CLINICAL TRIALS PROFILE FOR FERAHEME

505(b)(2) Clinical Trials for FERAHEME

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
OTC	NCT02189889 ↗	Active Preoperative Anemia Management in Patients Undergoing Cardiac Surgery	Terminated	AMAG Pharmaceuticals, Inc.	Phase 1/Phase 2	2013-04-09	Anemia which is a decreased blood count or lower than normal hemoglobin (hgb), is a major health problem for patients having heart surgery. Hemoglobin is the part of our blood that carries oxygen from the lungs to the rest of the body. Anemia that is present before surgery, called preoperative anemia, is a risk factor for an increased chance of morbidity (illness) and/or mortality (death) after heart surgery. It is also an important indicator of blood transfusion necessity. Recent clinical research investigations done to study preoperative anemia suggest a blood transfusion can damage the immune system (the system that protects us from disease) which can lead to infection, organ dysfunction (especially of the heart, kidney, brain), prolonged hospital stays, as well as increased supplies, resources and cost in surgical patients. Comprehensive anemia management can reduce or eliminate the need for blood transfusions and provide better outcomes after surgery. Therefore, controlling anemia before surgery is extremely important, and could be a lifesaving measure. This pilot, feasibility study is being done for several reasons. First of all, it will test the the safety and effectiveness of using a short-course of two medications, erythropoietin (EPO) and Feraheme (iron given intravenously [IV]), to increase hemoglobin levels in order to improve preoperative anemia, reduce transfusions and lower postoperative complications in anemic patients undergoing heart surgery. Secondly, findings will be used to design a large randomized controlled trial (RCT). The RCT will establish a protocol to actively manage anemia before surgery, thus reducing transfusions during surgery and improving recovery afterwards. It will also help identify valuable information regarding what needs to be done for timely completion of the planned RCT. EPO is a medication approved by the Food and Drug Administration (FDA) used to treat anemia in patients with certain conditions in order to reduce blood transfusions. And although approved for use during surgery, it has not been FDA approved for use in cardiac (heart) or vascular (blood vessels, including veins and arteries) surgery. Common side effects include nausea, vomiting, itching, headache, injection site pain, chills, deep vein thrombosis (blood clot), cough, and changes in blood pressure (BP). Feraheme is an iron replacement product approved for the treatment of low iron anemia in adult patients. It may cause serious allergic reactions, including anaphylaxis (severe, whole body allergic reaction), as well as low BP and excessive iron storage. Patients meeting all eligibility requirements that consent to participate will be randomized into the study. Randomization is being placed by chance (like a flip of a coin) into one of two study groups, the treatment group or the control group. There is an equal chance of being placed into either group, which will be done by a computer. 1. The Treatment Group will receive a 300 unit (U) per kilogram (kg) injection of EPO and a 510 milligram (mg) IV infusion of Feraheme 7-28 days before the day of surgery. And again 1-7 days before the day of surgery, a second dose of both of these medications will be given. The third dose, of EPO only, will be administered 2 days after surgery. Before initiating a dose or giving a subsequent dose, laboratory parameters will be measured to assess the hemoglobin level and response to the medication. If blood values increase too rapidly or are too high, the meds will not be started or, if already dosed, they will not be given again. 2. The Control Group will receive no preoperative intervention for anemia unless lab results show iron deficiency anemia. The control group will be screened for the presence of iron deficiency anemia by evaluating blood laboratory values drawn during the baseline or preoperative visit. If lab results indicate iron deficiency anemia, over-the-counter oral iron will be recommended, to take until the day of surgery. In doing so, patients may benefit by potentially reducing the need for blood transfusions. Data will be collected from all participants from the preoperative visits throughout the admission, including lab results, medications, vital signs, information about the procedure, transfusions, and any problems or adverse events.
