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Ferriprox (DEFERIPRONE)
Ferriprox works by binding to iron in the body and removing it, thereby reducing iron overload.
Ferriprox (Deferiprone) is a small molecule iron chelator developed by Apopharma Inc and currently owned by Chiesi. It targets deoxyhypusine hydroxylase to treat iron overload in patients with beta thalassemia. Ferriprox was FDA approved in 2011 and has three generic manufacturers. The commercial status of Ferriprox is not off-patent, but the number of generic manufacturers suggests that it may be approaching generic status. Key safety considerations include monitoring of liver function and potential side effects such as gastrointestinal issues.
At a glance
| Generic name | DEFERIPRONE |
|---|---|
| Sponsor | Chiesi |
| Drug class | Iron Chelator [EPC] |
| Target | Deoxyhypusine hydroxylase |
| Modality | Small molecule |
| Therapeutic area | Other |
| Phase | FDA-approved |
| First approval | 2011 |
Mechanism of action
Deferiprone is chelating agent with an affinity for ferric ions (iron III). Deferiprone binds with ferric ions to form neutral 3:1 (deferiprone:iron) complexes that are stable at physiological pH.
Approved indications
- Iron overload
- beta Thalassemia
Boxed warnings
- 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 )
Common side effects
- Nausea
- Abdominal pain/discomfort
- Vomiting
- Neutropenia
- Alanine Aminotransferase increased
- Arthralgia
- Agranulocytosis
- Diarrhea
- Dyspepsia
- Weight increased
- Aspartate Aminotransferase increased
- Increased appetite
Drug interactions
- algeldrate
- aluminum carbonate
- cabazitaxel
- carbamazepine
- carboplatin
- chlorambucil
- cidofovir
- clozapine
- docetaxel
- ferrous gluconate
- ferrous sulfate
- ganciclovir
Key clinical trials
- Cardiac MRI-guided Deferiprone Therapy for Acute Myocardial Infarction Patients (PHASE2)
- Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients (PHASE2)
- Conservative Iron Chelation as a Disease-modifying Strategy in Parkinson's Disease (PHASE2)
- Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia (PHASE2)
- Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis (PHASE2,PHASE3)
- Ferritin and Iron Burden in SAH sIRB (PHASE1,PHASE2)
- Early Screening and Treatment of Heart Complication in Sickle Cell Disease (PHASE2)
- Iron Chelation in the Prevention of Secondary Degeneration After Stroke (PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
| 11723874 | 2038-10-25 | Method of Use |
| 11357731 | 2038-10-25 | Method of Use |
| 10940115 | 2038-10-25 | Formulation |
| 10780055 | 2038-10-25 | Formulation |
| 11458103 | 2038-10-25 | Formulation |
| 10940116 | 2038-10-25 | Formulation |
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 |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Ferriprox CI brief — competitive landscape report
- Ferriprox updates RSS · CI watch RSS
- Chiesi portfolio CI