Novartis has filed regulatory submissions for Exjade® (deferasirox), a once-daily oral iron chelator for the treatment of chronic iron overload due to blood transfusions, in the United States and the European Union (EU). Submissions in other countries will follow shortly.
Exjade, also known as investigational agent ICL670, has been granted fast-track status in the U.S. and Switzerland. Priority review has been requested in the U.S. Furthermore, Exjade has received Orphan Drug status in the U.S., EU and Australia.
An easy to administer novel oral iron chelator, Exjade is simply taken once daily, after dispersing tablets in a glass of water. Exjade was developed to extend the benefits of iron chelation to a greater number of patients receiving blood transfusions and to address the needs of thousands of adult and pediatric patients who have been using Desferal® (deferoxamine). Patients have been frustrated for years by the inconvenience and pain that can result from daily insertion of the deferoxamine infusion needle. In many patients, the need for transfusion and chelation therapy may be life-long.
“Novartis has demonstrated a long-term commitment to help improve the lives of patients at risk for iron overload. First by developing a highly effective drug, deferoxamine, and then by conducting research on hundreds of new compounds to find an easy-to-take oral alternative to this product,” said Diane Young, MD, vice president and global head of Clinical Development at Novartis Oncology. “We understand the needs of patients and know that the burdensome administration of deferoxamine limits its use. In an effort to bring the benefits of effective iron chelation to more patients, we will work diligently with health authorities to expedite the approval of this important advancement.”
Iron overload is a life-threatening cumulative toxicity which results from lifesaving blood transfusions required to treat certain types of anemias and other disorders, including thalassemia, sickle cell disease, other rare anemias, and myelodysplastic syndromes. If left undiagnosed or untreated, iron overload can lead to damage to the liver, heart and endocrine glands. Transfused patients may require concomitant removal of excess iron with a type of drug therapy called iron chelation, to treat iron overload.
Deferoxamine, the current standard of care in iron chelation, is effective but typically requires subcutaneous infusion lasting eight to twelve hours per day, for five to seven days a week for as long as the patient continues to receive blood transfusions.
“Desferal infusion therapy is difficult for children, who dislike needles and fear the pain. But it is extremely burdensome for older teens and young adults, who often will not comply with therapy, even though they see first-hand the risks of iron overload including deformities, organ failure, and premature death,” said Durhane Wong-Rieger, PhD, president and chief executive officer, Anemia Institute for Research and Education. “For many patients, the infusions, lasting up to 12 hours per day, stigmatize them, severely limit their social activities and interfere with intimate relationships. Patients and caregivers are very hopeful for a once-daily oral chelator that will enable them to continue to treat iron overload.”