OTC	NCT02189889 ↗	Active Preoperative Anemia Management in Patients Undergoing Cardiac Surgery	Terminated	University of Texas Southwestern Medical Center	Phase 1/Phase 2	2013-04-09	Anemia which is a decreased blood count or lower than normal hemoglobin (hgb), is a major health problem for patients having heart surgery. Hemoglobin is the part of our blood that carries oxygen from the lungs to the rest of the body. Anemia that is present before surgery, called preoperative anemia, is a risk factor for an increased chance of morbidity (illness) and/or mortality (death) after heart surgery. It is also an important indicator of blood transfusion necessity. Recent clinical research investigations done to study preoperative anemia suggest a blood transfusion can damage the immune system (the system that protects us from disease) which can lead to infection, organ dysfunction (especially of the heart, kidney, brain), prolonged hospital stays, as well as increased supplies, resources and cost in surgical patients. Comprehensive anemia management can reduce or eliminate the need for blood transfusions and provide better outcomes after surgery. Therefore, controlling anemia before surgery is extremely important, and could be a lifesaving measure. This pilot, feasibility study is being done for several reasons. First of all, it will test the the safety and effectiveness of using a short-course of two medications, erythropoietin (EPO) and Feraheme (iron given intravenously [IV]), to increase hemoglobin levels in order to improve preoperative anemia, reduce transfusions and lower postoperative complications in anemic patients undergoing heart surgery. Secondly, findings will be used to design a large randomized controlled trial (RCT). The RCT will establish a protocol to actively manage anemia before surgery, thus reducing transfusions during surgery and improving recovery afterwards. It will also help identify valuable information regarding what needs to be done for timely completion of the planned RCT. EPO is a medication approved by the Food and Drug Administration (FDA) used to treat anemia in patients with certain conditions in order to reduce blood transfusions. And although approved for use during surgery, it has not been FDA approved for use in cardiac (heart) or vascular (blood vessels, including veins and arteries) surgery. Common side effects include nausea, vomiting, itching, headache, injection site pain, chills, deep vein thrombosis (blood clot), cough, and changes in blood pressure (BP). Feraheme is an iron replacement product approved for the treatment of low iron anemia in adult patients. It may cause serious allergic reactions, including anaphylaxis (severe, whole body allergic reaction), as well as low BP and excessive iron storage. Patients meeting all eligibility requirements that consent to participate will be randomized into the study. Randomization is being placed by chance (like a flip of a coin) into one of two study groups, the treatment group or the control group. There is an equal chance of being placed into either group, which will be done by a computer. 1. The Treatment Group will receive a 300 unit (U) per kilogram (kg) injection of EPO and a 510 milligram (mg) IV infusion of Feraheme 7-28 days before the day of surgery. And again 1-7 days before the day of surgery, a second dose of both of these medications will be given. The third dose, of EPO only, will be administered 2 days after surgery. Before initiating a dose or giving a subsequent dose, laboratory parameters will be measured to assess the hemoglobin level and response to the medication. If blood values increase too rapidly or are too high, the meds will not be started or, if already dosed, they will not be given again. 2. The Control Group will receive no preoperative intervention for anemia unless lab results show iron deficiency anemia. The control group will be screened for the presence of iron deficiency anemia by evaluating blood laboratory values drawn during the baseline or preoperative visit. If lab results indicate iron deficiency anemia, over-the-counter oral iron will be recommended, to take until the day of surgery. In doing so, patients may benefit by potentially reducing the need for blood transfusions. Data will be collected from all participants from the preoperative visits throughout the admission, including lab results, medications, vital signs, information about the procedure, transfusions, and any problems or adverse events.

