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Kim Fox
Novartis Oncology
+1 862 778 7692 (direct)
kim.fox@novartis.com
Novartis International AG
Novartis Global Communications
CH-4002 Basel
Switzerland
http://www.novartis.com
Corinne Hoff
Novartis Global Media Relations
+41 61 324 9577 (direct)
+41 61 324 2200 (main)
corinne.hoff@novartis.com
MEDIA RELEASE •
COMMUNIQUE AUX MEDIAS • MEDIENMITTEILUNG
Exjade®, a breakthrough once-daily oral iron chelator, receives first approval
worldwide in the US
• Exjade offers new alternative to
burdensome standard therapy in children and
adults who require blood transfusions for chronic anemias
• Approval makes iron chelation more accessible to
patients suffering from
diseases such as thalassemia, sickle cell and myelodysplastic syndromes
Basel, November 3, 2005 – Novartis announced today the first approval worldwide
for Exjade®
(deferasirox) – the first and only once-daily oral iron chelator – by the US
Food and Drug Administration. Exjade has been approved for the treatment of
chronic iron overload due to blood transfusions in adults and children age two
and older. Exjade is the only iron chelator administered as a drink (the tablets
are dispersed in a glass of orange juice, apple juice or water), compared to the
current standard of care, which often requires a subcutaneous infusion lasting
eight to 12 hours per night, for five to seven nights a week for as long
as the patient continues to receive blood transfusions or has excess iron within
the body. As a result, many patients may have stopped or avoided iron chelation
therapy, thus risking the toxic effects of iron overload.
The approval of Exjade is expected to greatly enhance the acceptance of iron
chelaton therapy, especially for children, and offer a new alternative to the
burdensome continuous infusion therapy.
“The approval of Exjade is an advance for people like me, who have been having
blood transfusions
and iron chelation for most of our lives,” said Jasmine Williams, who has sickle
cell disease and participated in a clinical trial of Exjade. “With Exjade I
won’t have to worry about using my needle and pump. I just have to drink my
medicine and not think about it again until the next day. Exjade
has really made a difference in my life.”
Iron overload is a potentially life-threatening and unavoidable consequence of
frequent blood transfusions used to treat certain types of rare chronic blood
disorders, including thalassemia and sickle cell disease, as well as other rare
anemias and myelodysplastic syndromes. Signs of iron overload may be detected
after transfusion of about 20 units of blood. If left undiagnosed or
untreated, the excess iron in the body is likely to lead to damage to the liver,
heart and endocrine glands. The body has no inherent mechanism to remove excess
iron, so iron chelation is used as an effective treatment for
transfusion-related iron overload.
“We believe Exjade is a significant breakthrough that will fill an important gap
in protecting patients from the cumulative toxicity of iron overload by making
iron chelation therapy much more acceptable. Until now, patients may have
avoided the potentially life-saving benefits of iron chelation because the
standard therapy can be difficult to use,” said David Epstein, CEO of Specialty
Medicines and President of Novartis Oncology.
Exjade was approved after being granted
priority review by the FDA and also after the Blood
Products Advisory Committee to the FDA voted unanimously to give Exjade a
positive recommendation for approval. Designated an orphan drug in the US,
Switzerland, Australia, and the EU, Exjade has also been granted a priority
review in Switzerland, Canada, Australia and New
Zealand. Additional regulatory submissions have been made around the world.
Filing data
The Exjade filings were based on the results of a clinical trials program that
included a Phase III trial, which showed that after one year Exjade produced
reductions in liver iron concentration (LIC).
The clinical trials, which included more than 1,000 adults and children, were
part of the largest prospective global clinical trials program ever implemented
for an investigational iron chelator. LIC is an indicator for body iron content
in patients receiving blood transfusions. It is a measure of iron
accumulation in the liver. The studies demonstrated that Exjade, at 20-30
mg/kg/day, led to the maintenance or reduction of iron burden in transfused
patients with thalassemia and sickle cell disease as well as other rare anemias
and myelodysplastic syndromes. In the clinical studies, Exjade was generally
well tolerated, with the most frequently reported adverse events being nausea,
vomiting, diarrhea, abdominal pain, skin rash and increases in serum creatinine.
