The clinical evidence supporting deferasirox spans pivotal randomised trials, long-term extension studies and real-world post-authorisation experience across thalassemia, sickle-cell disease and myelodysplastic syndromes (MDS).
Pivotal Programme Overview #
| Study | Population | Key Endpoint | Outcome |
|---|---|---|---|
| Pivotal randomised vs. deferoxamine | Adults & pediatrics with transfusion-dependent thalassemia | Change in LIC after 1 year | Non-inferior LIC reduction at appropriate doses (20-30 mg/kg/day); ferritin reductions in heavily-loaded subset |
| Sickle-cell disease programme | Adults & pediatrics with SCD on chronic transfusion | LIC reduction; ferritin trend | Significant reductions in LIC and ferritin over 12 months |
| EPIC (Evaluation of Patients' Iron Chelation with Exjade) | Large open-label registry across multiple anemias | Real-world ferritin reduction | Demonstrated population-level ferritin reduction at clinically used doses |
| MDS sub-population | Lower-risk MDS with transfusional iron overload | Ferritin reduction; safety signals | Ferritin reductions; tolerability considerations including cytopenia monitoring |
| Cardiac iron studies | Patients with myocardial iron loading (T2* < 20 ms) | Cardiac T2* improvement | Improvement in cardiac T2* with sustained multi-year therapy |
Representative Numerical Outcomes #
- Liver iron concentration (LIC): reductions of approximately 1-9 mg Fe/g dry weight over 12 months at 20-30 mg/kg/day, dose-dependent.
- Serum ferritin: mean reductions in the range of 500-1500 ng/mL across 12 months in heavily-loaded populations on adequate doses.
- Cardiac T2*: sustained therapy associated with improvement from baseline values < 20 ms toward normal range over 24-36 months.
Note: precise numerical results depend on baseline iron burden, transfusion intensity and dose adherence; numerical ranges above are summary representations of published programme data and should not be substituted for the locally approved prescribing information.
Long-Term Safety Findings #
Long-term extension studies have characterised the safety profile of sustained chelation including the renal, hepatic, gastrointestinal and rare severe cutaneous reactions described on the Safety Profile page. The benefit-risk balance of long-term chelation in transfusion-dependent populations is well-established.
Application to Tender Documentation #
Clinical-study summary tables - including representative LIC and ferritin numerical outcomes - are included in our Module 2.5 Clinical Overview within the CTD dossier and can be furnished to procurement / tender teams under CDA. Comparative bioequivalence data versus the reference product is also available.