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Deferiprone (DFP)
Deferiprone is an iron chelator that binds and removes excess iron from the body, reducing iron-induced tissue damage.
Deferiprone is an iron chelator that binds and removes excess iron from the body, reducing iron-induced tissue damage. Used for Iron overload in patients with thalassemia major, Iron overload in other chronic transfusion-dependent anemias.
At a glance
| Generic name | Deferiprone (DFP) |
|---|---|
| Also known as | DFP (Apotex, Canada) |
| Sponsor | Azienda Ospedaliera V. Cervello |
| Drug class | Iron chelator |
| Target | Ferric iron (Fe³⁺) |
| Modality | Small molecule |
| Therapeutic area | Hematology |
| Phase | FDA-approved |
Mechanism of action
Deferiprone is a bidentate chelating agent that forms stable complexes with ferric iron (Fe³⁺), facilitating its urinary excretion. By reducing labile iron pools and preventing iron-catalyzed free radical formation, it mitigates oxidative damage to organs such as the heart and liver in patients with chronic iron overload.
Approved indications
- Iron overload in patients with thalassemia major
- Iron overload in other chronic transfusion-dependent anemias
Common side effects
- Agranulocytosis
- Neutropenia
- Gastrointestinal disturbances (nausea, abdominal pain)
- Arthralgia
- Elevated liver enzymes
Key clinical trials
- Conservative Iron Chelation as a Disease-modifying Strategy in Parkinson's Disease (PHASE2)
- Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis (PHASE2, PHASE3)
- Study of Parkinson's Early Stage With Deferiprone (PHASE2)
- Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients (PHASE3)
- Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration (PHASE3)
- Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN) (PHASE3)
- Pharmacokinetic Study of Deferiprone in Paediatric Patients (PHASE2)
- Evaluation of the Safety, Efficacy, and Pharmacokinetics of Intravenous Deferiprone in HIV-Positive Subjects (PHASE1)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |