美国FDA批准Obizur[抗血友病因子（重组体），猪序列]用于获得性A型血友病（获得性因子VIII [FVIII]缺乏）成人患者出血发作治疗。
获得性A型血友病是一种罕见但潜在危及生命的出血疾病，由体内产生直接对抗人体自身FVIII的抗体（免疫系统蛋白）引起，因子FVIII是一种对血液凝结非常重要的蛋白。当FVIII被这些抗体灭活时，人的血液就不能正常凝结，导致失血过多，这种失血可自然而然发生或出现在损伤或手术后。
与遗传性血友病不一样，获得性A型血友病不是一种遗传性疾病，它对男女均有影响。获得性A型血友病的发展与其它条件或健康状态相关，如妊娠、癌症或某些药物使用。然而，在大约一半的病例中，找不到根本病因。这种疾病的诊断可能比较困难，出血的严重性可能会使治疗面临挑战。
“这款产品的批准为患有这种罕见疾病的患者提供了一种重要的治疗选择，”FDA生物制剂评价与研究中心主任、医学博士Midthun称。Obizur含有一种重组猪FVIII类似物。使用猪FVIII是因为它足以与人FVIII相似，可以在血液凝结中产生效果，但它不太可能受到获得性A型血友病患者体内存在的对抗人FVIII抗体的影响。
Obizur的安全性及有效性在有29名获得性A型血友病患者参与的一项临床试验中得到评价，试验中这些患者接受Obizur治疗其严重出血发作。试验证明了Obizur在出血发作治疗中的有效性。试验未证实有安全性问题。Obizur获得FDA孤儿药资格，因为这款药物旨在用于一种罕见疾病或病症治疗。
New Drugs Online Report for susoctocog alfa
Information
Generic Name: susoctocog alfa
Trade Name: Obizur
Synonym: OBI 1, recombinant porcine factor VIII
Entry Type: New molecular entity
Developmental Status
UK: Pre-registration (Filed)
EU: Pre-registration (Filed)
US: Approved (Licensed)
UK launch Plans: Available only to registered users
Actual UK launch date:
Comments
Oct 14: The FDA has approved Obizur for the treatment of bleeding episodes in adults with acquired hemophilia A. [10]
27/10/2014 10:07:16
Jul 14: Filed in the EU via the centralised procedure [9].
17/09/2014 12:13:29
Dec 13: Filed in the US for treatment of patients with acquired haemophilia A [8].
11/12/2013 21:56:11
Nov 12: FDA assigns fast track status to OBI-1 in acquired haemophilia A [5].
22/11/2012 09:26:15
Nov 10: PIII study started [2].
23/11/2010 09:27:24
Granted Orphan drug status in the US (2004) and the EU (2010) [1]
23/11/2010 09:27:06
Trial or other data
Dec 13: Results reported from the global, PII/III open label trial in 18 adults with acquired hemophilia A who presented with a serious bleed and were treated with an initial dose of OBI-1 (200 units/kg), followed by additional doses based on clinical eva luation and target factor VIII activity levels. The primary efficacy endpoint was defined by clinical assessment as effective or partially effective control of bleeding and FVIII activity levels at 24 hours after initiation of OBI-1 therapy. All patients in the study responded positively (14 effective / 4 partially effective) in the first 24 hours. No treatment-related serious adverse events were reported; non-serious mild AEs were reported in two patients (11.1%) who developed anti-porcine inhibitors to OBI-1 [8]
11/12/2013 22:01:03
Jan 13: Inspiration and Ipsen have agreed to sell OBI-1 (recombinant porcine FVIII) to Baxter International [6].
26/02/2013 10:27:31
July 12: Data from ongoing Phase 2/3 Accur8 Auto-antibody trial (n=15 to date). . The primary objective of the study is to eva luate the efficacy of OBI-1 treatment (200 units/kg on first injection, then dose based on target FVIII levels) for serious (life- or limb-threatening) bleeds in individuals age 18 and older with acquired haemophilia A, as measured by the control of bleeding 24 hrs after initial OBI-1 dose administration. Seven out of seven pts receiving OBI-1 experienced control of bleeds within 24 hrs and subsequent resolution of bleeds. Therapeutic FVIII activity levels were achieved and maintained with intermittent OBI-1 administration based on FVIII levels. [4]
10/07/2012 09:38:25
Nov 11: The second of two pivotal studies from OBI-1´s Accur8 clinical trial programme has started (NCT01434511). It will enrol 28 children ≥6 years of age with congenital hemophilia A, who have developed inhibitory antibodies to their human FVIII replacement therapy. OBI-1 will be given intravenously for 2-3 hourly during the first 24 hours of treatment. The study is being conducted in S. Africa and is due to complete Sep 14 [3].
29/11/2011 11:12:07
Nov 10: Inspiration Biopharmaceuticals has started the first of two PIII studies of OBI-1. It is a prospective, non-randomized, open-label study eva luating the efficacy of OBI-1 for the treatment of serious bleeding episodes in individuals with acquired hemophilia. Serious bleeding episodes include those that are a threat to a patient´s life or vital organs or limbs, or which require a blood transfusion. In addition, the study will obtain data about the pharmacokinetic behavior of OBI-1 [2].
23/11/2010 09:40:40
Jan 10: Inspiration Biopharmaceuticals has in-licensed OBI-1 from Ipsen will be responsible for the clinical development, regulatory process and commercialization of the product [2].
23/11/2010 09:40:19
Approximately one-third of individuals with hemophilia A develop an immune reaction to human FVIII (hFVIII). Current therapies, specifically human factor VIIa (NovoSeven®) and FEIBA, work by bypassing the natural hemostatic pathway and forcing coagulation with much higher levels of FVIIa than normal. OBI-1 is a recombinant form of porcine FVIII that possesses low cross reactivity to anti-hFVIII antibodies. OBI-1 should allow clinicians to correlate activity and efficacy with a biomarker and guide dosing to better predict treatment outcomes [2].
23/11/2010 09:40:08
References
Available only to registered users
Category
BNF Category: Drugs used in hypoplastic, haemolytic, and renal anaemias (09.01.03)
Pharmacology: recombinant porcine factor VIII (FVIII)
Epidemiology: Acquired hemophilia, an autoimmune disease, is rare and is typically a disorder of older adults, both males and females. In acquired hemophilia, individuals typically bleed into the skin, muscles and soft tissues [2]. There were 5,424 patients with haemophilia A in the UK in 2011 (preva lence 8.7 per 100,000 population) [7]
Indication: Haemophilia A
Additional Details:
Method(s) of Administration
Intravenous infusion
Company Information
Name: Baxter
US Name: Baxter
NICE Information
In timetable: - |
