FDA APPROVES NOVARTIS' EXJADE
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."