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 详细处方信息以本药内容附件PDF文件（200821618504132.pdf）的“原文Priscribing Information”为准
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部分中文Elaprase处方资料（仅供参考）

Elaprase (idursulfase) 治疗粘多糖贮积症Ⅱ型 (Hunter 综合症)
美国FDA批准Elaprase(通用名idursulfase)，首个用于治疗享特综合征(Hunter syndrome，又名Mucopolysaccharidosis II［Ⅱ型粘多糖贮积症］，或MPS Ⅱ)的产品，享特综合征是一种罕见遗传疾病，它可能导致过早死亡。Elaprase是一个新分子实体，它是一种以前从未在美国上市的活性成分。

享特综合征，通常在儿童1至3岁时变得明显，患这种疾病的人的身体在产生一种化学物质induronate-2-sulfatase上有缺陷，induronate-2-sulfatase是充分分解人体内产生的复杂糖分所需的。各种症状包括生长延缓，关节僵硬，以及面部特征粗糙。在严重病例中，患者遭受呼吸疾病和心脏疾病，肝脾肥大，神经缺陷和死亡。

Elaprase被FDA指定为罕见病药。罕见病药(注：又称“孤儿药”)，例如Elaprase，通常被开发用于治疗影响美国20万人以下的罕见疾病或病况。《罕见病药法》对首个获得指定罕见病药的上市许可的申办者提供了一个7年的独家销售期。大约65000到132000个新生儿中有1个被诊断患有享特综合征。

“这是首个能帮助为数很少的患有严重疾病但没有其它治疗选择的人的产品，”CDER主任Steven Galson博士称。“这个批准是罕见病药计划如何用急需产品使公众健康受益的一个很好的例子，要不然这些产品不能在商业上可供获得。”

Elaprase在对96名患有享特综合征的患者进行了一项随机双盲安慰剂对照研究后被批准，该研究表明接受治疗的患者改善了行走能力。在53周的治疗后，接受了Elaprase输液的患者比安慰剂组6分钟内的行走距离平均增加了38码（1码≈0.914米）。

试验期间报告的最严重的不良事件是可能危及生命的对Elaprase的超敏反应。它们包括：呼吸抑制、血压降低、颠痫。其它频繁但较不严重的不良事件包括发烧、头痛、关节痛。

因为可能的严重超敏反应，在Elaprase给药的同时应当可以获取立即的适当的医疗支持。鼓励患者及其医生参与自愿的享特综合征治疗结果调查，该调查的目的是为了监测和评价用Elaprase进行长期治疗的安全性和有效性。

Elaprase由马萨诸塞州Cambridge市的Shire Human Genetic Therapies公司生产。

Indication
ELAPRASE® (idursulfase) is indicated for patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II). ELAPRASE has been shown to improve walking capacity in these patients.

Important Safety Information
Some patients in the clinical trials experienced life-threatening immediate allergic reactions to ELAPRASE (idursulfase) infusion. Patients who have experienced severe allergic reactions may experience another allergic reaction approximately 24 hours after the initial reaction and may require prolonged observation. Patients with compromised respiratory function or acute respiratory disease may have a higher risk of life-threatening reactions to ELAPRASE infusion and require additional monitoring.

Life-threatening allergic reactions have been seen in some patients during ELAPRASE infusions. Reactions included difficulty breathing, lack of oxygen, seizure or loss of consciousness, hives, and/or swelling of the throat or tongue. Patients received an antihistamine to reverse the allergic reaction or corticosteroid to decrease inflammation prior to or during later infusions. ELAPRASE was given more slowly or was stopped early if patients had serious reactions. With these measures, no patients stopped treatment permanently due to an infusion reaction.

Because of the potential for life-threatening complications, the ELAPRASE infusion may have to be delayed for patients who have respiratory illness and/or fever.

The most common side effects that required intervention had to do with infusions. These included muscle and joint aches, headache, fever, rash, hives, itching and increased blood pressure. These side effects decreased over time with continued ELAPRASE treatment.

In clinical trials, patients given ELAPRASE once a week for 52 weeks had the following side effects at a rate greater than placebo and at least 10%. Approximately:
2 out of 3 patients had a fever or headache.
1 out of 3 patients had joint pain.
1 out of 4 patients had itching or high blood pressure.
1 out of 5 patients had general discomfort or disturbed vision or wheezing.
1 out of 6 patients had abscess or muscle/bone pain or chest wall pain or hives.
1 out of 7 patients had anxiety/irritability or irregular heart rhythm or stomach upset or swelling at the infusion site or a skin disorder or itchy rash or slight injury or a side effect from injury.

About half of the patients in clinical studies produced antibodies to treatment with ELAPRASE and these patients had an increase in infusion reactions. The presence of antibodies on the effectiveness of ELAPRASE is unknown.

Please see full Prescribing Information including Boxed Warning

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 详细处方信息以本药内容附件PDF文件（200821618504132.pdf）的“原文Priscribing Information”为准
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