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尼替西农胶囊|Orfadin(nitisinone capsules)

2016-08-27 05:24:54 作者：新特药房来源：互联网浏览次数：0 文字大小：【大】【中】【小】

简介： 2016年6月15日，Sobi(Swedish Orphan Biovitrum AB)公司宣布美国食品药品管理局（FDA）已经批准的Orfadin（尼替西农）用于治疗I型遗传性酪氨酸血症（HT-1）。HT-1是一种罕见的遗传疾病，影响婴幼儿和儿童 ...

关键字：胶囊尼替西农 nitisinone 商品名 ORFADIN capsules 用于治疗I型遗传性酪氨酸血症（HT-1）

2016年6月15日，Sobi(Swedish Orphan Biovitrum AB)公司宣布美国食品药品管理局（FDA）已经批准的Orfadin（尼替西农）用于治疗I型遗传性酪氨酸血症（HT-1）。
HT-1是一种罕见的遗传疾病，影响婴幼儿和儿童。它是渐进的并可能导致肝脏和肾脏并发症，如果不治疗可能致命。
批准日期：2016年6月15日 公司：Sobi Inc
ORFADIN®（尼替西农 nitisinone）胶囊口服使用
ORFADIN®（尼替西农 nitisinone）口服混悬液
美国初始批准：2002
作用机理
尼替西农为4-羟基苯基丙酮酸双加氧酶的竞争性抑制剂，酪氨酸分解代谢途径延胡索酰乙酰乙酸水解酶上游的酶（FAH）。通过用HT-1抑制酪氨酸的正常代谢的患者，尼替西农防止分解代谢中间体和maleylacetoacetate延胡索酰乙酰乙酸的积累。患者用HT-1，论文分解代谢中间体转化成毒性代谢物和琥珀酰丙酮succinylacetoacetate，魁是负责观察肝和肾毒性。琥珀酰丙酮进行相反的能抑制卟啉合成途径导致5-氨基乙酰丙酸，负责HT-1的卟啉危机特性的神经毒素的积累。
尼替西农抑制氨基酸酪氨酸的分解代谢，并可能导致酪氨酸的血浆水平升高。该因此，尼替西农治疗需要从食物中摄取酪氨酸和苯丙氨酸，防止预防与酪氨酸的血浆水平升高相关的毒性的限制[见警告和注意事项]。
适应症和用法
ORFADIN是指示用于与酪氨酸和苯丙氨酸的饮食限制在组合遗传性酪氨酸1型（HT-1）的处理4羟基苯加氧酶抑制剂。
用法用量
推荐用量：
推荐的初始剂量为0.5毫克/公斤，每天两次口服。
滴定基于生化和/或临床应答的剂量，如在完整处方信息中描述。
的最大剂量为1mg/kg的每日两次口服。
编制和管理说明：
有关预付费，测量和管理口服混悬液说明，请参阅完整的处方信息。
维持酪氨酸和苯丙氨酸饮食限制
取ORFADIN胶囊之前至少一小时，两个小时，饭后具有
对于患者具备吞咽困难胶囊和不能耐受口服混悬液，胶囊可能是ouvert和内容悬浮在使用前立即水，配方或苹果酱少量时间。
就拿ORFADIN口服混悬液不考虑饭菜。
剂型和规格
胶囊：2毫克，5毫克，10毫克。
口服混悬液：4毫克/毫升
禁忌症
无
警告和注意事项
提高血浆酪氨酸水平，眼部症状，发育迟缓和角化板：酪氨酸和苯丙氨酸的摄入量不足的限制可能会导致血浆酪氨酸升高，魁高于500微摩尔/升的水平可能会导致症状，智力残疾和发育迟缓或痛苦的角化在鞋底和手掌的斑块;不以降低血浆酪氨酸浓度调整ORFADIN剂量。事先得到治疗，如症状发展复试裂隙灯检查。评估患者血浆酪氨酸水平与神经功能状态的突然变化。
严重的白细胞减少和血小板减少：血小板监测及白细胞计数。
由于内容的甘油20ml的口服混悬液ORFADIN可能因头痛，胃部不适和腹泻的剂量：不良反应由于ORFADIN口服混悬液的甘油含量的危险。考虑改用患者ORFADIN胶囊。
不良反应
大多数反对常见的反应（>1％）是酪氨酸水平升高，血小板减少，白细胞减少症，结膜炎，角膜混浊，角膜炎，畏光，眼痛，眼睑炎，白内障，粒细胞减少，鼻衄，皮肤瘙痒，剥脱性皮炎，皮肤干燥，斑丘疹和脱发。
报告可疑的不良反应，跟瑞典孤儿Biovitrum公司在1-866-773-5274或FDA电话1-800-FDA-1088或www.fda.gov/medwatch。
药物相互作用
CYP2C9底物：潜力这些共同给药的药物的增加的全身性暴露，额外的监测可能有必要。
Orfadin® (nitisinone) .Swedish Orphan Biovitrum has the worldwide rights to this product.
What is Orfadin?
Orfadin is a hard white capsule for oral use. Treatment with Orfadin should be started and monitoredby doctors who are experienced in the treatment of hereditary tyrosinemia type 1 (HT-1) patients.Orfadin contains the active substance nitisinone.
What is Orfadin used for?
Orfadin is used to treat hereditary tyrosinemia type 1 (HT-1), a rare childhood disease. In this diseasethe body is unable to completely break down the amino acid tyrosine. Harmful substances will beformed and accumulate in the body, causing progressive liver failure and liver cancer in youngchildren. It is used with a diet that restricts the amino acids tyrosine and phenylalanine.
Because hereditary tyrosinemia type 1 is a rare disease Orfadin was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 29 December 2000.
The medicine can only be obtained with a prescription.
How is Orfadin used?
Orfadin should be started as early as possible and the dose of Orfadin adjusted to the patient. The recommended starting dose is 1 mg/kg body weight/day divided in 2 doses administered orally. The capsules may be opened and their contents mixed in a small amount of water or formula diet just before swallowing. Orfadin is intended for long-term use.
How does Orfadin work?
Tyrosine is broken down in the body by a number of enzymes. Patients with HT-1 lack one of these enzymes, and the tyrosine in their body is not properly eliminated, but is transformed instead into harmful substances. Nitisinone, the active substance in Orfadin, blocks the breakdown of tyrosinebefore it can be transformed into harmful substances. However, tyrosine will remain in the body andtherefore a special diet (with low tyrosine and phenylalanine content) must be followed when takingOrfadin.
