CLINICAL TRIALS PROFILE FOR FERRIPROX

505(b)(2) Clinical Trials for Ferriprox

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	NCT02442310 ↗	Comparison of Deferiprone Delayed Release Tablets and Deferiprone Oral Solution in Healthy Volunteers	Completed	Algorithme Pharma Inc	Phase 1	2015-05-01	The purpose of this study is to look at the pharmacokinetics of a new formulation of deferiprone (deferiprone delayed release tablets) under fed and fasting conditions.
New Formulation	NCT02442310 ↗	Comparison of Deferiprone Delayed Release Tablets and Deferiprone Oral Solution in Healthy Volunteers	Completed	ApoPharma	Phase 1	2015-05-01	The purpose of this study is to look at the pharmacokinetics of a new formulation of deferiprone (deferiprone delayed release tablets) under fed and fasting conditions.
New Formulation	NCT02465489 ↗	Comparison of Deferiprone Extended Release Tablets and Ferriprox Immediate Release Tablets in Healthy Volunteers	Completed	ApoPharma	Phase 1	2015-06-01	The purpose of this study is to look at the pharmacokinetics of a new formulation of deferiprone (deferiprone extended release tablets) under fed and fasting conditions.

All Clinical Trials for Ferriprox

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00529152 ↗	Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients	Completed	ApoPharma	Phase 3	2007-08-01	- The primary objective is to assess the safety of Ferriprox oral solution for the treatment of iron overload in pediatric patients with transfusion-dependent anemia. - The secondary objective is to assess the efficacy of Ferriprox oral solution in reducing iron overload in pediatric patients with transfusion-dependent anemia.
NCT00897221 ↗	A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia	Completed	ApoPharma	Phase 2	2009-06-01	The primary objective of this study is to evaluate the long-term safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA). The secondary objective is to evaluate the long-term efficacy of deferiprone for the treatment of FRDA. The tertiary objectives are to evaluate the effect of deferiprone on: 1. cardiac function, 2. quality of life, and 3. functional status.
NCT00907283 ↗	Ferrochelating Treatment in Patients Affected by Neurodegeneration With Brain Iron Accumulation (NBIA)	Active, not recruiting	Ente Ospedaliero Ospedali Galliera	Phase 2	2008-11-01	This trial is a multicenter, unblinded, single-arm pilot study, lasting one year (plus one year extension Amendment n.3 25 August 2009, plus two years follow-up Amendment n.7) , to evaluate the efficacy and safety of the chelator therapy with deferiprone on cerebral iron accumulations. The drug will be administered in the dosage of 15 mg/kg twice daily. The safety and tolerability of the drug will be evaluated by measuring hemochrome every seven days with leukocyte formula count. At 3, 6 and 12 months from the start of treatment, a neurological evaluation will be performed using several specific evaluation scales (International Cooperative Ataxia Rating Scale (ICARS); Unified Parkinson's Disease Rating Scale (UPDRS); Burke-Fahn-Marsden (BFM)). Every 6 months of treatment, a brain magnetic resonance image (MRI) aimed at measuring iron overload quantitatively, if possible.
NCT00943748 ↗	Efficacy and Safety of the Iron Chelator Deferiprone in Parkinson's Disease	Completed	University Hospital, Lille	Phase 2/Phase 3	2009-10-01	Few available drugs can slow the progression of neurodegenerative pathologies such as Parkinson's disease (PD). One of the recent hypotheses concerning the reduction of oxidative stress and neuron death features a harmful effect of iron, which may reach abnormally high levels in the substantia nigra (SN) pars compacta (iron overload has been seen in the substantia nigra in parkinsonian patients and in the 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP) mouse model of PD). Iron overload is harmful because it reacts with hydrogen peroxide (H2O2) produced during the oxidative deamination of dopamine, generating hydroxyl radicals which then damage proteins, DNA and phospholipid membranes and may be responsible for neuron death. The use of an iron chelator (clioquinol) produces a reduction in neuron death in the MPTP mouse model. In humans, a special, partially refocused interleaved multiple echo (PRIME) MR sequence has been used to study the relaxation time (T2*) and quantify iron overload in the SN of PD patients and the nucleus dentatus of patients with Friedreich's ataxia. T2* sequences have revealed a decrease in iron overload following treatment with the chelator deferiprone, in parallel with a clinical improvement in these patients. Furthermore, the very recent open label use of deferiprone in rare serious, systemic, neurological iron overload diseases (Neurodegeneration with Brain Iron Accumulation (NBIA)) has revealed a clinical improvement after 6 months, with 2 case reports from our group and another from an Italian group (Forni et al., 2008). The safety of the low dosages of deferiprone (20 to 30 mg/kg/day) used in neurology appears to be much greater than for the high dosages (75 to 100 mg/kg/day in 3 doses) used in hematology to decrease post-transfusion iron overloads in thalassemia major. Hence, the investigators wish to evaluate the effect of treatment with an oral iron chelator which is capable of crossing the blood-brain barrier (deferiprone) on iron overload in the SN(as assessed by the T2* sequence) with respect to the progression of clinical sign in PD. It is expected a 6-month course of deferiprone able to produce a moderate reduction in iron overload of the SN, associated with a drop in the motor handicap score. Depending on the risk/benefit balance determined in this initial pilot study, a larger, multicenter neuroprotection study could be envisaged.
NCT01511848 ↗	Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload	Unknown status	Ain Shams University	Phase 2/Phase 3	2012-02-01	Interventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload Primary Outcome Measures: • The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and deferasirox compared to combined DFP and DFO in conditions with severe chronic iron overload; showing an up-trend of SF over previous 12 months on single chelator. Secondary Outcome Measures: • The secondary outcome measure is to determine the number of patients who will develop adverse events in order to assess safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.

