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Last Updated: February 17, 2025

CLINICAL TRIALS PROFILE FOR DEFERASIROX


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505(b)(2) Clinical Trials for Deferasirox

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
New Formulation NCT02435212 ↗ Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload Active, not recruiting Novartis Pharmaceuticals Phase 2 2015-10-21 This is a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox DT formulation in children and adolescents aged ≥ 2 and < 18 years at enrollment with any transfusion-dependent anemia requiring chelation therapy due to iron overload, to demonstrate the effect of improved compliance on iron burden. Randomization will be stratified by age groups (2 to <10 years, 10 to <18 years) and prior iron chelation therapy (Yes/ No). There will be two study phases which include a 1 year core phase where patients will be randomized to a 48 week treatment period to either Deferasirox DT or granules, and an optional extension phase where all patients will receive the granules up to 5 years. Patients who demonstrated benefit to granules or DT in the core phase, and/or express the wish to continue in the optional extension phase on granules, will be offered this possibility until there is local access to the new formulation (granules or FCT) or up to 5 years, whichever occurs first.
>Trial Type >Trial ID >Title >Status >Phase >Start Date >Summary

All Clinical Trials for Deferasirox

Trial ID Title Status Sponsor Phase Start Date Summary
NCT00061750 ↗ Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions Completed Novartis Pharmaceuticals Phase 3 2003-05-01 The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
NCT00061763 ↗ Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias Completed Novartis Pharmaceuticals Phase 2 2003-05-01 The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions. Beta-thalassemia patients unable to be treated with deferoxamine or patients with rare chronic anemias such as Myelodysplastic Syndrome, Fanconi's Syndrome, Blackfan-Diamond Syndrome, and Pure Red Blood Cell Anemia are eligible for this study. Liver iron content will be measured by liver biopsy at the beginning of the study and after one year of treatment. However, those patients living in the San Francisco/Oakland area may have a SQUID in place of the liver biopsy if the biopsy is not medically possible for them. The SQUID is a non-invasive magnetic means to measure liver iron content.
NCT00067080 ↗ Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions Completed Novartis Pharmaceuticals Phase 2 2003-05-01 The purpose of this study is to determine if the new orally active iron chelator, ICL670, is as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
NCT00110266 ↗ Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients Completed Novartis Pharmaceuticals Phase 2 2005-07-25 The purpose of this trial is to examine the safety and efficacy of deferasirox in patients with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.
NCT00110617 ↗ Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients Completed Novartis Pharmaceuticals Phase 2 2005-05-01 This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
NCT00117507 ↗ Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients Completed Novartis Pharmaceuticals Phase 4 2005-09-01 Thirty patients were to be enrolled and 24 patients were actually enrolled into this open-label, single-arm trial designed to assess the safety and tolerability of oral deferasirox in adult transfusion dependent myelodysplastic syndrome (MDS) patients with iron overload. Patients enrolled in this study had low or intermediate (INT-1) risk MDS per International Prognostic Scoring System (IPSS) criteria. All patients initiated treatment with 20mg/kg/day deferasirox. Deferasirox were administered orally once per day for 12 months.
NCT00171171 ↗ A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis Completed Novartis Phase 3 2004-05-01 Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for Deferasirox

Condition Name

Condition Name for Deferasirox
Intervention Trials
Iron Overload 16
Myelodysplastic Syndromes 12
Beta-thalassemia 7
Transfusional Iron Overload 7
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Condition MeSH

Condition MeSH for Deferasirox
Intervention Trials
Iron Overload 52
Thalassemia 36
Myelodysplastic Syndromes 25
beta-Thalassemia 25
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Clinical Trial Locations for Deferasirox

Trials by Country

Trials by Country for Deferasirox
Location Trials
United States 206
Italy 71
Canada 21
United Kingdom 19
China 19
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Trials by US State

Trials by US State for Deferasirox
Location Trials
California 23
New York 18
Illinois 15
Pennsylvania 14
Texas 12
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Clinical Trial Progress for Deferasirox

Clinical Trial Phase

Clinical Trial Phase for Deferasirox
Clinical Trial Phase Trials
Phase 4 21
Phase 3 11
Phase 2/Phase 3 3
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Clinical Trial Status

Clinical Trial Status for Deferasirox
Clinical Trial Phase Trials
Completed 62
Terminated 14
Unknown status 11
[disabled in preview] 9
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Clinical Trial Sponsors for Deferasirox

Sponsor Name

Sponsor Name for Deferasirox
Sponsor Trials
Novartis Pharmaceuticals 52
Novartis 11
Boston Children’s Hospital 2
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Sponsor Type

Sponsor Type for Deferasirox
Sponsor Trials
Other 88
Industry 69
NIH 3
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Deferasirox: Clinical Trials, Market Analysis, and Projections

Introduction to Deferasirox

Deferasirox, marketed under the brand name Exjade, is an oral iron chelator used primarily to treat iron overload in patients with transfusion-dependent thalassemia and other conditions. Here, we will delve into recent clinical trials, market analysis, and future projections for this drug.

