The Food and Drug Administration has approved the use of Jadenu (deferasirox) tablets for the treatment of patients as young as two with chronic iron overload due to blood transfusions, and for the treatment of patients as young as 10 with chronic iron overload in non-transfusion-dependent thalassemia syndrome (NTDT).
Jadenu, made by Novartis, is a new oral formulation of Exjade (which also has the generic name deferasirox) tablets for oral suspension. Exjade is a dispersible tablet that must be mixed in liquid and taken on an empty stomach, whereas Jadenu oral tablets can be swallowed whole, with or without a light meal. Long-term daily chelation therapy is often required for patients with myelodysplastic syndromes, sickle cell disease, or thalassemia who need repeated blood transfusions.
Jadenu was approved under the FDA's accelerated approval program, which allows approval of a drug to treat a serious or life-threatening disease based on clinical data showing the drug has an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients. The program provides earlier patient access to promising new drugs while the company conducts confirmatory clinical trials. Continued approval for these indications for Jadenu may be contingent upon verification and description of clinical benefit in confirmatory trials.
This FDA approval for Jadenu is based on equivalence to Exjade as demonstrated in pharmacokinetic studies of healthy volunteers, and there are ongoing studies to find out how Jadenu works over a longer period of time.
The most common side effects for deferasirox are: nausea, vomiting, stomach pain, diarrhea, rash, and increased kidney laboratory values. Other serious reactions for deferasirox (since it has been marketed) have included neutropenia, agranulocytosis, worsening anemia, thrombocytopenia, serious allergic reactions (including swelling of throat), severe skin reactions (including Stevens Johnson syndrome and erythema multiforme), decreased hearing, and vision changes.
Jadenu is not intended for use by patients with preexisting severe kidney and liver problems; high-risk myelodysplastic syndromes; advanced cancer; low platelet counts; or an allergy to Jadenu.