CLINICAL TRIALS PROFILE FOR DEFERASIROX

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-	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

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.

Clinical Trial Conditions for Deferasirox

Condition Name

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

Condition MeSH

Condition MeSH for Deferasirox
Intervention	Trials
Iron Overload	53
Thalassemia	37
beta-Thalassemia	26
Preleukemia	25

Clinical Trial Locations for Deferasirox

Trials by Country

Trials by Country for Deferasirox
Location	Trials
United States	208
Italy	71
Canada	21
China	20
United Kingdom	19

Trials by US State

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

Clinical Trial Progress for Deferasirox

Clinical Trial Phase

Clinical Trial Phase for Deferasirox
Clinical Trial Phase	Trials
PHASE2	3
Phase 4	21
Phase 3	11

Clinical Trial Status

Clinical Trial Status for Deferasirox
Clinical Trial Phase	Trials
Completed	63
Terminated	14
Unknown status	11

Clinical Trial Sponsors for Deferasirox

Sponsor Name

Sponsor Name for Deferasirox
Sponsor	Trials
Novartis Pharmaceuticals	52
Novartis	11
National Heart, Lung, and Blood Institute (NHLBI)	2

Sponsor Type

Sponsor Type for Deferasirox
Sponsor	Trials
Other	92
Industry	69
NIH	5

Clinical Trials Update, Market Analysis, and Projection for Deferasirox

Last updated: October 28, 2025

Introduction

Deferasirox, an oral iron chelator marketed under the brand name Exjade among others, has been a critical therapy for managing chronic iron overload in patients requiring repeated blood transfusions. Since its FDA approval in 2005, Deferasirox has cemented its role in the hematology landscape. This article provides a comprehensive overview of recent clinical trial developments, market dynamics, and future projections for Deferasirox, offering valuable insights into its therapeutic trajectory and commercial potential.

Clinical Trials Update

Current and Recent Clinical Trials

Recent years have seen ongoing investigations into Deferasirox’s efficacy, safety profile, and expanded indications. Notably:

• Iron Overload in Non-Transfusion-Dependent Patients: Several trials (e.g., NCT03595185) are examining Deferasirox's utility in non-transfusion-dependent thalassemia and other iron overload conditions, aiming to broaden its application scope. Early results demonstrate promising iron reduction efficacy with manageable safety profiles.

• Combination Therapies: Trials are exploring Deferasirox in combination with other agents, such as deferiprone or deferoxamine, to enhance chelation efficacy. For example, NCT02335948 evaluated the safety of combining Deferasirox with deferiprone in pediatric populations, indicating good tolerability and improved iron clearance.

• Long-term Safety and Efficacy: Extended follow-up studies, like NCT03210250, assess long-term safety over five years, affirming its chronic use safety but emphasizing vigilance to potential adverse events such as renal impairment and gastrointestinal disturbances.

New Formulations and Dosing Strategies

Innovation in formulation aims to improve patient adherence:

• Dispersible Tablets: Development of dispersible formulations has shown benefits in pediatric populations, with ongoing trials indicating comparable bioavailability and tolerability.

• Flexible Dosing Regimens: Trials are examining adjusted dosing, tailored to patient weight and iron burden, to optimize therapeutic outcomes while minimizing side effects.

Regulatory and Post-Marketing Surveillance

Regulatory bodies, including the FDA and EMA, continue to monitor safety signals via post-marketing surveillance. Recent updates include warnings about renal toxicity and hepatic monitoring, prompting more rigorous clinical oversight.

Market Analysis

Market Landscape and Competitive Position

Deferasirox holds a substantial share in the iron chelation therapy market, valued at approximately $650 million in 2022 (estimated), with projected compounded annual growth rates (CAGR) of around 4-6% over the next five years [1].

Its primary competitors include deferoxamine (parenteral) and deferiprone (oral). While deferoxamine remains effective, drawbacks such as administration complexity limit its use. Deferasirox’s oral formulation and once-daily dosing confer significant advantages, securing its market position.

Market Drivers

• Growing Prevalence of Transfusion-Dependent Anemias: Thalassemia and sickle cell disease populations are expanding globally, especially in regions like the Middle East, Asia, and parts of Africa, increasing demand for iron chelation therapy.

