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美国FDA批准Nuwiq用于治疗成年及儿童A型血友病

2015-09-22 02:31:59 作者：新特药房来源：互联网浏览次数：2 文字大小：【大】【中】【小】

简介： 美国FDA批准Octapharma静脉注射药物Nuwiq用于血友病A成年及儿童患者。Nuwiq的批准包括按需治疗及出血发作控制；用于减少出血发作频次的常规预防；及出血的围术期管理。Nuwiq是源于人细胞系的首个B-结构域 ...

关键字：抗血友病因子(重组) simoktokog alfa 商品名nuwiq 血友病治疗或预防治疗

美国FDA批准Octapharma静脉注射药物Nuwiq用于血友病A成年及儿童患者。Nuwiq的批准包括按需治疗及出血发作控制；用于减少出血发作频次的常规预防；及出血的围术期管理。
Nuwiq是源于人细胞系的首个B-结构域缺失重组因子VIII（FVIII），它未进行化学修饰或与其它蛋白融合，这款药物旨在治疗先天性FVIII缺乏的血友病A患者。“Octapharma多年来一直致力于出血性疾病领域，其解决血友病A挑战的努力十年来从未动摇，”OctapharmaUSA总裁Nielsen称。
“在Nuwiq的开发中，早期开发战略是完整的，随着FDA的批准，这些初步目标已经实现。Nuwiq在全球临床试验中已证明安全性及有效性，在未来的岁月里，它有可能积极地影响到患者的生活质量。Octapharma致力于为血友病A提供能够提高与拯救生命的治疗药物，公司期望将Nuwiq带到美国市场。”
Nuwiq去年获得欧盟委员会批准
欧盟委员会于2014年8月率先批准这款治疗药物。Nuwiq目前已在许多国家获得批准，包括英国、澳大利亚、加拿大、德国、意大利、瑞典及阿根廷。
“我们很高兴用于血友病A成年及儿童患者的治疗选择继续向前推进，有越来越多的治疗药物能够在美国获得批准，”美国国家血友病基金会首席执行官Bias称。“为出血疾病社区开发提高生命产品的持续努力绝对是至关重要的。为患者及供应商提供治疗选择、教育及支持项目，对患有血友病A的患者来说是极其重要的。”
Nuwiq® (human coagulation factor VIII recombinant DNA, simoctocog alfa)
Human coagulation factor VIII (rDNA), simoctocog alfa is a purified protein. Nuwiq® is produced by recombinant DNA technology in genetically modified human embryonic kidney (HEK) cells. No animal or human derived materials are added during the manufacturing process or to the final medicinal product.
Powder and solvent for solution for injection available in vials of
•250 IU
•500 IU
•1000 IU
•2000 IU
New Drugs Online Report for simoctocog alfa
Information
Generic Name: simoctocog alfa
Trade Name: Nuwiq
Entry Type: New molecular entity
Development and Regulatory status
UK: Launched
EU: Launched
US: Approved (Licensed)
UK launch Plans: Available only to registered users
Actual UK launch date: October 2014
Comments
Sept 15: US FDA has approved Nuwiq. Octapharma USA aims to have Nuwiq available in the U.S. marketplace by early 2016 [6].
16/09/2015 09:59:10
Nov 14: Net price: 76p per IU [5]
01/12/2014 14:09:50
Oct 14: Launched in the UK [4].
22/10/2014 14:01:03
Jul 14: Approved in the EU [3].
22/10/2014 13:45:33
May 14: EU positive opinion [1].
09/06/2014 17:16:17
Trial or other data
May 14: The benefit with Nuwiq in terms of prevention and treatment of bleedings and haemostatic efficacy for surgical procedures was studied in 3 pivotal trials; study GENA-01 in 22 adolescent and adult subjects where overall, 94.4% of the bleeding episodes (BEs) were treated on-demand with excellent or good efficacy and a large majority of BEs required only 1 infusion; study GENA-08 investigated prophylaxis of bleeding events and the treatment of break-through bleeds in 32 adults, where the mean bleeding rates per patient during the prophylactic treatment period were 0.188/month for all types of bleeds. In study GENA-03 in 59 paediatric subjects between 2 and 11 years of age, the mean rate of all BEs in prophylaxis was 0.338 BEs/month; the monthly rate of all BEs was lower in patients aged 2 to 5 than in those aged 6 to 12 years (0.213 BEs/month and 0.459 BEs/month, respectively). In this study 68.6% of break-through bleeds were treated with one infusion and 81.3% with one or 2 infusions. No side effects were commonly reported in the safety database of 135 previously treated patients. The immunogenicity of Nuwiq was evaluated in clinical trials in 135 previously treated patients with severe haemophilia A (74 adult and 61 paediatric patients). None of the patients developed inhibitors [1].
09/06/2014 17:18:59
Evidence Based Evaluations
EPAR http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/002813/WC500179342.pdf
References
Available only to registered users
Category
BNF Category: Antifibrinolytic drugs and haemostatics (02.11)
Pharmacology: A recombinant blood coagulation factor VIII
Epidemiology: Haemophilia A is caused by factor VIII deficiency and occurs in 1 in 5,000 to 1 in 10,000 male live births. Between 50% and 70% of people with haemophilia A have severe disease (<1% factor VIII activity). There were 5,424 patients with haemophilia A in the UK in 2011 (prevalence 8.7 per 100,000 population), 1,057 were aged 0-13 years [2].
Indication: Haemophilia A
Additional Details: treatment & prophylaxis of bleeding in all age groups
Method(s) of Administration
Intravenous
Company Information
Name: Octapharma
US Name: Not Known
Further Information
Anticipated commissioning route (England) NHSE
High cost drug list? Awaiting Update
Implications Available only to registered users