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Ferriprox (deferiprone)
Ferriprox (deferiprone) is a Iron chelator Small molecule drug developed by ApoPharma. It is currently FDA-approved for Transfusional iron overload in patients with chronic anemia, Iron overload in thalassemia major.
Deferiprone is an iron chelator that binds excess iron in the body and facilitates its excretion through urine.
Ferriprox (deferiprone) is a small molecule used to treat various conditions, including iron overload, sickle cell disease, and other anemias, as well as Parkinson's disease. Its mechanism of action is currently unknown.
At a glance
| Generic name | Ferriprox (deferiprone) |
|---|---|
| Sponsor | ApoPharma |
| 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⁺), preventing iron-catalyzed free radical formation and tissue damage. It crosses cell membranes to access intracellular iron stores, particularly in cardiac myocytes and hepatocytes, and the iron-deferiprone complex is readily excreted in the urine, reducing total body iron burden in iron-overload conditions.
Approved indications
- Transfusional iron overload in patients with chronic anemia
- Iron overload in thalassemia major
Common side effects
- Agranulocytosis
- Neutropenia
- Arthralgia
- Gastrointestinal disturbance (nausea, abdominal pain)
- Zinc deficiency
Key clinical trials
- Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients (PHASE2)
- Early Screening and Treatment of Heart Complication in Sickle Cell Disease (PHASE2)
- Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children (PHASE4)
- Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias (PHASE4)
- Ferrochelating Treatment in Patients Affected by Neurodegeneration With Brain Iron Accumulation (NBIA) (PHASE2)
- Compassionate Use of Deferiprone in Patients With PKAN
- Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias (PHASE4)
- Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Ferriprox (deferiprone) CI brief — competitive landscape report
- Ferriprox (deferiprone) updates RSS · CI watch RSS
- ApoPharma portfolio CI
Frequently asked questions about Ferriprox (deferiprone)
What is Ferriprox (deferiprone)?
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What drug class is Ferriprox (deferiprone) in?
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What does Ferriprox (deferiprone) target?
Related
- Drug class: All Iron chelator drugs
- Target: All drugs targeting Ferric iron (Fe³⁺)
- Manufacturer: ApoPharma — full pipeline
- Therapeutic area: All drugs in Hematology
- Indication: Drugs for Transfusional iron overload in patients with chronic anemia
- Indication: Drugs for Iron overload in thalassemia major
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing