Oral Iron Chelator Formula Gets FDA Nod

An oral formulation of deferasirox is approved for the treatment of chronic iron overload due to blood transfusions.

The FDA has approved Novartis's deferasirox (Jadenu), an oral formulation of the manufacturer’s Exjade, for the treatment of chronic iron overload due to blood transfusions in patients aged 2 years and older, and chronic iron overload in non-transfusion-dependent thalassemia syndromes (NTDT) in patients aged 10 years and older.

Chronic iron overload is a life-threatening cumulative toxicity that results from blood transfusions used to treat sickle cell disease, myelodysplastic syndromes, thalassemia, and other conditions. Chronic iron overload also can occur in patients with NTDT due to increased iron absorption in the stomach and intestines.

Patients with chronic iron overload must manage their condition with an iron chelator such as Exjade. While Exjade must be mixed in liquid and taken on an empty stomach, Jadenu can be swallowed whole and taken with or without a light meal.

“Jadenu is an exciting development for patients with chronic iron overload who have been eager for alternative treatment options,” said Elliott Vichinsky, MD, Director of Hematology and Oncology at the University of California, San Francisco Benioff Children's Hospital Oakland, in a press release. “Taking iron chelation therapy every day has sometimes been a challenge for them. The administration of Jadenu oral tablets once a day is simple.”

The FDA gave its nod to Jadenu under accelerated approval based on a reduction of liver iron concentrations and serum ferritin levels in trial participants. The drug’s continued approval may be dependent upon verification and description of clinical benefit in confirmatory trials.

“Novartis has had a long-term commitment to improving the lives of patients with chronic iron overload,” said Novartis Oncology President Bruno Strigini in a press release. “Exjade transformed iron chelation therapy. We responded to feedback from patients and their physicians, and now Jadenu, by simplifying treatment administration, offers an important new option to help meet these patients' needs.”