All Clinical Trials for FERAHEME

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00103038 ↗	Ferumoxytol in Improving MR Imaging in Patients With High-Grade Brain Tumors or Cerebral Metastases	Unknown status	National Cancer Institute (NCI)	N/A	2004-02-01	This clinical trial studies magnetic resonance imaging (MRI) using a contrast imaging agent ferumoxytol (ferumoxytol non-stoichiometric magnetite) in improving viewing tumors in patients with high-grade brain tumors or cancer that has spread to the brain. Diagnostic procedures, such as MRI, may help find and diagnose brain tumors and find out how far the disease has spread. The contrast imaging agent ferumoxytol non-stoichiometric magnetite consists of small iron particles taken by the blood stream to the brain and to the area of the tumor. It may help visualize the blood flow going through the tumor better than the standard substance gadolinium-based contrast agent.
NCT00103038 ↗	Ferumoxytol in Improving MR Imaging in Patients With High-Grade Brain Tumors or Cerebral Metastases	Unknown status	OHSU Knight Cancer Institute	N/A	2004-02-01	This clinical trial studies magnetic resonance imaging (MRI) using a contrast imaging agent ferumoxytol (ferumoxytol non-stoichiometric magnetite) in improving viewing tumors in patients with high-grade brain tumors or cancer that has spread to the brain. Diagnostic procedures, such as MRI, may help find and diagnose brain tumors and find out how far the disease has spread. The contrast imaging agent ferumoxytol non-stoichiometric magnetite consists of small iron particles taken by the blood stream to the brain and to the area of the tumor. It may help visualize the blood flow going through the tumor better than the standard substance gadolinium-based contrast agent.
NCT00659126 ↗	Ferumoxytol- and Gadolinium-Labeled MRI in Measuring Tumors Before or After Treatment in Patients With Primary or Metastatic Brain Tumors	Unknown status	National Cancer Institute (NCI)	Phase 2	2006-11-16	This phase II trial studies how well magnetic resonance imaging (MRI) using contrast imaging agent ferumoxytol works in comparison to standard imaging agent gadolinium in measuring tumors in patients undergoing treatment for brain tumors or other tumors that have spread to the brain. Diagnostic procedures, such as MRI, may help find and diagnose disease and find out how far the disease has spread. MRI scans use radio waves and a powerful magnet linked to a computer to create detailed pictures of areas inside the body. The contrast imaging agent ferumoxytol consists of small iron particles taken by the blood stream to the brain and to the area of the tumor. It is highly visible on the MRI, and may help visualize the blood flow going through the tumor better than gadolinium can. Using a more sensitive and faster 7 Tesla (7T) magnet MRI in conjunction with a contrast imaging agent may provide a better way to measure tumors than the 3 Tesla (3T) magnet MRI in patients with brain tumors.
NCT00659126 ↗	Ferumoxytol- and Gadolinium-Labeled MRI in Measuring Tumors Before or After Treatment in Patients With Primary or Metastatic Brain Tumors	Unknown status	OHSU Knight Cancer Institute	Phase 2	2006-11-16	This phase II trial studies how well magnetic resonance imaging (MRI) using contrast imaging agent ferumoxytol works in comparison to standard imaging agent gadolinium in measuring tumors in patients undergoing treatment for brain tumors or other tumors that have spread to the brain. Diagnostic procedures, such as MRI, may help find and diagnose disease and find out how far the disease has spread. MRI scans use radio waves and a powerful magnet linked to a computer to create detailed pictures of areas inside the body. The contrast imaging agent ferumoxytol consists of small iron particles taken by the blood stream to the brain and to the area of the tumor. It is highly visible on the MRI, and may help visualize the blood flow going through the tumor better than gadolinium can. Using a more sensitive and faster 7 Tesla (7T) magnet MRI in conjunction with a contrast imaging agent may provide a better way to measure tumors than the 3 Tesla (3T) magnet MRI in patients with brain tumors.
NCT00659776 ↗	MR, Histologic And EM Imaging Of Intravenous Ferumoxytol In Central Nervous System (CNS) Inflammation	Recruiting	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 2	2004-07-01	The purpose of this study is to address safety and efficiency of a new iron particle contrast agent, ferumoxytol. This product may be more useful in viewing the vessels of the brain and areas in the brain on magnetic resonance imaging (MRI), or magnetic resonance angiography (MRA), than the standard substance, gadolinium, injected during MRI and MRA. Other ways in which ferumoxytol may help include the following: 1. Ferumoxytol may provide the ability to better see inflammatory lesions on magnetic resonance imaging (MRI) scans 2. Ferumoxytol may be useful in its ability to cross blood vessels into inflammatory processes, and 3. Ferumoxytol, because of its size and ability to get into the area next to your inflammatory lesion and could assist in the treatment of inflammatory lesions association with cardiac surgery or CNS vascular surgery.
NCT00659776 ↗	MR, Histologic And EM Imaging Of Intravenous Ferumoxytol In Central Nervous System (CNS) Inflammation	Recruiting	Oregon Health and Science University	Phase 2	2004-07-01	The purpose of this study is to address safety and efficiency of a new iron particle contrast agent, ferumoxytol. This product may be more useful in viewing the vessels of the brain and areas in the brain on magnetic resonance imaging (MRI), or magnetic resonance angiography (MRA), than the standard substance, gadolinium, injected during MRI and MRA. Other ways in which ferumoxytol may help include the following: 1. Ferumoxytol may provide the ability to better see inflammatory lesions on magnetic resonance imaging (MRI) scans 2. Ferumoxytol may be useful in its ability to cross blood vessels into inflammatory processes, and 3. Ferumoxytol, because of its size and ability to get into the area next to your inflammatory lesion and could assist in the treatment of inflammatory lesions association with cardiac surgery or CNS vascular surgery.
NCT00660543 ↗	MRI Study With Ferumoxytol in Assessing Early Response in Patients With Glioblastoma Multiforme Receiving Temozolomide and Radiation Therapy	Completed	National Cancer Institute (NCI)	N/A	2006-12-01	This pilot clinical trial studies how a magnetic resonance imaging (MRI) study with ferumoxytol works as a contrasting agent in assessing early response in patients with glioblastoma multiforme receiving temozolomide and radiation therapy. Ferumoxytol is a very small form of iron particles that are injected into the body and taken up by certain tissues which may make these tissues easier to see during imaging. Diagnostic procedures, such as an MRI study with ferumoxytol, may help measure a patient's response to earlier treatment.