How has Orfadin been studied?
The largest study of Orfadin has been in 257 patients in 87 different hospitals in 25 countries, as part of a ‘compassionate use’ programme (this is a programme through which doctors can request a medicine for one of their patients before the medicine is fully authorised). The effect of Orfadin on survival was studied, and compared to historical records (report of patient survival with diet only as published in medical journals).
What benefit has Orfadin shown during the studies?
The main benefit is that Orfadin has been shown to greatly extend life. For example, a baby less than 2 months old with HT-1 would normally have only a 28% chance of surviving 5 years using diet alone. With additional Orfadin treatment, the survival rate increases to 82%. The sooner treatment is started, the better the rate of survival.
What is the risk associated with Orfadin?
The most common side effects of Orfadin are the result of high tyrosine levels caused by patients not eating the right foods as well as rare cases of a decrease in platelet and white blood cell counts. For the full description of the side effects reported with Orfadin, please see the package leaflet. Orfadin should not be used in people who may be hypersensitive (allergic) to nitisinone or any of the other ingredients.
Why has Orfadin been approved?
The Committee for Medicinal products for Human Use (CHMP) decided that Orfadin seems to be an effective treatment of HT-1, particularly if started early before the patient’s liver is too damaged, and it provides a better outcome for patients than that reported in literature with diet alone. The CHMP decided that Orfadin’s benefits are greater than its risks and recommended that Orfadin be given marketing authorisation.
What information is still awaited for Orfadin?
The company that makes Orfadin has established a post-marketing surveillance program to monitor the use and safety of the medicine in the treatment of HT-1.
Other information about Orfadin:
The European Commission granted a marketing authorisation valid throughout the European Union for Orfadin to Swedish Orphan International AB on 21 February 2005.
完整处方资料附件：
https://dailymed.nlm.nih.gov/dailymed/archives/fdaDrugInfo.cfm?archiveid=15667
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产地国家：美国
中文参考药品译名：
尼替西农
原产地英文药品名：
NITISINONE
中文参考商品译名：
欧佛定 2毫克/胶囊 60胶囊/瓶
原产地英文商品名：
ORFADIN 2mg/Cap 60Caps/bottle
生产厂家中文参考译名：
Sobi Inc
生产厂家英文名：
Sobi Inc
---------------------------------------------------------------
产地国家：美国
中文参考药品译名：
尼替西农
原产地英文药品名：
NITISINONE
中文参考商品译名：
欧佛定 5毫克/胶囊 60胶囊/瓶
原产地英文商品名：
ORFADIN 5mg/Cap 60Caps/bottle
生产厂家中文参考译名：
Sobi Inc
生产厂家英文名：
Sobi Inc
---------------------------------------------------------------
产地国家：美国
中文参考药品译名：
尼替西农
原产地英文药品名：
NITISINONE
中文参考商品译名：
欧佛定 10毫克/胶囊 60胶囊/瓶
原产地英文商品名：
ORFADIN 10mg/Cap 60Caps/bottle
生产厂家中文参考译名：
Sobi Inc
生产厂家英文名：
Sobi Inc
---------------------------------------------------------------
产地国家：美国
中文参考药品译名：
尼替西农
原产地英文药品名：
NITISINONE
中文参考商品译名：
欧佛定 20毫克/胶囊 60胶囊/瓶
原产地英文商品名：
ORFADIN 20mg/Cap 60Caps/bottle
生产厂家中文参考译名：
Sobi Inc
生产厂家英文名：
Sobi Inc
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产地国家：西班牙
中文参考药品译名：
尼替西农
原产地英文药品名：
NITISINONE
中文参考商品译名：
欧佛定 20毫克/胶囊 60胶囊/瓶
原产地英文商品名：
ORFADIN 20mg/Cap 60Caps/bottle
生产厂家中文参考译名：
Sobi Inc
生产厂家英文名：
Sobi Inc