ST LOUIS (MD Consult) - On June 30, 2009, AMAG Pharmaceuticals, Inc, announced that the US Food and Drug Administration (FDA) has granted marketing approval for Feraheme (ferumoxytol) for the treatment of iron deficiency anemia in adults with chronic kidney disease. According to Brian J. G. Pereira, MD, president and chief executive officer of AMAG, "Feraheme offers patients across the continuum of chronic kidney disease, including patients not on dialysis and patients on dialysis, a new paradigm for the treatment of iron deficiency anemia."

 

Feraheme is used as an iron replacement therapy and is administered intravenously (IV). The recommended dose schedule is an initial 510-mg injection, followed by a second 510-mg injection given 3 to 8 days later. Feraheme should be administered as an undiluted IV injection delivered at a rate of up to 1 mL/s (ie, 30 mg/s). The recommended Feraheme dose may be readministered to patients with persistent or recurrent iron deficiency anemia.

The FDA approval of Feraheme was granted on the basis of safety and efficacy data from 4 phase 3 studies of patients with chronic kidney disease and iron deficiency anemia. These studies consisted of 3 open-label, multicenter, randomized, safety and efficacy clinical studies and a fourth double-blind, multicenter, randomized, placebo-controlled, crossover safety study. Each of the pivotal safety and efficacy studies achieved statistical significance in their primary end point: the mean change in hemoglobin levels from baseline to day 35 after the first dose. The studies showed that the use of Feraheme was associated with significantly increased hemoglobin levels, compared with the use of oral iron, across the spectrum of chronic kidney disease. Overall, 1,726 patients were exposed to Feraheme in the development program, including 1,562 patients with all stages of chronic kidney disease.

 

AMAG plans to conduct 2 post marketing studies in the pediatric chronic kidney disease population: one in patients receiving dialysis and the other in patients not receiving dialysis. Approximately 75 patients will be enrolled in each study. In these trials, pharmacokinetic, safety, and efficacy data will be collected to compare Feraheme with oral iron therapy. The company expects to commence these studies in 2010.

 

In clinical trials, the most commonly reported (≥2%) adverse reactions in Feraheme-treated patients versus oral-iron–treated patients were diarrhea (4.0% vs 8.2%), nausea (3.1% vs 7.5%), dizziness (2.6% vs 1.8%), hypotension (2.5% vs 0.4%), constipation (2.1% vs 5.7%), and peripheral edema (2.0% vs 3.2%). In clinical trials, adverse reactions leading to treatment discontinuation and occurring in 2 or more Feraheme-treated patients included hypotension, chest pain, diarrhea, dizziness, ecchymosis, pruritus, urticaria, infusion-site swelling, increased serum ferritin level, and chronic renal failure.

圣路易斯(MD Consult)——2009年6月30日，美国AMAG制药公司宣布，美国食品药品管理局(FDA)已批准Feraheme(ferumoxytol)上市，用于治疗成人慢性肾病缺铁性贫血。据AMAG总裁兼首席执行官Brian J. G. Pereira博士称，“Feraheme为各类慢性肾病患者提供了一个治疗缺铁性贫血的新方案，且不受透析治疗的影响。”

Feraheme可作为一种铁替代疗法，经静脉用药(IV)。推荐用法为，初始静脉滴注510 mg，3~8d后再次静脉滴注510 mg。该药静脉用药时无需稀释，给药速度达1 ml/s(即，30mg/s)。对于患有顽固性或复发性缺铁性贫血的患者，Feraheme给药时仍可采用其推荐剂量。

此次批准是基于4项对慢性肾病缺铁性贫血患者进行的III期试验的安全性和有效性资料。这些研究包括3项开放标签、多中心、随机、安全性和有效性临床试验和1项双盲、多中心、随机、安慰剂对照、交叉的安全性试验。主要终点为基线至首剂后35d血红蛋白水平的平均变化值，在此方面，各项核心的安全性和有效性研究均达到了统计学意义。这些研究显示，无论患有何类型的慢性肾病，与口服铁剂相比，应用Feraheme与血红蛋白水平显著增加均具有相关性。研发计划中，共有1,726例患者接受Feraheme治疗，其中包括1,562例处于不同期肾病的患者。

AMAG拟于儿童慢性肾病群体中进行2项上市后研究：一项针对接受透析的患者，另一项针对不接受透析的患者。各研究将募集大约75例患者。在这些试验中，研究人员将收集Feraheme相关的药代动力学、安全性和有效性资料，并与口服铁剂疗法作比较。公司期冀于2010年正式启动上述研究。

在临床试验中， Feraheme治疗组患者与口服铁剂治疗的患者相比，最常报告(≥2%)的不良反应如下：腹泻(4.0%对8.2%)，恶心(3.1%对7.5%)，头晕(2.6%对1.8%)，低血压(2.5%对0.4%)，便秘(2.1%对5.7%)，以及外周水肿(2.0%对3.2%)。