FDA APPROVES EXJADE TO REMOVE EXCESS IRON IN SOME PATIENTS

(The following is a reformatted version of a press release
issued by The U.S. Food and Drug Administration and received via
electronic mail. The release was confirmed by the sender.) 
Jan. 23, 2013 
Media Inquiries: Stephanie Yao, 301-796-0394,
stephanie.yao@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA 
FDA approves Exjade to remove excess iron in patients with
genetic blood disorder 
First imaging companion diagnostic to detect liver iron
concetration also cleared 
The U.S. Food and Drug Administration today expanded the
approved use of Exjade (deferasirox) to treat patients ages 10
years and older who have chronic iron overload resulting from a
genetic blood disorder called non-transfusion-dependent
thalassemia (NTDT). 
NTDT is a milder form of thalassemia that does not require
individuals to get frequent red blood cell transfusions.
However, over time, some patients with NTDT are still at risk
for iron overload that can lead to damage to vital organs. 
The FDA is also authorizing marketing of FerriScan as an imaging
companion diagnostic for Exjade. The agency previously cleared
FerriScan for measuring liver iron concentration (LIC), but its
use in Exjade clinical studies to select patients for therapy,
and to manage therapy, defined its role as an imaging companion
diagnostic necessary for Exjade’s safe and effective use.
FerriScan measures LIC non-invasively using magnetic resonance
imaging. 
An estimated 1,000 people in the United States have thalassemia,
according to the National Heart, Lung, and Blood Institute.
Thalassemia conditions can cause the body to make fewer healthy
red blood cells and less hemoglobin, a protein that carries
oxygen to all parts of the body and returns carbon dioxide to
the lungs so it can be exhaled. Some patients with thalassemia
require frequent transfusions of red blood cells to maintain an
acceptable level of hemoglobin. Iron overload is common in these
patients. 
Exjade was previously approved for treatment of chronic iron
overload due to blood transfusions in patients ages 2 years and
older, and this approval extends its use to treat patients with
NTDT who show iron overload. Exjade should be used in patients
with NTDT who have an LIC of at least 5 milligrams of iron per
gram of dry liver tissue weight. 
Exjade’s new indication is being approved under the FDA’s
accelerated approval program, which provides patients earlier
access to promising new drugs intended to treat serious or life-threatening illnesses while the company conducts additional
studies to confirm the drug’s clinical benefit. Exjade was
approved based on clinical data showing it can reduce LIC to
less than 5 mg/g dry weight, a surrogate endpoint that is judged
reasonably likely to predict a clinical benefit to patients. 
“Using our accelerated approval process, FDA is able to expedite
the availability of this drug to patients who need to reduce
excess iron,” said Richard Pazdur, M.D., director of the Office
of Hematology and Oncology Products in the FDA’s Center for Drug
Evaluation and Research. “Exjade is the first drug approved to
treat patients with NTDT who show iron overload.” 
The safety and effectiveness of Exjade to treat chronic iron
overload in patients with NTDT were established in two clinical
trials designed to measure the number of patients whose LIC was
reduced to less than 5 mg/g dry weight after 52 weeks of
treatment. In the first trial, 166 patients were randomly
assigned to receive 5 mg/kg of Exjade, 10 mg/kg of Exjade, or a
placebo daily. Results showed 15 percent and 27 percent of
Exjade-treated patients achieved the target LIC, respectively,
compared with 4 percent in placebo-treated patients. The second
trial contained 133 patients from the first study who received
an additional year of Exjade treatment or switched from placebo
to Exjade treatment. Thirty-five percent of the evaluable
patients in this extension trial achieved the target LIC. 
The FDA reviewed data for the FerriScan through the de novo
classification process, a regulatory pathway for medical devices
that are generally moderate-risk but are not comparable to an
already legally marketed device. The FDA’s granting of the de
novo request for FerriScan was based largely on data from the
Exjade clinical studies that used FerriScan LIC results as the
primary outcome measure. Additionally, investigators conducted a
230-patient study that found FerriScan results were as accurate
as liver biopsy for measuring LIC. 
“The FerriScan device is a non-invasive test that helps
physicians to select appropriate patients for Exjade therapy as
well as monitor their response to the drug, and discontinue
therapy when LIC reaches safe levels,” said Alberto Gutierrez,
Ph.D., director of the Office of In Vitro Diagnostics and
Radiological Health in the FDA’s Center for Devices and
Radiological Health. 
Exjade is marketed by East Hanover, N.J.-based Novartis.
FerriScan is marketed by Resonance Health, based in Australia. 
For more information: 
FDA: Office of Hematology and Oncology Products 
FDA: Approved Drugs: Questions and Answers 
FDA: Medical Devices
NHLBI: What are Thalassemias? 
The FDA, an agency within the U.S. Department of Health and
Human Services, protects the public health by assuring the
safety, effectiveness, and security of human and veterinary
drugs, vaccines and other biological products for human use, and
medical devices. The agency also is responsible for the safety
and security of our nation’s food supply, cosmetics, dietary
supplements, products that give off electronic radiation, and
for regulating tobacco products. 
Read our Blog: FDA Voice 
For more information contact:
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