10月14日，美国食品药品管理局（FDA）发布公告称，已批准奥贝安可(Ferriprox，通用名：去铁酮)用于治疗因输血所致铁超负荷的地中海贫血患者，这些患者对以往的铁超负荷螯合治疗反应不佳。
地中海贫血患者因频繁接受输血而导致体内铁过多（输血性铁超负荷），这种状态后果严重甚至可能是致命的。可能导致患者产生患肝脏疾病、糖尿病、关节炎、心衰或心律失常的危险。
输血性铁超负荷的标准治疗是螯合治疗，即用化学药物去除体内的重金属。而去铁酮则在螯合治疗效果不佳时应用。
“去铁酮是自2005年以来FDA批准的首个治疗上述疾患的新药”，FDA药物研究与评价中心血液病和肿瘤产品办公室主任理查（Richard Pazdur）说。
去铁酮的安全性和有效性基于对12项纳入236例患者临床研究数据的分析。所有参试的患者对既往的铁螯合治疗无反应，而去铁酮可成功治疗血清铁蛋白下降20%以上的患者。在上述研究中，半数患者的铁蛋白水平下降20%以上。
在接受去铁酮治疗的患者中，最常见的副作用包括恶心、呕吐、腹痛、关节疼痛、尿变色（色素尿）、白细胞计数下降和肝酶水平升高。2%的病人有最严重的副作用，即患粒细胞缺乏症。
该药通过FDA的快速批准程序获准。
Manufacturer:
ApoPharma USA Inc.
Pharmacological Class:
Iron chelating agent.
Active Ingredient(s):
Deferiprone 500mg; scored tabs.
Indication(s):
Treatment of transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate.
Pharmacology:
Deferiprone is a synthetic, orally-active chelating agent with an affinity for ferric iron (iron III). Deferiprone binds with ferric ions to form neutral 3:1 (deferiprone:iron) complexes that are stable over a wide range of pH values. Deferiprone has a lower binding affinity for other metals (eg, copper, aluminum, zinc) than for iron.
Clinical Trials:
In a prospective, planned, pooled analysis of patients from several studies, the efficacy of Ferriprox was assessed in transfusion-dependent iron overload patients in whom previous iron chelation therapy had failed or was considered inadequate due to poor tolerance. The main criterion for chelation failure was serum ferritin >2,500mcg/L before treatment with Ferriprox. Ferriprox therapy (35–99mg/kg/day) was considered successful in individual patients who experienced a ≥20% decline in serum ferritin within one year of starting therapy.
Data from a total of 236 patients were analyzed. Of the 224 patients with thalassemia who received deferiprone monotherapy and were eligible for serum ferritin analysis, 105 (47%) were male and 119 (53%) were female. The mean age of these patients was 18.2 years. For the patients in the analysis, the endpoint of at least a 20% reduction in serum ferritin was met in 50% (of 236 subjects), with a 95% confidence interval of 43% to 57%.
Legal Classification:
Rx
Adults:
Individualize. Initially 25mg/kg three times daily (total dose 75mg/kg/day). Max: 33mg/kg three times daily (total dose 99mg/kg/day). Round dose to the nearest 250mg (half-tablet). Adjust dose to individual response and therapeutic goals. Consider temporary dose interruption if serum ferritin falls consistently <500mcg/L.
Children:
Not recommended.
Warnings/Precautions:
Not established for use in treating other chronic anemias. Risk of neutropenia or fatal agranulocytosis. Measure ANC before starting therapy and monitor weekly during. Interrupt therapy if infection or neutropenia develops (ANC <1.5X109/L). If neutropenia occurs, obtain CBCs, WBCs, ANC, and platelets daily until recovery (ANC ≥1.5X109/L). History of QT prolongation (eg, those with CHF, bradycardia, diuretic use, cardiac hypertrophy, hypokalemia, hypomagnesemia). Monitor serum ALT monthly; consider interruption if persistent increase in transaminase levels. Monitor serum ferritin every 2–3 months. Monitor plasma zinc, supplement if deficient. Pregnancy (Cat.D); avoid use. Nursing mothers: not recommended.