Clinical Trial Conditions for Ferriprox

Condition Name

Condition Name for Ferriprox
Intervention	Trials
Iron Overload	7
Sickle Cell Disease	4
Parkinson's Disease	3
Healthy	3

Condition MeSH

Condition MeSH for Ferriprox
Intervention	Trials
Iron Overload	10
Thalassemia	4
beta-Thalassemia	4
Anemia, Sickle Cell	4

Clinical Trial Locations for Ferriprox

Trials by Country

Trials by Country for Ferriprox
Location	Trials
United States	21
Canada	9
Egypt	6
Italy	4
France	4

Trials by US State

Trials by US State for Ferriprox
Location	Trials
California	4
Pennsylvania	4
Illinois	3
South Carolina	2
New York	2

Clinical Trial Progress for Ferriprox

Clinical Trial Phase

Clinical Trial Phase for Ferriprox
Clinical Trial Phase	Trials
Phase 4	6
Phase 3	2
Phase 2/Phase 3	4

Clinical Trial Status

Clinical Trial Status for Ferriprox
Clinical Trial Phase	Trials
Completed	20
Unknown status	3
Enrolling by invitation	1

Clinical Trial Sponsors for Ferriprox

Sponsor Name

Sponsor Name for Ferriprox
Sponsor	Trials
ApoPharma	17
University Hospital, Lille	3
Ain Shams University	2

Sponsor Type

Sponsor Type for Ferriprox
Sponsor	Trials
Industry	20
Other	13
U.S. Fed	1

Ferriprox (Deferiprone): Clinical Trials, Market Analysis, and Projections

Introduction

Ferriprox, known scientifically as deferiprone, is an orally active iron chelator approved for the treatment of chronic iron overload due to blood transfusions in patients with thalassemia and other conditions. Here, we delve into the current clinical trials, market analysis, and future projections for this crucial medication.

Clinical Trials Update

Ongoing and Planned Trials

As of the latest updates, a clinical trial targeting TMPRSS6 with an antibody to increase hepcidin and reduce iron levels is set to initiate in the second half of 2023. This trial, though still in its early stages, aims to enhance the efficacy of deferiprone by modulating hepcidin levels, a key regulator of iron metabolism[1].

Phase and Patient Enrollment

The upcoming trial is classified as a Phase 1 (Proof-of-Mechanism) study, focusing on healthy volunteers. The anticipated completion date for this trial is 2024, although specific details such as the number of patients enrolled and study sites are yet to be disclosed[1].

Efficacy and Safety

Previous clinical trials have shown that deferiprone is effective in reducing serum ferritin levels and cardiac iron overload. In pooled clinical trials, 50% of patients achieved a ≥20% reduction in serum ferritin within one year of therapy, and there was a notable decrease in cardiac iron load as indicated by MRI T2* measurements[4].

However, safety concerns remain, with agranulocytosis being a significant adverse reaction associated with deferiprone. This condition occurred in 1.7% of patients treated with the drug in clinical trials, highlighting the need for careful monitoring and post-marketing studies[3].

Market Analysis

Market Size and Growth

The deferiprone market is projected to grow steadily, with an expected market size of approximately USD 54 million by 2033, up from USD 38.3 million in 2023. This growth is anticipated at a Compound Annual Growth Rate (CAGR) of 3.5% from 2024 to 2033[2].

Market Segmentation

In 2023, the market was dominated by tablets, which held a substantial 78.6% market share, followed by oral solutions and capsules. The therapeutic focus was primarily on iron overload disorders, which commanded a 37.5% market share, driven by conditions such as hemochromatosis. Transfusional iron overload was the leading indication, accounting for 61.1% of the market share[2].

Regional Dominance

North America emerged as the dominant region in the deferiprone market, holding more than 39.1% of the market share in 2023. This dominance is attributed to the high prevalence of iron overload disorders, advanced healthcare infrastructure, and stringent regulatory environments that ensure the safety and efficacy of the drug[2].

Key Drivers and Challenges

Key Drivers

The growth of the deferiprone market is driven by several key factors:

• Rising Prevalence of Iron Disorders: Increasing incidence of conditions such as thalassemia and hemochromatosis.
• Ongoing R&D Investments: Continuous research and development aimed at improving the efficacy and safety of deferiprone.
• Emerging Markets: Potential for growth in emerging markets through strategic collaborations and personalized medicine approaches[2].

Challenges

Despite the growth potential, the market faces several challenges:

• Adverse Reactions: Agranulocytosis and other adverse reactions pose significant safety concerns.
• Patient Compliance Issues: Ensuring consistent patient adherence to treatment regimens.
• Stringent Regulatory Approvals: Navigating rigorous regulatory requirements for approval and post-marketing surveillance[2].

Opportunities and Trends

Emerging Markets and Strategic Collaborations

Emerging markets offer significant growth opportunities for deferiprone, particularly through strategic collaborations and the development of personalized medicine approaches. These initiatives can help in expanding the market reach and improving patient outcomes[2].

Pediatric Applications

There is a growing need for effective iron chelation therapies in pediatric patients, presenting an opportunity for deferiprone to be tailored for this demographic. This could involve specialized formulations and dosing regimens to meet the unique needs of pediatric patients[2].

Noteworthy Trends

The market is also influenced by trends such as the increasing adoption of early diagnosis and treatment protocols for iron overload, which drives the demand for effective chelation therapies like deferiprone[5].

Conclusion

Ferriprox (deferiprone) is a critical medication for managing chronic iron overload, particularly in patients with thalassemia and other transfusion-dependent conditions. The ongoing clinical trials and market projections indicate a promising future for this drug, despite the challenges it faces.

Key Takeaways

• Clinical Trials: Ongoing trials aim to enhance the efficacy of deferiprone by targeting TMPRSS6.
• Market Growth: The market is expected to grow at a CAGR of 3.5% from 2024 to 2033.
• Regional Dominance: North America leads the market due to high healthcare spending and advanced infrastructure.
• Key Drivers: Rising prevalence of iron disorders and ongoing R&D investments drive market growth.
• Challenges: Adverse reactions, patient compliance issues, and stringent regulatory approvals are significant challenges.

FAQs

Q: What is the primary use of Ferriprox (deferiprone)?

A: Ferriprox (deferiprone) is primarily used to treat chronic iron overload due to blood transfusions in patients with thalassemia and other conditions.

Q: What are the anticipated outcomes of the upcoming clinical trial for Ferriprox?

A: The trial aims to increase hepcidin levels and reduce iron overload by targeting TMPRSS6 with an antibody, potentially enhancing the efficacy of deferiprone.

Q: What are the major adverse reactions associated with Ferriprox?

A: Agranulocytosis is a significant adverse reaction associated with Ferriprox, occurring in 1.7% of patients treated with the drug.

Q: Which region dominates the Ferriprox market?

A: North America holds the largest market share due to its advanced healthcare infrastructure and high prevalence of iron overload disorders.

Q: What are the key drivers of the Ferriprox market growth?

A: The rising prevalence of iron disorders and ongoing R&D investments are key drivers of the Ferriprox market growth.

Sources

1. Thalassaemia International Federation: Ferriprox® (deferiprone) – TIF.
2. Market.us: Deferiprone Market Size, Share, and Growth | CAGR Of 3.5%.
3. FDA: 021825Orig1s000 - accessdata.fda.gov.
4. Ferriprox: Ferriprox (deferiprone): Efficacy in thalassemia for HCP.
5. Data Insights Market: Analyzing Deferasirox: Opportunities and Growth Patterns 2025-2033.