DEFERIPRONE
Deferiprone is an iron chelator that treats transfusional iron overload in adults with thalassemia syndromes. It is used when a patient's current chelation therapy is not working well enough to manage the excess iron. This medication helps patients who have accumulated too much iron in their bodies as a result of receiving frequent blood transfusions.
How DEFERIPRONE Works
This drug works by binding to ferric ions (iron III) to form stable complexes. These neutral complexes consist of three deferiprone molecules for every one iron ion. By binding to the iron in this way at physiological pH, the medication helps remove excess iron from the body.
Details
- Status
- Prescription
- First Approved
- 2019-02-08
- Routes
- ORAL
- Dosage Forms
- TABLET
DEFERIPRONE Approval History
What DEFERIPRONE Treats
2 indicationsDEFERIPRONE is approved for 2 conditions since its original approval in 2019. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Transfusional Iron Overload
- Thalassemia
DEFERIPRONE Boxed Warning
AGRANULOCYTOSIS AND NEUTROPENIA Deferiprone can cause agranulocytosis that can lead to serious infections and death. Neutropenia may precede the development of agranulocytosis. [see Warnings and Precautions (5.1) ] Measure the absolute neutrophil count (ANC) before starting deferiprone therapy and monitor regularly while on therapy. Interrupt deferiprone therapy if neutropenia develops. [see Warnings and Precautions (5.1) ] Interrupt deferiprone if infection develops, and monitor the ANC more fr...
WARNING: AGRANULOCYTOSIS AND NEUTROPENIA Deferiprone can cause agranulocytosis that can lead to serious infections and death. Neutropenia may precede the development of agranulocytosis. [see Warnings and Precautions (5.1) ] Measure the absolute neutrophil count (ANC) before starting deferiprone therapy and monitor regularly while on therapy. Interrupt deferiprone therapy if neutropenia develops. [see Warnings and Precautions (5.1) ] Interrupt deferiprone if infection develops, and monitor the ANC more frequently. [see Warnings and Precautions (5.1) ] Advise patients taking deferiprone to report immediately any symptoms indicative of infection. [see Warnings and Precautions (5.1) ] WARNING: AGRANULOCYTOSIS AND NEUTROPENIA See full prescribing information for complete boxed warning. Deferiprone can cause agranulocytosis that can lead to serious infections and death. Neutropenia may precede the development of agranulocytosis. ( 5.1 ) Measure the absolute neutrophil count (ANC) before starting deferiprone and monitor regularly while on therapy. ( 5.1 ) Interrupt deferiprone therapy if neutropenia develops. ( 5.1 ) Interrupt deferiprone if infection develops and monitor the ANC more frequently. ( 5.1 ) Advise patients taking deferiprone to report immediately any symptoms indicative of infection. ( 5.1 )
DEFERIPRONE Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to DEFERIPRONE
3 of 5FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
15 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT02164253 SAFEFAIRALS | 2012_69 2013-001228-21 | Ph 2 | completed | Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients |
| NCT02655315 FAIRPARKII | 2015_22 2015-003679-31, Grant agreement No 633190 | Ph 2 | completed | Conservative Iron Chelation as a Disease-modifying Strategy in Parkinson's Disease |
| NCT03293069 FAIR-ALS II | 2016_76 2017-003763-35 | Ph 2, Ph 3 | completed | Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis |
| NCT07023666 | INOVA-2023-159 | Ph 2 | recruiting | Early Screening and Treatment of Heart Complication in Sickle Cell Disease |
| NCT02728843 SKY results posted | LA48-0215 | Ph 2 | completed | Study of Parkinson's Early Stage With Deferiprone |
| NCT02443545 results posted | LA38-EXT | Ph 4 | terminated | Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias |
| NCT02041299 FIRST results posted | LA38-0411 | Ph 4 | terminated | Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias |
| NCT01825512 results posted | DEEP-2 | Ph 3 | completed | Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients |
| NCT01391520 | CRMD001-3001 | Ph 3 | withdrawn | Efficacy and Safety of Deferiprone in Patients With Chronic Kidney Disease Undergoing a Cardiac Catheterization and Receiving Contrast Agent |
| NCT02477631 | SHEBA-15-1908-DM-CTIL | Ph 2 | completed | Effect of Deferiprone on Oxidative-Stress and Iron-Overload in Low Risk Transfusion-Dependent MDS Patients |
| NCT01989455 results posted | LA42-0113 | Ph 1 | completed | A Blinded, Placebo-Controlled Study of the Safety and Pharmacokinetics of Single Doses of Intravenous Deferiprone in Healthy Volunteers |
| NCT01860703 results posted | LA37-1111 | Ph 4 | completed | Evaluation of Whether Deferiprone Affects QT Interval in Healthy Subjects |
| NCT01770652 results posted | LA39-0412 | Ph 4 | completed | An Open-label, Non-randomized, Parallel Group Study in Subjects With Mild, Moderate, Severe, or No Renal Impairment |
| NCT00943748 FAIR-PARK-I | 2008-006842-25 2008_19/0838, A90113-62 | Ph 2, Ph 3 | completed | Efficacy and Safety of the Iron Chelator Deferiprone in Parkinson's Disease |
| NCT00530127 | LA29-0207 | Ph 1, Ph 2 | completed | A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
DEFERIPRONE FDA Label Details
Indications & Usage
DEFERIPRONE is indicated for the treatment of Transfusional Iron Overload; Thalassemia.
WARNING: AGRANULOCYTOSIS AND NEUTROPENIA Deferiprone can cause agranulocytosis that can lead to serious infections and death. Neutropenia may precede the development of agranulocytosis. [see Warnings and Precautions (5.1) ] Measure the absolute neutrophil count (ANC) before starting deferiprone ther...
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FERRIPROX
Full clinical data, patents, trials, and competitive landscape for deferiprone.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.