• Expanded Indications: Emerging evidence supports Deferasirox's use beyond traditional transfusional iron overload, including non-transfusion-dependent iron overload conditions, broadening the market.

• Improved Patient Compliance: Easier administration routes and dosing flexibility improve adherence, boosting market penetration.

Market Challenges

• Safety Concerns: Risks of renal impairment, hepatic dysfunction, and gastrointestinal issues necessitate continuous monitoring, potentially impacting prescribing patterns.

• Pricing and Reimbursement: High costs and variable reimbursement policies across regions influence market access. Generic versions are emerging, exerting downward pressure on prices.

• Competitive Innovations: New chelators and combination therapies under clinical development threaten Deferasirox’s market dominance.

Regional Market Dynamics

• North America and Europe: Mature markets with high affordability and healthcare infrastructure favor established therapies like Deferasirox. Regulatory bodies advocate for vigilant safety monitoring.

• Asia-Pacific: Rapid growth fueled by increasing thalassemia prevalence, improving healthcare access, and evolving clinical guidelines. Local generics are also entering markets, influencing pricing strategies.

• Emerging Markets: Potential for market expansion, though price sensitivity and regulatory hurdles pose barriers.

Future Market Projections

Forecasted Growth and Opportunities

The Deferasirox market is expected to reach approximately $930 million by 2028, growing at a CAGR of 4-6%. Key growth drivers include:

• Enhanced Clinical Evidence: Ongoing trials demonstrating efficacy in broader patient populations will encourage wider adoption.

• Regulatory Approvals for New Indications: Approval for non-transfusion-dependent iron overload conditions will unlock new revenue streams.

• Technological Innovations: Improved formulations and personalized dosing capabilities will bolster patient adherence and therapeutic outcomes.

Potential Disruptors

• Emerging Chelators: Novel agents with improved safety profiles, such as deferiprone derivatives, could challenge Deferasirox’s market share.

• Biosimilars and Generics: Entry of cost-effective generic versions will pressure price points but may also expand access.

• Gene Therapies and Curative Approaches: Advances toward curative treatments for hemoglobinopathies could diminish long-term reliance on chelation therapies.

Strategic Outlook

Manufacturers should focus on post-marketing safety management, expanding indications through clinical evidence, and investing in patient-centric formulations. Collaborations with global health organizations can accelerate access in underserved regions, ensuring sustained growth.

Key Takeaways

• Robust Clinical Pipeline: Deferasirox continues to be evaluated in diverse patient populations, with promising results in non-traditional indications and combination therapies.

• Market Position and Growth Potential: The drug maintains a leading position due to its oral, once-daily dosing, with future growth driven by expanding indications and regional market penetration.

• Safety and Monitoring: Vigilant safety profiling remains essential, with ongoing monitoring influencing prescriber confidence and regulatory updates.

• Competitive Landscape: While facing emerging competitors and generics, Deferasirox’s established profile and patient adherence advantages sustain its leadership.

• Investment and Development Strategies: Companies should prioritize innovative formulations, expanded indications, and strategic collaborations to capitalize on market opportunities.

FAQs

1. What are the main clinical indications for Deferasirox?
Deferasirox is primarily indicated for chronic iron overload due to transfusions in conditions like beta-thalassemia major and other anemias. Emerging trials suggest potential applications in non-transfusion-dependent iron overload.

2. How does Deferasirox compare to other iron chelators?
Compared to deferoxamine (parenteral) and deferiprone (oral), Deferasirox offers the advantage of oral, once-daily dosing, improving patient adherence. However, safety profiles require monitoring for renal and hepatic toxicity.

3. Are there ongoing trials that could expand Deferasirox’s use?
Yes, ongoing studies are exploring its efficacy in non-transfusion-dependent iron overload, pediatric populations, and combinations with other chelators, which could broaden its therapeutic scope.

4. What factors influence the global market for Deferasirox?
Prevalence of hemoglobinopathies, healthcare infrastructure, safety profile management, pricing strategies, and regulatory approvals are key factors shaping its market dynamics.

5. How might emerging therapies impact Deferasirox’s market share?
Innovations such as improved chelators, biosimilars, and gene therapies could challenge Deferasirox’s dominance, emphasizing the need for continuous clinical innovation and strategic positioning.

References

[1] MarketWatch, "Iron Chelators Market Size, Share & Trends Analysis," 2022.