Clinical Trials Update

Pilot Pharmacokinetic Study

A notable clinical trial, identified by the ClinicalTrials.gov identifier NCT00749515, was conducted to investigate the pharmacokinetics and response mechanisms of deferasirox in patients with inadequate response to the drug. This pilot study, sponsored by Boston Children's Hospital and Novartis, involved 15 participants, including 10 patients with poor responses and 5 control patients with good responses.

  • Study Design: The study had two parts. In Part I, all patients received a dose of 35 mg/kg of deferasirox and underwent inpatient physiological assessments, including pharmacokinetic monitoring and nuclear medicine scans. In Part II, inadequate responders continued on deferasirox for three months, while control patients resumed their previous dosing regimens[1].

  • Outcome Measures: The study focused on the area under the curve (AUC) of deferasirox, half-life, and other pharmacokinetic parameters. It also included a deferoxamine challenge to assess iron excretion and total iron binding capacity (TIBC)[1].

Study on Myelodysplastic Syndromes

Another study, conducted between 2013 and 2015, investigated the use of deferasirox in people with myelodysplastic syndromes (MDS). This study aimed to maintain iron levels within a specific range over 12 months.

  • Results: Despite difficulties in recruiting participants, the study found that deferasirox was acceptable, with most side effects being mild. Out of 13 participants, 8 maintained acceptable iron levels at 12 months. Common side effects included low platelet levels and anemia[4].

Market Analysis

Current Market Size and Growth

The global deferasirox market has been experiencing significant growth driven by several factors:

  • Market Size: As of 2023, the market size was valued at USD 2.9 billion and is projected to reach USD 4.6 billion by 2033, growing at a CAGR of 4.7%[3].
  • Segmentation: The market is segmented by product type (e.g., 500mg, 250mg, 215mg), application (e.g., β-thalassemia, sickle cell disease, MDS), population type (adults, pediatric), end-users (specialty clinics, hospitals, home care), and distribution channels (hospital pharmacy, retail pharmacy, online pharmacy)[2].

Key Drivers of Growth

Several factors are driving the growth of the deferasirox market:

  • Advancements in Treatment Efficiency: Novel oral formulations, such as granules, have improved flexibility in dosing and better absorption, enhancing patient compliance[2].
  • Technological Advancements: Integration of biosensors and point-of-care devices for real-time iron level monitoring has fueled demand for deferasirox[2].
  • Increased Awareness and Reimbursement Policies: Growing awareness of iron overload disorders and favorable reimbursement policies have contributed to market expansion[2].

Geographical Market

  • North America: Expected to dominate the market due to a large patient population and favorable government support for product approval. The region's well-established healthcare infrastructure also contributes to its significant market share[2].
  • Europe: Showing significant growth due to its diverse distribution network and advancements in healthcare infrastructure[2].

Market Projections

Forecast Period

  • 2025-2032: The global deferasirox market is projected to grow from USD 5.22 billion in 2024 to USD 7.95 billion by 2032, at a CAGR of 5.4%[2].
  • 2024-2033: Another forecast indicates the market will grow from USD 2.9 billion in 2023 to USD 4.6 billion by 2033, at a CAGR of 4.7%[3].

Emerging Trends

  • New Therapeutic Areas: Ongoing clinical trials exploring the efficacy of deferasirox in new therapeutic areas, such as non-transfusion-dependent thalassemia, are expected to further accelerate market expansion[2].
  • Patient Epidemiology and Pipeline Analysis: Detailed market reports include patient epidemiology, pipeline analysis, and regulatory frameworks, providing comprehensive insights into market dynamics[2].

Key Takeaways

  • Clinical Trials: Recent trials have focused on understanding pharmacokinetics and response mechanisms, as well as expanding the therapeutic use of deferasirox.
  • Market Growth: The deferasirox market is driven by advancements in treatment efficiency, technological integration, and increased awareness of iron overload disorders.
  • Geographical Dominance: North America and Europe are expected to lead the market due to their well-established healthcare infrastructures and favorable policies.
  • Future Projections: The market is projected to grow significantly over the next decade, driven by new therapeutic areas and technological advancements.

FAQs

What is the primary use of deferasirox?

Deferasirox is primarily used to treat iron overload in patients with transfusion-dependent thalassemia and other conditions.

What are the common side effects of deferasirox?

Common side effects include low platelet levels, anemia, and other mild adverse reactions.

How is the deferasirox market segmented?

The market is segmented by product type, application, population type, end-users, and distribution channels.

What are the key drivers of the deferasirox market growth?

Key drivers include advancements in treatment efficiency, technological advancements, increased awareness of iron overload disorders, and favorable reimbursement policies.

What is the projected market size of deferasirox by 2032?

The global deferasirox market is projected to reach USD 7.95 billion by 2032, growing at a CAGR of 5.4% from 2025 to 2032.

Sources

  1. ClinicalTrials.gov: Pilot Pharmacokinetic Study In Patients With Inadequate Response To Deferasirox (Exjade)[1].
  2. Data Bridge Market Research: Global Deferasirox Market Size, Trends, Growth & Analysis By 2032[2].
  3. GlobeNewswire: Deferasirox Market to Reach $4.6 Billion, Globally, by 2033[3].
  4. Cancer Research UK: A study looking at the drug deferasirox in people with myelodysplastic syndromes[4].
  5. 360 Research Reports: Deferasirox Market Size & Share 2025-2030[5].

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