As with deferoxamine (Desferal®), cases of ocular and auditory disturbances have
been reported.
Mild, non-progressive increases in serum creatinine, mostly within the normal
range, occur in about one-third of Exjade treated patients. These are
dose-dependent, often resolve spontaneously and can sometimes be alleviated by
reducing the dose. Serum creatinine should be assessed before initiating
therapy and should be monitored monthly thereafter to determine if dose
modification or discontinuation is necessary. Liver function should be monitored
monthly and if there is an unexplained, persistent, or progressive increase in
serum transaminase levels Exjade should be interrupted or discontinued.
About iron chelation
In iron chelation, an agent binds to iron in the body and tissues and helps
remove it through the urine and/or feces. The goal of iron chelation therapy is
to remove the amount of iron administered in transfusions and, as required, to
reduce the existing iron burden. In many patients the need for transfusions may
be life-long. To date, only deferoxamine is globally available for the
first-line treatment of transfusion related iron overload. While deferoxamine is
effective, due to its burdensome administration, many patients do not undergo
iron chelation therapy, exposing themselves to the dangers of iron overload.
Novartis believes the approval of Exjade will not only help patients currently
receiving iron chelation, but also extend the benefits of iron chelation to
those
not currently undergoing therapy.
The foregoing release contains forward-looking statements that can be identified
by terminology such as “significant breakthrough/breakthrough,” “will fill an
important gap,” “first,” “is expected,” “is likely,” “potentially,” “greatly
enhance,” or similar expressions, or by express or implied
discussions regarding potential additional marketing approvals or future sales
of Exjade. Such forward-looking statements involve known and unknown risks,
uncertainties and other factors that may cause actual results with Exjade to be
materially different from any future results, performance or achievements
expressed or implied by such statements. There can be no guarantee that Exjade
will receive any additional marketing approvals in any other countries, or that
it will reach any particular sales levels. In particular, management's
expectations regarding commercialization of Exjade could be affected by, among
other things, additional analysis of Exjade clinical data; new clinical data;
unexpected clinical trial results; unexpected regulatory actions or delays or
government regulation generally; the company's ability to obtain or maintain
patent or other proprietary
ntellectual property protection; competition in general; increased
government, industry, and general public pricing pressures; and other risks and
factors referred to in the Company's current Form 20-F on file with the U.S.
Securities and Exchange Commission. Should one or more of these risks or
uncertainties materialize, or should underlying assumptions prove incorrect,
actual results may vary materially from those anticipated, believed, estimated
or expected. Novartis is providing the information in this press release as of
this date and does not undertake any obligation to update any
forward-looking statements contained in this press release as a result of
new information, future events or otherwise.
For prescribing information on deferoxamine (Desferal®) please contact your
local Novartis affiliate.
More Information For Health-Care Providers:
Some clinical trials with Exjade are ongoing. To learn more about Exjade
clinical trials, health-care providers can call +44 (0) 1506 814899 or
+1-800-340-6843 in the US
About Novartis
Novartis AG (NYSE: NVS) is a world leader in pharmaceuticals and consumer
health. In 2004, theGroup's businesses achieved net sales of USD 28.2 billion
and pro forma net income of USD 5.6 billion. The Group invested approximately
USD 4.1 billion in R&D. Headquartered in Basel, Switzerland, Novartis Group
companies employ about 91,700 people and operate in over 140 countries around
the world.
For further information please consult
http://www.novartis.com.
# # #
Media contacts
Kim Fox
Novartis Oncology
+1 862 778 7692 (direct)
kim.fox@novartis.com
Corinne Hoff
Novartis Global Media Relations
+41 61 324 9577 (direct)
+41 61 324 2200 (main)
corinne.hoff@novartis.com
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