FDA Approves Simpler Formulation for Administering Deferasirox

The FDA has approved deferasirox (Jadenu) for the treatment of chronic iron overload, a condition that can affect patients with myelodysplastic syndromes (MDS), sickle cell disease, and thalassemia.

Jadenu is a new oral formulation of Exjade tablets for oral suspension. While Exjade is a dispersible tablet that must be mixed in liquid and taken on an empty stomach, Jadenu can be taken in a single step, with or without a light meal, simplifying administration of treatment for chronic iron overload.

Jadenu contains the same active ingredient as Exjade, which is currently the most-prescribed chelator in the United States, according to the drug’s manufacturer, Novartis.

"Novartis has had a long-term commitment to improving the lives of patients with chronic iron overload," Bruno Strigini, President, Novartis Oncology, said in a statement. “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."

Chronic iron overload is a life-threatening cumulative toxicity that results from blood transfusions required to treat sickle cell disease, MDS, thalassemia, and other conditions. Chronic iron overload also can occur in patients with non-transfusion—dependent thalassemia syndromes due to increased iron absorption in the stomach and intestines. If left untreated, chronic iron overload can damage the liver and heart.

“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 (UCSF) Benioff Children's Hospital Oakland and Professor, UCSF School of Medicine, said in a statement. “Taking iron chelation therapy every day has sometimes been a challenge for them. The administration of Jadenu oral tablets once a day is simple.”

Deferasirox has a Boxed Warning, The drug may cause serious kidney problems, liver problems, and bleeding in the stomach or intestines, and in some cases, these problems were fatal. Kidney problems occurred more in patients with multiple medical conditions and those who were very ill due to their disease. Bleeding in the stomach or intestines occurred more often in elderly patients, and liver problems were more likely to happen in patients older than 55 years.