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欧盟批准Obizur治疗成年血友病患者

2016-03-28 10:53:10 作者：新特药房来源：互联网浏览次数：1 文字大小：【大】【中】【小】

简介： 2015年11月16日，欧盟委员会对Baxalta旗下Obizur在因子VIII（FVIII）抗体引起的获得性血友病成年患者中用于出血发作治疗授予上市许可。Obizur 是首款在欧洲上市的重组猪序列 FVIII 治疗药物，用于治疗获 ...

关键字：猪抗血友病因子(重组) 商品名obizur 血友病罕见形式新治疗孤儿药物指定

2015年11月16日，欧盟委员会对Baxalta旗下Obizur在因子VIII（FVIII）抗体引起的获得性血友病成年患者中用于出血发作治疗授予上市许可。Obizur 是首款在欧洲上市的重组猪序列 FVIII 治疗药物，用于治疗获得性血友病A，其旨在使医师除了临床评价之外能够通过检测FVIII活性水平来监测治疗响应。
在欧洲授予该首款重组猪序列FVIII选择用于获得性血友病反映了Baxalta改善患者生活的承诺，Baxalta执行副总裁、血液病学总裁Goff称。我们继续建立一个广泛的、全球性的、旨在降低出血疾病患者负担的治疗产品组合，因为我们寻求一个没有出血疾病的世界。
Obizur的获批基于一项2/3期试验的结果
此次批准基于一项2/3期开放式试验的阳性结果，该试验在获得性血友病A成年患者中检测了Obizur用于严重出血发作治疗的有效性与安全性。所有以Obizur治疗的患者显示有阳性响应（出血停止或降低）及临床改善，在初始输注后24小时，FVIII活性水平达到20%或更高。
在86%的患者中可以观察到初步出血发作的成功控制，而在Obizur作为一线治疗的患者中，有94%的患者可以观察到初步出血发作成功控制。猪序列因子VIII抗体的发生是逾5%患者报道的唯一副作用。OBIZUR目前已在美国、加拿大及欧洲获得批准，其在瑞士、澳大利亚及哥伦比亚的上市申请正在审评中
Baxalta (BXLT) Gains EU Marketing Authorization To Provide OBIZUR To Adult Patients With Acquired Hemophilia A
European Commission has granted Marketing Authorization for OBIZUR [Antihaemophilic Factor (Recombinant), Porcine Sequence].
OBIZUR is indicated for the treatment of bleeding episodes in adult patients with acquired haemophilia caused by antibodies to Factor VIII (FVIII), a very rare and potentially life-threatening acute bleeding disorder. OBIZUR is the first recombinant porcine sequence FVIII treatment available in Europe to treat acquired haemophilia A, and is designed to enable physicians to monitor treatment response by measuring FVIII activity levels in addition to clinical assessments.
“Gaining Marketing Authorization for this first recombinant porcine option for acquired haemophilia in Europe reflects Baxalta’s commitment to improving patient lives,” said Brian Goff, executive vice president and president, Haematology, Baxalta. “We continue to build a broad, global portfolio of treatments that aim to reduce the burden of bleeding disorders for patients as we pursue a world without bleeds.”
The approval is supported by the positive results of a global, prospective, controlled, multi-centre Phase II/III open-label clinical trial that examined the efficacy and safety of OBIZUR in the treatment of serious bleeding episodes in adults with acquired haemophilia A (29 patients evaluated for safety, 28 evaluated for efficacy). All patients treated with OBIZUR (28/28) showed a positive response (bleeding stopped or reduced) and clinical improvement, with FVIII activity levels at 20 percent or higher at 24 hours after the initial infusion. Successful control (resolution) of the initial bleeding episode was observed in 86 percent (24/28) of all patients and in 94 percent (16/17) of those treated with OBIZUR as the first-line treatment. The development of antibodies to porcine factor VIII was the only adverse reaction reported in more than five percent of patients.
Acquired haemophilia A is a rare haemorrhagic disease in which autoantibodies against coagulation factor VIII, called FVIII-neutralizing antibodies (inhibitors), weaken the inherent coagulation system. As the inhibitors developed in AHA are autoantibodies, the disease may be caused by and is often associated with autoimmune disease. Acute haemorrhage associated with AHA may be fatal, however AHA is often unrecognized or misdiagnosed and should thus be considered in the differential diagnosis particularly in postpartum women and the elderly with bleeding tendency or prolonged activated partial thromboplastin time.
About OBIZUR in Europe
OBIZUR [Antihaemophilic Factor (Recombinant), Porcine Sequence] is indicated for the treatment of bleeding episodes in adults with acquired haemophilia caused by antibodies to Factor VIII.
OBIZUR is now approved in the United States, Canada and Europe, and is under regulatory review in Switzerland, Australia and Colombia.
Important Risk Information for OBIZUR
This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.
CONTRAINDICATIONS
OBIZUR should not be used in patients with known anaphylactic reactions to the active substance, hamster protein, or to any of the excipients.
WARNINGS & PRECAUTIONS
Hypersensitivity
Allergic type hypersensitivity reactions are possible with OBIZUR. The product contains trace amounts of hamster proteins.
If symptoms of hypersensitivity occur, patients should be advised to discontinue use of the medicinal product immediately and contact their physician. Patients should be informed of the early signs of hypersensitivity reactions including hives, generalised urticaria, and tightness of the chest, wheezing, hypotension, and anaphylaxis.
In case of shock, standard medical treatment for shock should be implemented.
Development of inhibitory antibodies
Inhibitory antibodies against porcine Factor VIII (measured using a modification of the Nijmegen variation of the Bethesda assay) were detected before and after exposure to OBIZUR. Inhibitor titres of up to 29 Bethesda units were recorded at baseline yet subjects responded positively to OBIZUR. It is recommended that treatment should be based on clinical judgement and not based on detection of inhibitory antibodies by the Bethesda assay.
There is a lack of clinical information on the development of inhibitory antibodies to OBIZUR following repeated administration. Therefore, OBIZUR must only be administered when considered clinically necessary. Extensive cutaneous purpura do not necessarily require treatment.
OBIZUR is produced by recombinant DNA technology in baby hamster kidney cells. Antibodies to baby hamster kidney cell protein were not detected in subjects either before or after exposure to OBIZUR.
High and sustained Factor VIII activity in blood may predispose to thromboembolic events. Those with pre-existing cardiovascular disease and the elderly are at particular risk.
If venous catheterization is required, the risk of catheter-related complications such as catheter site thrombosis should be considered.
Factor VIII activity determined by the chromogenic assay is generally lower than Factor VIII activity determined by the one-stage clotting assay. Measurement of Factor VIII activity must always be carried out using the same assay methodology on any one patient. The one-stage assay is recommended because it has been used in determination of the potency and the mean recovery rate of OBIZUR.
Sodium content
Each vial contains 4.4 mg (198 mM) sodium per ml of reconstituted solution. To be taken into consideration by patients on a controlled sodium diet.
ADVERSE REACTIONS
Common adverse reactions observed in greater than 5% of subjects in the clinical trial were development of inhibitors to porcine factor VIII.
About Acquired Haemophilia A
Acquired haemophilia A is a rare, potentially life-threatening bleeding disorder, which, unlike congenital haemophilia, typically affects older adults and occurs in both males and females.3,4 In acquired haemophilia A, individuals typically experience subcutaneous, soft tissue, and post-surgical bleeding.4,5,6 The comorbidities in this typically elderly population also pose a particular challenge to treat serious bleeding episodes.3