Deferasirox (CGP-72670) is an oral iron chelating agent used to treat chronic iron overload.

Orally administered Deferasirox in rats achieves an absorption rate of at least 75% with a bioavailability of 26%. Monotherapy with Deferasirox modestly increases the survival time in IPA mice. When administered, either intravenously or orally, Deferasirox mainly circulates in the blood as its unaltered form and its iron complex, with a 99.2% binding rate to plasma proteins. Deferasirox significantly enhances survival rates and reduces tissue fungal burden in mice with diabetic ketoacidosis or neutropenia suffering from mucormycosis, showing effects comparable to those of liposomal Amphotericin B. Additionally, Deferasirox bolsters the host inflammatory response against mucormycosis. The combined use of Deferasirox and liposomal Amphotericin B synergistically improves survival rates and decreases fungal load in tissues affected by mucormycosis.

Deferasirox exhibits antifungal activity against Aspergillus with MIC (Minimum Inhibitory Concentration) and MFC (Minimum Fungicidal Concentration) values of 25 and 50 mg/L, respectively. It effectively chelates iron ions from Rhizopus species, showing antiviral activity in vitro against 28 out of 29 Mucorales clinical isolates at concentrations much lower than those achievable in clinical serum levels. Deferasirox significantly inhibits the activity of NF-κB by chelating its active subunit p65 in an inactive form within the cytoplasm in 28 out of 40 peripheral blood samples. Additionally, it inhibits three human myeloid cell lines (K562, U937, and HL60) with IC50 values ranging from 17 to 50 mM.

Deferasirox is dissolved in DMSO. HL-60 or KG-1 cells are treated with 0, 5, 10, 50 μM of deferasirox for 24 or 48 h, and proliferation is determined by an MTT assay[2].