The FDA has approved an oral tablet formulation of deferasirox (Jadenu [ jade' new] – Novartis) for once-daily treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients ≥2 years old or chronic iron overload in patients ≥10 years old with non-transfusion-dependent thalassemia syndromes. A once-daily, oral tablet for suspension formulation of deferasirox (Exjade) was approved in 2005 for the same indications.1 Jadenu and Exjade are the only once-daily oral formulations for iron chelation available in the US.

No new clinical trials were required for approval of Jadenu, which was based on earlier clinical trials with Exjade. The most common adverse effects reported with use of deferasirox in clinical trials were diarrhea, vomiting, nausea, abdominal pain, rash, neutropenia, and increases in serum creatinine. Severe skin reactions, including Stevens-Johnson syndrome and erythema multiforme, have been reported rarely. Hearing loss and ocular disturbances, including cataracts, have been reported with deferasirox; patients taking the drug should have annual auditory and ophthalmic exams. Gastrointestinal ulceration and hemorrhage and renal and hepatic toxicity have also occurred.

1. Deferasirox (Exjade): a new iron chelator. Med Lett Drugs Ther 2006; 48:35.

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The FDA has approved deferiprone (de fer’ i prone; Ferriprox – ApoPharma), an oral chelating agent available in Europe since 1999, for use as a second-line treatment of iron overload due to blood transfusions (transfusional hemosiderosis) in patients with thalassemia. Two other iron chelating agents, deferoxamine (Desferal, and others), which is usually administered subcutaneously, and oral deferasirox (Exjade), are available for use as first-line therapy in the US.

Deferasirox (Exjade - Novartis), an oral chelating agent, recently received accelerated approval from the FDA as an orphan drug for oral treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients >2 years old. Deferasirox is a tridentate (2 molecules of deferasirox bind to one atom of iron) chelating agent with high affinity for iron. It has much lower affinity for zinc and copper.