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美国FDA批准Adynovate用于治疗A型血友病

2015-11-14 11:54:49 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： Adynovate[Antihemophilic Factor (Recombinant), Pegylated]为血友病患者提供一种重要的新治疗选择2015年11月13日，美国食品和药品监管局(FDA)批准Adynovate，抗血友病因子(重组)，PEG化在成年和青少年 ...

关键字：重组peg化抗血友病商品名adynovate 血友病a预防治疗

Adynovate[Antihemophilic Factor (Recombinant), Pegylated]为血友病患者提供一种重要的新治疗选择
2015年11月13日，美国食品和药品监管局(FDA)批准Adynovate，抗血友病因子(重组)，PEG化在成年和青少年，年龄12 岁和以上，血友病A使用。 Adynovate被修饰在血中持续更长时间和潜在地需要较少频繁注射比未修饰的抗血友病因子当用于减低出血频数时。
Adynovate被批准为按需要治疗和控制出血发作和以减低出血发作的频数(预防)在有血友病A患者中。Adynovate由全长凝血因子VIII分子(历史上被称为抗血友病因子)连接至其他分子，被称为聚乙二醇(PEG化)组成。这个连接使产品在患者血中持续更长时间。
FDA的生物制品评价和研究中心主任Karen Midthun，M.D.说：“Adynovate的批准提供为有血友病A患者医疗护理中使用一个重要的治疗选择和减少因子VIII输注需要频数以避免出血。”
血友病A是一种遗传，性-关联，血-凝固疾病主要地影响男性，它是因子VIII基因中发现缺陷所致。根据美国疾病控制和预防中心，在美国血友病A每5,000出生男性受影响一人。有血友病A 患者可能经受反复严重出血发作，主要地至关节，作为结国关节被严重损伤。
在137例成年和青少年年龄12岁和以上一项临床试验，试验比较推荐的常规预防治疗方案与按需要治疗评价Adynovate安全性和疗效。试验证实在常规医疗护理中Adynovate是有效地减少出血发作数量。此外，Adynovate在治疗和控制出血发作有效。试验期间未确定安全性担忧。
Adynovate是总部在加州Westlake Village的Baxalta美国有限公司制造。
New Drugs Online Report for rurioctocog alfa pegol
Information
Generic Name: rurioctocog alfa pegol
Trade Name: Adynovate
Synonym: BAX855; factor VIII recombinant PEGylated
Entry Type: New formulation
Development and Regulatory status
UK: Phase III Clinical Trials
EU: Phase III Clinical Trials
US: Pre-registration (Filed)
UK launch Plans: Available only to registered users
Actual UK launch date:
Comments
Jul 15: Remains PIII for use during surgery, PK guided, in paediatrics and continuation use [9].
03/09/2015 14:26:08
Dec 14: Baxter plans to file in Europe in 2016 after an on-going paediatric study is complete [5].
02/12/2014 10:08:39
Dec 14: Baxter submitted a biologics license application for US FDA approval Of BAX 855, extended half-life recombinant FVIII for Hemophilia A - the application is based on the ADVATE study (n=138) [4].
02/12/2014 09:34:27
Aug 14: Baxter plans to file in the US by end 2014 and later, after completion of the paediatric study, in the EU [3].
21/08/2014 20:38:22
Nov 13: Company expects to file for approval late in 2014 [2]
18/11/2013 15:39:59
Trial or other data
Jul 15: Results of BAX 855 trials published in blood journal [8].
20/07/2015 15:13:43
Jul 15: Baxalta announces positive data. Patients in the twice-weekly arm of the trial demonstrated a 95% reduction in median annualised bleed rate compared with the on-demand arm (1.9 vs. 41.5, respectively). BAX 855 was also effective in treating all bleeding episodes, 95.9% of which were controlled with one or two infusions at a median dose of 29.0 IU/kg per infusion [7].
17/07/2015 10:28:43
Aug 14: In top-line results from a global 6 month PIII study (n=138) BAX 855 met its primary endpoint in the control and prevention of bleeding, routine prophylaxis and perioperative management for patients ≥ 12 years. Patients on twice-weekly prophylaxis arm had a 95% reduction in median annualized bleed rates (ABR) vs those in the on-demand arm (1.9 vs. 41.5, respectively). BAX 855 was also effective in treating bleeding episodes, 96% of which were controlled with one or two infusions. The half-life of BAX 855 was 1.4 - 1.5 times that of ADVATE. No patients developed inhibitors to BAX 855 and no treatment-related serious AEs, including hypersensitivity, were reported [3].
21/08/2014 20:38:36
NCT01913405 is a PIII, multi-centre, open label study of efficacy and safety of PEGylated rFVIII (BAX 855) in 30 previously treated patients with severe Haemophilia A undergoing surgical or other invasive procedures. The primary outcome is Global Hemostatic Efficacy Assessment score (GHEA). the pre-operative loading dose and any intra-operative and post-operative dosing will be based on individual PK results as well as target trough level for type of surgery. The study started Sep 13 and is due to complete Dec 16 [1]
18/11/2013 15:38:27
NCT01736475 (PROLONG-ATE) is a PII/III, multi-center, open label study of efficacy, safety, and pharmacokinetics of PEGylated recombinant Factor VIII (BAX 855) administered for prophylaxis and treatment of bleeding in 146 previously treated patients (aged 12-65 years) with severe Haemophilia A. Patients will receive treatment twice weekly (45 IU/kg) and followed for 6 months. The primary outcome is annualized bleeding rate. The study started Jun 13 and is due to complete Sep 14. Baxter is also initiating a continuation study (NCT01945593) [1].
18/11/2013 15:38:11
References
Available only to registered users
Category
BNF Category: Antifibrinolytic drugs and haemostatics (02.11)
Pharmacology: Full-length recombinant factor VIII conjugated to polyethylene glycol
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.
Indication: Haemophilia A
Additional Details: severe in adults and children from 0 to 75 years, Second or subsequent line, for treatment and prevention of bleeding, routine prophylaxis and perioperative bleeding
Method(s) of Administration
Intravenous
Company Information
Name: Baxalta
US Name: Baxalta
Further Information
Anticipated commissioning route (England) NHSE
High cost drug list? Yes
Implications Available only to registered users