| 瑞士罕见病制药商Swedish Orphan Biovitrum(SOBI)口服悬液配方Orfadin(nitisinone，尼替西农)近日喜获欧盟批准，用于遗传性酪氨酸血症I型 (Hereditary Tyrosinaemia type-1，HT-1)患者的治疗。HT-1是一种罕见的常染色体隐性遗传病，是由酪氨酸代谢障碍所致的一种遗传代谢病，多数患儿为急性型，起病于新生儿或婴儿期，主要累及肝脏和肾脏，若不治疗会导致肝、肾衰竭甚至死亡。 胶囊配方Orfadin于2005年上市，目前已获欧盟及全球多个国家批准，联合酪氨酸和苯丙氨酸饮食限制，用于确诊为HT-1的患者。Orfadin结合饮食限制及广泛的新生儿筛查，能够使婴幼儿及早确诊治疗，从而显著改善患者的临床结果。而在Orfadin上市前，出生2个月内确诊为HT-1的婴儿，仅有三分之一能够存活到2岁。目前全球大约有1000例HT-1患者。 Orfadin口服悬液是一种婴幼儿友好的配方，可方便准确给药剂量，有助于改善患者及其照料者的生活质量。SOBI公司已计划在2015年下半年将Orfadin口服混悬液推向整个欧盟市场。 遗传性酪氨酸血症I型 (HT-1)是由于酪氨酸代谢过程的终末酶延胡索酰乙酰乙酸水解酶(FAH)编码基因发生突变以致FAH活性降低或缺失，酪氨酸分解代谢发生障碍，中间代谢产物如马来酰乙酰乙酸、延胡索酰乙酰乙酸、琥珀酰乙酰乙酸及琥珀酰丙酮等有毒副产物在体内蓄积，造成机体损伤，导致患儿肝功能衰竭、肾功能不全和神经系统并发症，症状在婴儿出生6个月内出现。 Orfadin(nitisinone)能够阻断酪氨酸的分解，从而减少体内有毒副产物的量。 New Drugs Online Report for nitisinone Information Generic Name: nitisinone Trade Name: Orfadin Entry Type: Licence extension / variation Development and Regulatory status UK: Phase III Clinical Trials EU: Phase III Clinical Trials US: Phase III Clinical Trials UK launch Plans: Available only to registered users Actual UK launch date: Comments Granted orphan drug status in EU (EU/3/02/096) in 2002 [1]. 06/08/2013 09:18:03 Trial or other data Jan 16: PIII SONIA 2 study (NCT01916382 ) is ongoing and has finished recruiting pts. Collection of primary outcome data is now expected to complete Feb 16 [4]. 04/02/2016 10:57:39 Aug 13: NCT01916382 (SONIA 2) is an international, multicentre, randomised, evaluator-blind, no-treatment controlled PIII study to assess the efficacy and safety of once daily nitisinone in 140 patients with alkaptonuria after 12 months of treatment, followed by an additional 36 month treatment period. The primary outcome is 24 hour urine homogentisic acid at 1 year. In clusion criteria include age ≥25 years and a diagnosis of AKU) with clinical symptoms. The study starts Nov 13 and is due to complete Apr 18 (primary outcome data collection Apr 15). The study is being run by the University of Liverpool [2]. 06/08/2013 09:18:41 References Available only to registered users Category BNF Category: Drugs used in metabolic disorders (09.08.01) Pharmacology: Homogentisic acid lowering drug via enzyme inhibition Epidemiology: Alkaptonuria (AKU) is a rare disease, which causes severe, early-onset osteoarthritis. AKU patients are born with the disease. However, with the exception of black urine, none of the symptoms present until patients reach their 20s. AKU is a rare disease with an estimated frequency of one in 250,000 to one in 500,000. In the UK, there are currently only 64 patients [3]. Indication: Metabolic disease Additional Details: alkaptonuria (AKU) Method(s) of Administration Oral Company Information Name: Swedish Orphan Biovitrum (Sobi) US Name: Swedish Orphan Biovitrum (Sobi) Further Information Anticipated commissioning route (England) NHSE High cost drug list? Yes Implications Available only to registered users |
新型口服悬液配方Orfadin（nitisinone）获欧盟批准简介：
瑞士罕见病制药商Swedish Orphan Biovitrum(SOBI)口服悬液配方Orfadin(nitisinone，尼替西农)近日喜获欧盟批准，用于遗传性酪氨酸血症I型 (Hereditary Tyrosinaemia type-1，HT-1)患者的治疗。HT-1是一种 ... 责任编辑：admin
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