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 详细处方信息以本药内容附件PDF文件（201181422060215.pdf）的“原文Priscribing Information”为准
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部分中文科望处方资料（仅供参考）

治疗罕见病新药Kuvan上市
总部位于瑞士日内瓦的默克集团的最大子公司默克—雪兰诺日前宣布，欧洲委员会已批准该公司生产的新药Kuvan——可用于治疗苯丙酮尿症(PKU)或高苯丙氨酸血症(HPA)——上市。此前欧洲药物审评局向Kuvan颁发了孤儿药证书。孤儿药是指用于罕见病、但商业价值较小、没有药厂愿意投资开发的药物。在美国或欧盟获得孤儿药资格的药物，可得到减税等优惠。

该公司副总裁Roberto Gradnik说：“PKU是一种遗传性疾病，如果患者没能严格控制饮食，PKU能够导致儿童脑部严重受损，导致成人暂时性甚至永久性的损害。欧洲一直没有可用于PKU治疗的药物。Kuvan上市后将为患者提供有效的治疗，更好地控制患者的血苯丙氨酸水平，有助于提高患者的生活质量，并可能最终减少脑部持续受损的风险。”

全欧大约35000人被确诊患有PKU或HPA，PKU或HPA发病率低，患者人数少，因此是一种罕见病。由于患者血液中苯丙氨酸水平过高，因此，严格限制苯丙氨酸含量较高的食品，如肉类、鱼类、坚果、乳类制品以及部分蔬菜和水果等，并补充氨基酸的饮食仍然是目前患者所能选择的唯一疗法，但长期下去可能会造成患者营养不良。

两项大型国际性双盲随机安慰剂对照Ⅲ期临床试验结果表明，Kuvan能够降低血苯丙氨酸水平，增加血苯基丙氨酸浓度处于目标范围内的患者数量，从而提高患者对于饮食中苯丙氨酸的耐受性，有助于减轻饮食中苯丙氨酸的限制。

欧洲委员会已为Kuvan颁发了在欧盟27个国家以及冰岛、列支敦士登和挪威的市场授权，其治疗适应症将在欧盟获得10年的保护。Kuvan预计将于今年上半年在欧洲上市。

默克认为Kuvan极具发展潜力，成为治疗这一罕见疾病的新药。据估计，在发达国家有50000人患有PKU，而他们中间30%-50%的人可以选择这种药物进行治疗。HPA的特征是患者体内血液中的苯丙氨酸含量增加，这会使神经细胞中毒，若不加以治疗，最终会导致脑损伤。

Kuvan® (sapropterin dihydrochloride) Tablets for PKU
KUVAN® (sapropterin hydrochloride) Tablets is the first and only FDA-approved medication for PKU to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4-) responsive PKU. Kuvan is a pharmaceutical formulation of BH4, the natural cofactor for the PAH enzyme, which stimulates activity of the residual PAH enzyme to metabolize Phe into tyrosine. Kuvan is to be used in conjunction with a Phe-restricted diet.

Kuvan at a Glance
•Approved in the United States, European Union, and Japan
•Designated an orphan drug in the United States and European Union
•Manufactured and commercialized by BioMarin in the United States

Regulatory Status
In December2007, Kuvan received marketing approval from the US Food and Drug Administration making it the first specific therapy for the treatment of PKU patients. A year later, Kuvan received approval in the European Union in December 2008. Kuvan has been designated an orphan drug in the US and EU, which grants market exclusivity for 7 and 10 years, respectively. (Biopten) was approved for PKU in Japan in July 2008.

Commercialization Plan
Kuvan is commercialized by BioMarin in the United States, Merck Serono in the European Union, and Asubio Pharma Co., Ltd. in Japan.

Full Indication and Important Safety Information (United States)
KUVAN® (sapropterin dihydrochloride) Tablets is approved to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN is to be used with a Phe-restricted diet.

Important Safety Information
High blood Phe levels are toxic to the brain and can lead to lower intelligence and decrease in the ability to focus, remember and organize information. Any change you make to your diet may impact your blood Phe level. Follow your doctor’s instructions carefully. Your doctor and dietitian will continue to monitor your diet and blood Phe levels throughout your treatment with KUVAN.

If you have a fever, or if you are sick, your Phe level may go up. Tell your doctor and dietitian as soon as possible so they can see if they have to adjust your treatment to help keep your blood Phe levels in the desired range.

KUVAN is a prescription medicine and should not be taken by people who are allergic to any of its ingredients. Tell your doctor if you have ever had liver or kidney problems, are nursing or pregnant or may become pregnant, have poor nutrition or are anorexic. Your doctor will decide if KUVAN is right for you. Tell your doctor about all the medicines you take.

The most common side effects reported when using KUVAN are headache, diarrhea, abdominal pain, upper respiratory tract infection (like a cold), throat pain, vomiting, and nausea.

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 详细处方信息以本药内容附件PDF文件（201181422060215.pdf）的“原文Priscribing Information”为准
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