尼替西农口服悬液(ORFADIN®Oral Suspension)获美国食品药品监督管理局(theFood and Drug Administration,FDA)批准，用于遗传性酪氨酸血症I型(Hereditary Tyrosinemia Type 1,HT-1)的治疗。使用尼替西农用药时，需限制酪氨酸和苯丙氨酸的摄入。
这种新制剂是胶囊药剂的一种替代物，其为婴幼儿提供了另一种选择。尼替西农口服悬液每支的剂量是4mg/mL。
“引进口服的悬液表明我们一直支持HT-1群体。这种新的液体制剂给服用ORFADIN的患儿的父母提供了新的选择，也同样适用于有吞服胶囊障碍的患者”，北美医学事务副总裁兼副主任莱恩·沃尔特说道。
HT-1是一种罕见的婴幼儿遗传病。病情会逐渐恶化，若不治疗会导致肝、肾并发症，甚至危及生命。在药物治疗手段出现之前，年龄小于2个月时就被确诊为HT-1的婴儿能存活至两岁只占不到三分之一。用ORFADIN治疗并结合饮食限制酪氨酸和苯丙氨酸的摄入，以及更普及的新生儿疾病筛查带来的早期诊断极大改善了HT-1患儿的预后。
尼替西农口服悬液将于2016年8月上市
尼替西农(ORFADIN®)
遗传性酪氨酸血症I型(HT-1)患者体内的酪氨酸分解能力失常。有毒副产物在体内形成并累积，可引起肝、肾及神经系统并发症。尼替西农能够阻断酪氨酸的分解，从而减少体内有毒副产物的含量。在最常见的疾病形式中，在儿童生命的前六个月内会出现症状。由于酪氨酸的不充分分解，患者必须维持与尼替西农治疗相结合的一种特殊的饮食。现今大约有1000人确诊患有HT-1。尼替西农是享有专利的产品，它由Sobi开发并在全球销售。
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。
完整处方资料附件：
https://dailymed.nlm.nih.gov/dailymed/archives/fdaDrugInfo.cfm?archiveid=15667
Sobi's Orfadin® oral suspension granted European patent
Swedish Orphan Biovitrum AB (publ) (Sobi) today announced that the European Patent Office (EPO) has decided to grant a European patent for the Orfadin® (nitisinone) oral suspension formulation, which was approved by the European Commission for the treatment of Hereditary Tyrosinaemia type-1 (HT-1) in 2015. HT-1 is a rare genetic disease that affects infants and children. It is progressive and may result in liver and kidney complications and can be fatal if untreated.
"Sobi has a long-term commitment to the HT-1 community and work to continuously improve the available treatment options. The oral suspension formulation is a demonstration of this commitment to the needs of the infants and children diagnosed with HT-1 early in life", said Geoffrey McDonough, CEO and President of Sobi. "The oral suspension of Orfadin for paediatric use facilitates dosing accuracy and simplifies ease of use."
Before pharmacological treatment was available, less than one third of infants diagnosed with HT-1 before two months of age lived past their second birthday.[i] Treatment with Orfadin combined with dietary restriction of tyrosine and phenylalanine and more widespread new-born screening, leading to early diagnosis have dramatically improved outcomes for HT-1 patients.[ii]
The patent, granted in January 2016, will confer protection to the Orfadin oral suspension formulation in each contracting and extension state to the European Patent Convention (EPC).
About Orfadin®
People with Hereditary Tyrosinaemia type-1 (HT-1) have problems breaking down an amino acid called tyrosine. Toxic by-products are formed and accumulate in the body, which can cause liver, renal and neurological complications. Orfadin® (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic by-products in the body. In the most common form of the disease, symptoms arise within the first six months of the child's life. Patients must maintain a special diet in combination with Orfadin treatment as tyrosine is not adequately broken down. Approximately 1,000 persons are identified as living with HT-1 today. Orfadin is a proprietary product and is developed by and marketed globally by Sobi.
About Sobi(TM)
Sobi is an international specialty healthcare company dedicated to rare diseases. Sobi's mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primarily focused on Haemophilia, Inflammation and Genetic diseases. Sobi also markets a portfolio of specialty and rare disease products for partner companies across Europe, the Middle East, North Africa and Russia. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2014, Sobi had total revenues of SEK 2.6 billion (USD 380 M) and about 600 employees. The share (STO: SOBI) is listed on NASDAQ OMX Stockholm. More information is available at www.sobi.com.
------------------------------------------------------
产地国家： 美国 
原产地英文商品名:
Orfadin oral suspension 90ml 4mg/ml/bottle 
原产地英文药品名:
nitisinone
中文参考商品译名:
Orfadin口服悬液 90毫升 4毫克/5毫升/瓶
中文参考药品译名:
尼替西农
生产厂家中文参考译名:
Sobi Inc
生产厂家英文名:
Sobi Inc