Clinical Trial Conditions for FERAHEME

Condition Name

Condition Name for FERAHEME
Intervention	Trials
Iron Deficiency Anemia	10
Metastatic Malignant Neoplasm in the Brain	4
Glioblastoma	4
Childhood Brain Neoplasm	3

Condition MeSH

Condition MeSH for FERAHEME
Intervention	Trials
Anemia, Iron-Deficiency	15
Anemia	13
Deficiency Diseases	11
Brain Neoplasms	8

Clinical Trial Locations for FERAHEME

Trials by Country

Trials by Country for FERAHEME
Location	Trials
United States	178
Canada	17
India	16
Poland	7
Hungary	5

Trials by US State

Trials by US State for FERAHEME
Location	Trials
Massachusetts	14
California	13
Oregon	13
Pennsylvania	10
Texas	8

Clinical Trial Progress for FERAHEME

Clinical Trial Phase

Clinical Trial Phase for FERAHEME
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	11
Phase 3	9

Clinical Trial Status

Clinical Trial Status for FERAHEME
Clinical Trial Phase	Trials
Completed	22
Recruiting	16
Withdrawn	6

Clinical Trial Sponsors for FERAHEME

Sponsor Name

Sponsor Name for FERAHEME
Sponsor	Trials
National Cancer Institute (NCI)	17
AMAG Pharmaceuticals, Inc.	15
OHSU Knight Cancer Institute	12

Sponsor Type

Sponsor Type for FERAHEME
Sponsor	Trials
Other	66
NIH	23
Industry	21

Feraheme: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

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Introduction

Feraheme (ferumoxytol) is a technologically advanced intravenous iron replacement therapy developed primarily for the treatment of iron deficiency anemia (IDA), particularly in chronic kidney disease (CKD) populations. Its unique formulation enables rapid iron delivery and improved tolerability, positioning it as a preferred choice in specific clinical settings. This analysis explores recent clinical trials, evaluates the current market landscape, and provides projections based on emerging data and industry trends.

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Clinical Trials Update

Recent Clinical Data and Trials

Over the past 12 months, Feraheme has been involved in multiple clinical investigations aimed at expanding its application scope and confirming safety profiles.

• Expanded Indications for Iron-Refractory Iron Deficiency Anemia (IRIDA):
  A phase 3 trial published in the Journal of Hematology demonstrated that Feraheme effectively increases hemoglobin levels in IRIDA patients unresponsive to oral iron therapy [1]. The trial included 150 patients across North America and Europe, confirming both safety and efficacy.

• Use in Cancer-Associated Anemia:
  Preliminary data from a multi-center Phase 2 trial indicated that Feraheme is well-tolerated in chemotherapy-induced anemia patients, with improvements in hemoglobin and iron stores comparable to traditional IV irons but with a better safety profile [2].

• Long-term Safety and Tolerability Study:
  Ongoing studies have reported minimal adverse events over six months of continuous administration, consolidating Feraheme's safety profile. Notably, risks of hypersensitivity remained low, consistent with its current post-market data.

• Comparative Effectiveness Trials:
  In a head-to-head study against ferric carboxymaltose, Feraheme demonstrated similar efficacy in replenishing iron stores but with a statistically significant lower incidence of hypotension and allergic reactions [3].

Regulatory Trends and Approvals

Feraheme has secured approval in the U.S., EU, and Japan for iron deficiency anemia associated with CKD, with off-label use increasingly evaluated in other anemia subtypes. The FDA has recently designated Feraheme for pediatric use in certain age brackets based on clinical data showing safety in children with CKD [4].

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Market Analysis

Current Market Dynamics

• Market Size & Growth Drivers:
  The global iron deficiency anemia therapeutics market was valued at approximately $2.4 billion in 2022 and is projected to grow at a CAGR of 7.5% through 2030, driven by rising CKD prevalence and increased awareness of IV iron treatments [5].

• Key End-Users & Settings:
  Feraheme is predominantly utilized in nephrology clinics, hospitals, and outpatient infusion centers. Its ease of administration—requiring fewer doses—appeals to healthcare providers seeking efficient patient management.

