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美国FDA批准拜BioMarin Pharmaceutical公司的二盐酸沙丙蝶呤片（sapropterin dihydrochloride,Kuvan）上市治疗苯丙酮尿症.本品是首个获准治疗苯丙酮尿症（PKU）的专一性治疗药,其上市对PKU患者及其家属和BioMarin Pharmaceutical公司都是一重要的里程碑。
在临床研究中，二盐酸沙丙蝶呤显示有助于控制PKU患者的苯丙氨酸血浓度。
本品适用于降低由与PKU相关的四氢生物蝶呤引起的高苯丙氨酸血症患者的苯丙氨酸血浓度，治疗期间需限苯丙氨酸饮食。为确定Kuvan的疗效，推荐初始剂量为二盐酸沙丙蝶呤一日1次10 mg/（kg·日），服用1月。若无效，药物剂量增至一日1次20 mg/（kg·日），服用1月；剂量可在5～20 mg/（kg·日）范围内调节。
二盐酸沙丙蝶呤系一合成的6R-BH4（四氢生物蝶呤），一种与苯丙氨酸羟化酶（PAH）结合代谢苯丙氨酸自然存在的辅酶。BioMarin Pharmaceutical公司和默克雪兰诺（Merck Serono）公司预测，Kuvan可用于治疗全球发达国家估计已诊断出约5万PKU患者的30%～50%。
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二盐酸沙丙蝶呤片（sapropterin dihydrochloride,商品名：Kuvan）处方
Generic Name for KUVAN
Sapropterin dihydrochloride 100mg; tabs.
Legal Classification:
Rx
Pharmacological Class for KUVAN
Enzyme cofactor (BH4).
Manufacturer of KUVAN
BioMarin Pharmaceuticals
Indications for KUVAN
In conjunction with a phenylalanine (Phe)-restricted diet: To reduce blood Phe levels in hyperphenylalaninemia (HPA) in responsive phenylketonuria (PKU).
Adults and Children:
<4 years: consult manufacturer. ≥4 years: Take with food. Dissolve tablet in 4–8oz water or apple juice (may crush tab) and drink within 15 minutes (tablet may not completely dissolve). Initially 10mg/kg once daily; if inadequate response may increase to 20mg/kg once daily; if inadequate response after 1 month, discontinue. Usual range 5–20mg/kg per day; adjust based on blood Phe levels.
Warnings/Precautions for KUVAN
Monitor blood levels of Phe after 1 week to assess effect, then periodically. Maintain Phe-restricted diet. Hepatic or renal impairment. Pregnancy (Cat.C). Nursing mothers: not recommended.
Interactions for KUVAN
Caution with drugs that can inhibit folate metabolism (eg, methotrexate), PDE-5 inhibitors (eg, sildenafil, tadalafil, vardenafil), other nitric oxide donors, levodopa.
Adverse Reactions for KUVAN
Headache, GI upset, upper respiratory infection/symptoms, throat pain, cough, fever, contusion, abdominal pain, rash; others (see literature).
How is KUVAN supplied?
Tabs—120
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原产地英文商品名:
KUVAN 100mg/tab 120tabs/bottle
原产地英文药品名:
SAPROPTERIN DIHYDROCHLORIDE
中文参考商品译名:
科望 100毫克/瓶 120片/瓶
中文参考药品译名:
盐酸沙丙蝶呤
生产厂家中文参考译名:
雪兰诺
生产厂家英文名:
BIOMARIN PHARM
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