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FDA批准Vonvendi（重组因子）为首个治疗血管性血友病

2015-12-11 01:42:20 作者：佚名来源：互联网浏览次数：0 文字大小：【大】【中】【小】

简介： Vonvendi (von Willebrand factor [recombinant]) -血管性血友病首选治疗药2015年12月8日,美国药品和食品监管局(FDA)批准Vonvendi，von Willebrand因子(重组)，为在18岁和以上有von Willebrand病(VWD)成年 ...

关键字：重组vonwillebrand因商品名vonvendi vmd 成年按需治疗孤儿产品指定

Vonvendi (von Willebrand factor [recombinant]) -血管性血友病首选治疗药
2015年12月8日,美国药品和食品监管局(FDA)批准Vonvendi，von Willebrand因子(重组)，为在18岁和以上有von Willebrand病(VWD)成年中使用。Vonvendi是第一个FDA-批准的重组von Willebrand因子，和是批准为按需治疗和在被诊断有VWD成年中控制出血发作。
VWD是最常见遗传出血病，影响美国人群接近1 %。男性和妇女是等同地受VWD影响，它是被von Willebrand因子，一种对正常凝血至关重要蛋白缺乏或缺陷所致。有VWD患者可能发生来自鼻，牙龈，和肠严重出血，以及至肌肉和关节。有VWD妇女可能有月经量过多持续较长于平均和小儿出生后过度出血。
FDA生物制品评价和研究中心主任Karen Midthun，M.D.：说 “有遗传性出血疾病患者应与卫生保健提供者商讨减低失血适当措施，” “Vonvendi的批准为有von Willebrand病患者中出血发作的治疗提供另外治疗选择。”
在69例有VWD成年参加者两项临床试验评价Vonvendi的安全性和疗效。这些试验证实 Vonvendi对按需治疗和来自机体不同部位出血发作的控制是安全和有效在试验中无确定的安全性担忧。观察到最常见不良反应是全身瘙痒。
FDA授予Vonvendi对这些使用孤儿产品指定。孤儿产品指定是给予药物意向治疗罕见病为了促进它们的发展。
Vonvendi由总部在加州Westlake Village的Baxalta美国有限公司制造。
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm476065.htm
New Drugs Online Report for von Willebrand factor, recombinant
Information
Generic Name: von Willebrand factor, recombinant
Trade Name: Vonvendi
Synonym: BAX 111, rVWF
Entry Type: New molecular entity
Development and Regulatory status
UK: Phase III Clinical Trials
EU: Phase III Clinical Trials
US: Approved (Licensed)
UK launch Plans: Available only to registered users
Actual UK launch date:
Comments
Dec 15. Approved in US [8].
09/12/2015 11:58:53
Jul 15: Filed for on-demand use in US; PIII for use during surgery [7].
03/09/2015 14:24:15
Dec 14. Baxter submits application for FDA approval of BAX111 for the treatment of Von Willebrand Disease. [5]
24/12/2014 11:32:30
Apr 14: Baxter plans to file for approval before end of 2014 and intends to pursue a study of BAX 111 in a prophylaxis treatment setting before the end of the year [4].
17/04/2014 09:02:40
Designated orphan drug status in US in Nov 10 [4].
17/04/2014 09:02:28
Designated orphan drug status in EU in Nov 10 [2].
14/10/2011 09:17:14
Sep 11: PIII study started [1].
14/10/2011 09:13:21
Trial or other data
Aug 15: PIII data published in Blood shows BAX 111 successfully managed bleeding episodes for patients with von Willebrand disease. The treatment met its goals by helping all of the trial´s 37 patients stem bleeding when used on demand, Baxalta said, and one infusion was enough to control about 82% of bleeds [6].
12/08/2015 11:14:40
Apr 14: Results reported from the PIII study. The study of BAX 111 met its primary efficacy endpoint, as all patients achieved pre-specified success in the on-demand treatment of bleeding events (100%, 22 of 22 patients who experienced bleeds in the trial). There were no reports of inhibitor development or thrombotic events. The most common AEs were headache, vomiting/nausea and iron deficiency anaemia, which were not considered to be related to treatment. There was one serious adverse event related to treatment, characterized by chest discomfort and increased heart rate during infusion, which rapidly resolved without further complication [4].
17/04/2014 09:02:48
Mar 13: Estimated NCT01410227 completion Date March 2014 [3]
04/04/2013 12:28:44
Oct 11: NCT01410227 is a non-randomised, crossover PIII study assessing efficacy and safety of recombinant von Willebrand factor (rVWF) for treating and preventing bleeding episodes in 32 subjects with von Willebrand Disease (VWD). The primary outcome is the number of subjects with treatment success (defined as a mean efficacy rating score of <2.5 for a subject´s bleeding within the 12 month treatment period. Scores used to assess the extent of control of the bleeding episodes: Excellent =1, Good =2, Moderate =3, None =4.) Subjects will be treated with 50 or 80IU/kg rVWF IV – with or without rFVIII (ADVATE). Subjects must have been diagnosed with type 3 VWD (VWD:Ag ≤3 IU/dL) or severe non-type 3 VWD (VWF:RCo <20 IU/dL) or type 2N VWD (FVIII:C <10% and documented genetics) and have had a minimum of 6 documented bleeds requiring VWF coagulation factor replacement therapy during the previous 2 years. The study started in Sep 11 and is due to complete Feb 13 [1].
14/10/2011 09:13:36
References
Available only to registered users
Category
BNF Category: Anaemias and some other blood disorders (09.01)
Pharmacology: Blood-coagulation-factor
Epidemiology: Affects <2 in 10,000 people in the EU - fewer than 101,000 people in total [2].
Indication: von Willebrand disease
Method(s) of Administration
Intravenous
Company Information
Name: Baxalta
US Name: Baxalta
Further Information
Anticipated commissioning route (England) NHSE
High cost drug list? Awaiting Update
Implications Available only to registered users