The FDA has approved Novartis' new drug application (NDA) for Exjade, an oral
alternative to a much more difficult treatment for patients suffering chronic
iron overload from blood transfusions.
The new drug should encourage more blood disorder patients to use iron chelation therapy, Novartis said.
The FDA approved Exjade (deferasirox) to treat chronic iron overload in patients age two and older. Patients with certain types of anemia, sickle cell disease and other conditions require frequent blood transfusions, which can cause iron to build up in the body and damage the liver, heart and endocrine glands. Exjade is an iron chelator, a drug that binds to the iron in the body and helps excrete it.
Exjade, a tablet dissolved in water or juice, would make it much easier to treat iron overload. The standard treatment, Desferal (deferoxamine) also made by Novartis is injected via a portable pump for eight to 12 hours a day, five to seven days a week, as long as the patient receives blood transfusions or has excess iron in the body.
Consequently, once-a-day oral Exjade should encourage more iron overload patients to seek treatment, Novartis said. In 2004, Desferal had U.S. sales of $43 million and global sales of $115 million.
"Until now, many transfused patients have developed serious complications from iron overload despite the availability of effective chelation therapy," Elliott Vichinsky, director of hematology/oncology at Children's Hospital & Research Center in Oakland, Ca., told FDAnews. "This occurred because the only available therapy required daily injections accompanied by 12 hour infusions. The invasiveness of the therapy resulted in a poor quality of life and difficulty with compliance. With Exjade, we have a treatment option for children and adults that will minimize or replace the use of injections and infusions."