Interaction(s):
Avoid concomitant use with other drugs associated with neutropenia or agranulocytosis. Allow at least 4-hour interval with antacids or mineral supplements containing polyvalent cations (eg, iron, aluminum, zinc). Concomitant UGT 1A6 inhibitors: closely monitor and may need dose adjustments or interruptions.
Adverse Reaction(s):
Chromaturia, GI upset, abdominal pain, increased ALT, arthralgia, neutropenia; agranulocytosis, possible Torsades de Pointes.
Notes:
This product is available from Centric Health Resources (CHR). CHR is a specialty pharmacy specializing in orphan drugs and is the sole distributor of Ferriprox in the US. For more information, contact Ferriprox Total Care at (866) 758-7071.
How Supplied:
Tabs—100
最后更新：
2012年2月2日
去铁酮Ferriprox在英国上市
Ferriprox（去铁酮，deferiprone）在英国上市，用于对去铁胺（deferoxamine）禁忌或不能耐受的重型地中海贫血症患者铁过载的治疗。其规格为500mg片剂，由瑞典OrPhan Intnl. AB公司供应。
研究显示Ferriprox能更有效去除海洋性贫血患者心脏内的过量铁患者心脏内的过量铁。
两项最新研究的结果显示，利用口服铁螯合剂 Ferriprox（TM）治疗不仅能够预防由铁引发的心脏病，而且能够明显降低需定期输血治疗的海洋性贫血患者过早心脏死亡的风险。
海洋性贫血是一种影响人体红血球细胞制造功能的遗传疾病。海洋性贫血是一种长期病症，需要患者每两到四周接受一次输血治疗，因而造成了人体内各器官（心脏、肝脏和内腺）毒性铁的大量沉积。数据显示，与目前的治疗标准 DFO（去铁胺）相比，Ferriprox 能够对心脏提供更好的保护。尽管 DFO 已降低了海洋性贫血患者的死亡率，然而心脏病依然是最常见的致死原因。接受 DFO 治疗的患者中有70%的人死于心脏病，年龄一般在20或30年龄段上。
铁螯合剂是从心脏中去除过量铁的唯一有效疗法。直到最近，DFO 还是唯一的螯合剂。该药物必须在患者皮下注射8到12个小时（每星期5到7个晚上）。Ferriprox 是第一种口服铁螯合剂，目前已在包括欧盟在内的48个国家和地区获得了批准，主要治疗铁负荷过重的重型海洋性贫血患者（不适于用 DFO 治疗）或用 DFO 治疗呈现严重毒性的患者。
使用 Ferriprox 治疗的患者最常见的不良反应是短暂的胃肠症状（恶心、呕吐或腹部疼痛）。这些症状通常在治疗的第一周出现，而在停止或减少药量后的三天内缓解。
药品中文名称 去铁酮片
药品名称说明
药品英文名称 Deferiprone Tablets
药品中文商品名 奥贝安可
药品英文商品名 Ferriprox
中文剂型 片剂
英文剂型
药品规格 0.5g
包装规格 100片/瓶
注册证号 H20030487
原注册证号
注册证号说明
分包装文号
公司中文名称
公司英文名称 Apotex Inc.
公司中文地址
公司英文地址 150 Signet Drive Toronto,Ontario Canada M9L 1T9
公司中文国别 加拿大
公司英文国别 Canada
生产厂商中文名
生产厂商英文名 Torpharm Inc.,A Division of Apotex Inc.
生产厂国内地址
生产厂国内地址 50 Steinway Boulevard Etobicoke,Ontario Canada M9W 6Y3
生产厂中文国别 加拿大
生产厂英文国别 Canada
批准日期 2003-09-23