been reported in clinical studies. Use with caution in patients who have exhibited symptoms of hypersensitivity to velaglucerase alfa, excipients in the drug product, or to other enzyme replacement therapies. Immunogenicity IgG antibodies to velaglucerase alfa may occur. Infusion-related reactions Infusion-related reactions (eg, dizziness, fatigue/asthenia, headache, hypertension, hypotension, nausea, pyrexia) may occur. Overdosage Symptoms No data available. Patient Information •Advise patient or caregiver that medication will be prepared and administered by a health care provider in a health care setting. •Advise patient or caregiver that reactions during or shortly after infusion occur commonly and that medications may be given before the infusion in an effort to prevent or reduce the severity of these reactions. •Advise patient or caregiver to notify health care provider if any of the following occur: pain, redness, or burning at infusion site; persistent or intolerable nausea, vomiting, stomach pain, diarrhea, or dizziness; any other unexplained symptom or sign.