FDA批准Elelyso（taliglucerase阿尔法）作为长期酶替代疗法治疗一类戈谢病（高雪病）。Elelyso是一种注射剂，对于确诊为1型（非神经型）戈谢病的患者，该药可以替代其所缺少的酶；Elelyso应当由专业医务人员隔周给药。

ELELYSO（taliglucerase阿尔法）注射，静脉注射使用

最初的美国2012年批准：
 
生产商：辉瑞公司

适应症
注射ELELYSO™（taliglucerase阿尔法）是水解溶酶体葡糖特定的酶的长期酶替代疗法（ERT），1型Gaucher病的确诊患者表示。

剂量和用法
作为一个60-120分钟的静脉滴注给药，每隔一周的60个单位/公斤
目前正在与伊米苷酶治疗Gaucher病的患者，可以切换到ELELYSO。以前稳定剂量的伊米苷酶治疗的患者，建议在相同剂量开始治疗时，他们从伊米苷酶切换到ELELYSO与ELELYSO。
医生可以根据每个病人的治疗目标的实现和维护调整剂量。临床试验评价剂量范围从11个单位/千克到73单位/公斤，每星期。
 
剂型和优势
注射用冻干粉重建与稀释剂
可在200单位单次用小瓶
 
禁忌
没有
 
注意事项：
过敏反应：过敏反应已被观察在与ELELYSO治疗的一些患者。如果发生过敏反应，应立即停止输液，并采取适当的治疗。
过敏反应和输液反应：输液反应（包括过敏反应）的最常见的症状是头痛，胸痛或不适，乏力，疲劳，荨麻疹，红斑，血压升高，背痛，关节痛，冲洗。如果发生过敏或输液反应，降低输注速率，暂时停止输液，或管理抗组胺药和/或建议退烧药。
 
不良反应
在临床研究中最常见的不良反应是输液反应。其他常用≥10％的患者在观察不良反应URTI /鼻咽炎，咽炎/喉咙发炎，头痛，关节痛，流感/流感，尿路感染/肾盂肾炎，腰痛，下肢疼痛。

报告疑似不良反应，接触辉瑞公司在（1-800-438-1985）或FDA在1-800-FDA-1088或www.fda.gov / medwatch

日期：05/2012

ELELYSO™ (taliglucerase alfa) for injection is supplied as 200 Units per vial and is available by prescription only.

INDICATION
ELELYSO™ (taliglucerase alfa) for injection is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for adults with a confirmed diagnosis of Type 1 Gaucher disease.

IMPORTANT SAFETY INFORMATION
As with any intravenous protein medicine, like enzyme replacement therapy (ERT), severe allergic reactions (including anaphylaxis) have been observed in patients treated with ELELYSO. If this occurs, your doctor may immediately discontinue ELELYSO. Patients who have experienced anaphylaxis to ELELYSO or another ERT should proceed with caution upon retreatment.

In addition, infusion reactions (including allergic reactions)—defined as a reaction occurring within 24 hours of the infusion—were the most commonly observed reactions to ELELYSO. The most commonly observed infusion reactions were headache, chest pain or discomfort, weakness, fatigue, hives, abnormal redness of the skin, increased blood pressure, back or joint pain, and flushing. Most of these reactions were mild and did not require treatment.

Management of infusion reactions is based on the type and severity of the reaction. Your doctor may manage infusion reactions by temporarily stopping the infusion, slowing the infusion rate, or treating with medications such as an antihistamine and/or a fever reducer. Treatment with antihistamines and/or corticosteroids prior to infusion with ELELYSO may prevent these reactions.

Other common adverse reactions observed were upper respiratory tract infections, throat infection, flu, urinary tract infection, and pain in extremities.

As with all therapeutic proteins, including ERTs, there is a possibility of developing antibodies to ELELYSO. However, it is currently unclear whether this has an impact on the clinical response or adverse reactions. Patients with an immune response to other ERTs who are switching to ELELYSO should continue to be monitored for antibodies. Comparison of the frequency of antibodies across ERTs may be misleading.

If you are pregnant, or plan to become pregnant, you should talk to your doctor about potential benefits and risks.

Please click here for full Prescribing Information.

The health information contained herein is provided for educational purposes only and is not intended to replace discussions with a health care provider. All decisions regarding patient care must be made with a health care provider, considering the unique characteristics of the patient.

FDA批准治疗Gaucher（高雪氏）病的新孤儿药

2012年5月1日，FDA宣布批准长效酶替代疗法taliglucerase alfa（Elelyso）用于治疗罕见遗传疾病——戈谢病。

戈谢病患者体内无法产生足够的葡萄糖脑苷脂酶。该酶的不足导致脂肪物质积蓄在脾、肝、肾等器官内。戈谢病的主要体征为肝脏或脾脏损害、贫血、血小板低和骨骼问题。

Elelyso是酶替代注射剂，由专业医护人员每隔一周给予1型（非神经性）戈谢病患者。在美国，预计大约有6,000名1型戈谢病患者。

由于该病影响到的人群较小，Elelyso疗效的评价在2项总共包含56名1型戈谢病患者的临床研究中进行，多数患者继续参加了后续扩展临床研究。

一项多中心、双盲、平行研究评价了Elelyso作为初始治疗用于31名没有接受过酶替代疗法的患者的作用。患者随机接受30或60units/kg剂量的Elelyso。两种剂量的Elelyso都有效减小了病人的脾脏体积（研究的首要终点），经过9个月的治疗，和基线相比，给予30 units/kg Elelyso的病人脾脏平均减小了29%，给予60 units/kg Elelyso的病人脾脏平均减小了40%。

另一项研究评价了Elelyso用于25名之前使用另一种酶替代治疗产品imiglucerase for Injection（注射用伊米苷酶，思而赞，Cerezyme）患者的作用。在这项多中心、开放式、单组研究中，之前这些至少使用了2年的imiglucerase的患者开始每隔一周给予和imiglucerase相同剂量的Elelyso。结果显示Elelyso在9个月评价期中有效维持了脾脏、肝脏体积、血小板计数和血红蛋白水平。

Elelyso临床研究最常见的不良反应为输液反应和过敏反应。输液反应的症状包括头疼、胸痛或不适、虚弱、疲劳、荨麻疹、皮肤发红、血压升高、背部疼痛、关节疼痛和脸红。和其他的静脉蛋白产品一样、Elelyso输液也出现过过敏反应。

其他常见的不良反应（>10%）包括上呼吸道感染，感冒样症状（鼻咽炎）、关节疼痛、流行性感冒、头疼、肢体末端疼痛、背部疼痛和尿道感染。

Elelyso由美国辉瑞负责生产销售，辉瑞是从Protalix BioTherapeutics获得的许可权。