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Deferasirox and Deferoxamine · 1 trial · 1 indication
Cardiac T2\* is the most sensitive and reproducible test in detecting myocardial iron load. A cardiac T2\* value of \<10 ms is defined as severe cardiac iron overload. Participants who do not have baseline T2\* or do not have any post-baseline T2\* are excluded from the analysis.
| Arm | Type | Description |
|---|---|---|
| Deferasirox | EXPERIMENTAL | Deferoxamine combination followed by Deferasirox monotherapy |
| Name | Type | Description |
|---|---|---|
| Deferasirox and Deferoxamine | DRUG | - |
Inclusion Criteria: * Patients with β-thalassemia major or Diamond-Blackfan anemia (DBA) or congenital sideroblastic anemia on chronic transfusion therapy * Myocardial T2\* value that is ≥ 5 and \< 10 ms * Left ventricular ejection fraction (LVEF) ≥ 56% as determined by Magnetic resonance imaging (...
Deferasirox is an oral iron chelator used to treat transfusional iron overload in beta-thalassemia and other chronic anemias, including sickle cell disease and myelodysplastic syndromes. It is also studied for cardiac iron overload. The drug binds excess iron in the body, allowing its excretion, thereby reducing iron-related organ damage.
Deferasirox targets iron by binding free and labile plasma iron to form a stable complex that is excreted primarily through the feces. This reduces total body iron stores in patients with transfusional iron overload. It is a small molecule iron chelator, not a conventional molecular target like a receptor or enzyme.
Deferasirox is developed by Novartis AG, a multinational pharmaceutical company headquartered in Basel, Switzerland. Novartis trades on the New York Stock Exchange under the ticker symbol NVS. The drug is also known by the brand name Exjade or Jadenu in some markets.
Deferasirox is in Phase 2 clinical development for myelodysplastic syndromes, according to the trial NCT00940602. However, it has completed Phase 3 trials for beta-thalassemia and transfusional iron overload. The drug is investigational for these indications and not yet approved in the studied settings.
Deferasirox has been studied in several completed trials. NCT00171210 evaluated iron chelation in beta-thalassemia patients with transfusional iron overload. NCT00171821 assessed efficacy and safety in transfusion-dependent iron overload. NCT00940602 is a Phase 2 study in myelodysplastic syndromes. All trials are completed.
Yes, Deferasirox is the same as Exjade, a brand name used by Novartis. It is also marketed as Jadenu in some regions. The generic name is deferasirox, and it is an oral iron chelator used to treat chronic iron overload from blood transfusions.