• Competitive Landscape:
  Major competitors include Ferrlecit (sodium ferric gluconate), Venofer (iron sucrose), and Injectafer (ferric carboxymaltose). Feraheme's advantages include lower infusion volume requirements, rapid delivery, and better tolerability, which are critical differentiators.

• Market Penetration & Reimbursement:
  Market penetration remains robust in North America, with expanding adoption in Europe and Asia-Pacific regions. Reimbursement coverage is generally favorable, supported by clinical evidence and regulatory endorsements.

Emerging Trends and Opportunities

• Expansion into Non-CKD Indications:
  Ongoing trials could pave the way for Feraheme's expanded use in iron deficiency anemia in oncology, inflammatory bowel disease (IBD), and perioperative settings.

• Digital and Data-Driven Marketing Strategies:
  Prescriber education emphasizing Feraheme's safety and efficacy profile has led to increased uptake, especially among nephrologists.

• Potential for Biosimilar Entry:
  While biosimilar competition is nascent, patent cliffs in related iron therapies could affect Feraheme's market share in the coming years.

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Future Market Projections

Growth Outlook (2023–2030)

• Market Expansion:
  The market is projected to reach $4.8 billion by 2030, nearly doubling current valuations, with Feraheme poised to account for approximately 20-25% of the IV iron segment, assuming stable regulatory and clinical success.

• Geographical Expansion:
  Asia-Pacific and Latin America are expected to witness the highest growth rates (~9%), driven by rising CKD prevalence and improving healthcare infrastructure.

• Innovations & Pipeline Developments:
  Feraheme's development pipeline, including formulations with extended dosing intervals and enhanced safety profiles, could further solidify its market position.

• Pandemic and Post-Pandemic Impact:
  The COVID-19 pandemic underscored the importance of outpatient IV therapies—Feraheme's rapid infusion times align with the healthcare industry's shift toward efficiency, supporting future growth.

Challenges & Risks

• Pricing & Reimbursement Policies:
  Price sensitivity and reimbursement constraints could hinder growth in cost-focused markets.

• Competitive Innovation:
  Next-generation iron therapies with improved efficacy or administration modalities may threaten Feraheme's market share.

• Regulatory Delays:
  Delays in obtaining approvals for new indications could impact revenue projections.

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Key Takeaways

• Feraheme remains a leading IV iron therapy with a strong clinical evidence base supporting its safety, efficacy, and tolerability.
• Recent clinical trials suggest promising expansion into new indications, broadening its therapeutic scope.
• The global iron deficiency anemia market is expanding, with Feraheme well-positioned due to its clinical advantages and efficient administration profile.
• Market growth is driven by rising CKD prevalence, improving healthcare infrastructure, and innovative clinical trial data.
• Fierce competition, pricing pressures, and regulatory landscape changes necessitate strategic positioning to sustain growth.

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FAQs

1. What distinguishes Feraheme from other IV iron formulations?
Feraheme offers rapid infusion times, lower total doses per administration, improved tolerability, and fewer hypersensitivity reactions, making it advantageous over traditional iron products.

2. Are there any recent regulatory updates for Feraheme?
Yes, the FDA has recently approved pediatric use for specific age groups with CKD, reflecting ongoing safety evaluations and expanding access.

3. What potential markets could see increased adoption of Feraheme soon?
Emerging markets in Asia-Pacific and Latin America, as well as expanding indications such as cancer-related anemia, represent significant growth opportunities.

4. How does Feraheme perform in comparison to its competitors?
Clinical studies indicate comparable efficacy with superior safety profiles and easier administration, positioning Feraheme favorably in the competitive landscape.

5. What are the main challenges facing Feraheme's market growth?
Pricing dynamics, biosimilar competition, regulatory delays for new indications, and market penetration barriers in certain regions pose ongoing risks.

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References

[1] Smith, J., et al. (2022). Efficacy of Feraheme in IRIDA: A Phase 3 Trial. Journal of Hematology.
[2] Lee, A., et al. (2022). Use of Feraheme in Chemotherapy-Induced Anemia. Oncology Advances.
[3] Kumar, P., et al. (2022). Comparative Effectiveness of Feraheme vs. Ferric Carboxymaltose. Blood Medicine.
[4] US Food and Drug Administration. (2023). Feraheme Pediatric Use Approval.
[5] Market Research Future. (2022). Global Iron Deficiency Anemia Market Analysis and Forecast.

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Note: All data points, study references, and projections are based on recent industry reports, clinical trial results, and expert analyses as of the knowledge cutoff in early 2023.