——静脉补铁剂Feraheme已获准用于治疗成人慢性肾病缺铁性贫血

 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。

该药物可用于所有阶段的CKD患者，包括未透析的与已透析的病人，提供了新的治疗缺铁性贫血的规范。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%)。

FDA Approves Feraheme™ to Treat Iron Deficiency Anemia in Adult Chronic Kidney Disease Patients

LEXINGTON, Mass.--(BUSINESS WIRE)--AMAG Pharmaceuticals, Inc. (NASDAQ:AMAG) today announced that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Feraheme™ (ferumoxytol) Injection for intravenous (IV) use as an iron replacement therapy for the treatment of iron deficiency anemia in adult patients with chronic kidney disease. The recommended dose of Feraheme is an initial 510 mg IV injection followed by a second 510 mg IV injection three to eight days later. Feraheme should be administered as an undiluted IV injection delivered at a rate of up to 1 mL/sec (30 mg/sec). The recommended Feraheme dose may be readministered to patients with persistent or recurrent iron deficiency anemia.

Feraheme is expected to be commercially available in the U.S. during the second half of July 2009. Feraheme will be distributed primarily through wholesalers and specialty distributors. The Company will market and sell Feraheme through its commercial organization consisting of approximately 150 seasoned professionals, including an 80-person specialized sales force, an experienced account management and reimbursement team, and a contract nurse team.

“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,” commented Brian J.G. Pereira, MD, President and Chief Executive Officer of AMAG. “We are extremely pleased with the FDA’s approval of Feraheme, and we are well prepared and excited to bring this new treatment option to patients and physicians.”

"Iron deficiency anemia is a significant problem in patients with chronic kidney disease and is frequently underdiagnosed and undertreated,1,2” said Bryan Becker, MD, President of the National Kidney Foundation. "We welcome the availability of a new therapy option for chronic kidney disease patients affected by iron deficiency anemia."

Clinical Data

Feraheme has been proven to be a safe and effective therapy for treating iron deficiency anemia in adult chronic kidney disease patients. The FDA approval of Feraheme was based on safety and efficacy results from four Phase III studies of patients with chronic kidney disease and iron deficiency anemia. These studies consisted of three open-label, multi-center, randomized safety and efficacy clinical studies and a fourth double-blind, multi-center, randomized, placebo-controlled cross-over safety study. Each of the three pivotal safety and efficacy studies achieved statistical significance in its primary endpoint: the mean change in hemoglobin from baseline at Day 35 after the first dose. Feraheme significantly increased hemoglobin levels as compared to oral iron across the spectrum of chronic kidney disease. Overall, 1,726 subjects were exposed to Feraheme in the development program, including 1,562 patients with all stages of chronic kidney disease.

In accordance with the Pediatric Research Equity Act (PREA) requirement, the Company will conduct two post-marketing studies in the pediatric chronic kidney disease population; one in patients on dialysis and the other in patients not on dialysis. Each study will enroll approximately 75 subjects, collecting pharmacokinetic, safety and efficacy data as compared to oral iron. The Company expects to commence these studies in 2010.

Important Safety Information

Feraheme is indicated for the treatment of iron deficiency anemia in adult patients with chronic kidney disease. Feraheme is contraindicated in patients with evidence of iron overload, known hypersensitivity to Feraheme or any of its components, and patients with anemia not caused by iron deficiency.

In clinical studies, hypotension was reported in 1.9% (33/1,726) of subjects receiving Feraheme, including three patients with serious hypotensive reactions. Adverse reactions potentially associated with hypersensitivity (e.g., pruritus, rash, urticaria or wheezing) were reported in 3.7% (63/1,726) of these subjects including 0.2% (3/1,726) with serious hypersensitivity reactions. Patients should be observed for signs and symptoms of hypersensitivity for at least 30 minutes following Feraheme injection and the drug should only be administered when treatment of hypersensitivity reactions is readily available. Excessive therapy with parenteral iron can lead to excess storage of iron with the possibility of iatrogenic hemosiderosis. Patients should be regularly monitored for hematologic response during parenteral iron therapy, noting that lab assays may overestimate serum iron and transferrin bound iron values in the 24 hours following administration of Feraheme. As a superparamagnetic iron oxide, Feraheme may transiently affect magnetic resonance diagnostic imaging studies for up to 3 months following the last Feraheme dose. Feraheme will not affect X-ray, computed tomography (CT), positron emission tomography (PET), single photon emission computed tomography (SPECT), ultrasound, or nuclear imaging.

In clinical trials, the most commonly occurring adverse reactions in Feraheme treated patients versus oral iron treated patients reported in ≥ 2% of chronic kidney disease 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, infusion site swelling, increased serum ferritin level, chest pain, diarrhea, dizziness, ecchymosis, pruritus, chronic renal failure, and urticaria.

Click here to view Feraheme’s product insert which is also available on our website at www.amagpharma.com.

Conference Call and Webcast Access

AMAG Pharmaceuticals, Inc. will host a webcast and conference call tomorrow at 8:30 a.m. ET to discuss today’s announcement.

To access the conference call via telephone, please dial (877) 412-6083 from the U.S. or (702) 495-1202 for international callers. A telephone replay will be available from approximately 11:30 a.m. ET on July 1, 2009 through midnight July 3, 2009. To access a replay of the conference call, dial (800) 642-1687 from the U.S. or (706) 645-9291 for international access. The passcode for the live call and the replay is 18122806.

An audio webcast of the call will be available through the Investors section of the Company’s website at www.amagpharma.com. A replay of the webcast will also be available from approximately 10:30 a.m. ET on July 1, 2009, through midnight July 31, 2009.

About AMAG Pharmaceuticals, Inc.

AMAG Pharmaceuticals, Inc. is a biopharmaceutical company that utilizes its proprietary technology for the development and commercialization of a therapeutic iron compound to treat iron deficiency anemia and novel imaging agents to aid in the diagnosis of cancer and cardiovascular disease. For additional company and product information please visit www.amagpharma.com.

Feraheme was developed by the Company and is composed of superparamagnetic iron oxide particles with a proprietary semi-synthetic carbohydrate coating.

Feraheme™ is a trademark of AMAG Pharmaceuticals, Inc.

FERAHEME