Novartis drug Exjade® first treatment approved by FDA for chronic iron overload in patients with non-transfusion-dependent

    Novartis drug Exjade® first treatment approved by FDA for chronic iron
       overload in patients with non-transfusion-dependent thalassemia

-- Pivotal placebo-controlled study data show Exjade significantly decreases
iron burden in NTDT patients versus placebo, with similar overall adverse
event rate1

-- Patients with NTDT accumulate excess iron increasing their risk of
complications, including liver fibrosis, cirrhosis, blood clots, and bone and
vascular disease2

-- At least three quarters of a million people worldwide have NTDT3-5 and many
patients are undiagnosed until serious symptoms arise6

PR Newswire

EAST HANOVER, N.J., January 23, 2013

EAST HANOVER, N.J., January 23, 2013 /PRNewswire/ --Novartis announced today
that the US Food and Drug Administration (FDA) has approved Exjade^®
(deferasirox) for the treatment of chronic iron overload in patients 10 years
of age and older with non-transfusion-dependent thalassemia (NTDT) syndromes
and with a liver iron concentration of at least 5 mg of iron per gram dry
weight and a serum ferritin measurement greater than 300 micrograms per liter.
Exjade is the first treatment indicated for patients with these types of
thalassemia in the United States.

The approval is based on results from the first prospective placebo-controlled
study of iron chelation in NTDT patients, THALASSA, which showed a significant
dose-dependent decrease in iron burden compared to placebo (p<0.001)^1. In
this pivotal study, Exjade significantly reduced the concentration of iron in
the liver, known as liver iron concentration (LIC), as well as the amount of
iron anywhere in the body, measured by serum ferritin^1. The overall adverse
event rate for Exjade was similar to the placebo arm^1.

"Patients with NTDT can suffer severe and life-changing complications from
chronic iron overload," said Elliott Vichinsky, MD, Medical Director,
Hematology/Oncology, Children's Hospital and Research Center, Oakland,
California. "In these thalassemia patients, excess iron starts to accumulate
at birth yet is often undetected until serious symptoms appear in early
adulthood. With this approval of Exjade, physicians will be able to offer NTDT
patients a treatment option, helping fulfill a critical unmet need."

Thalassemia refers to a diverse group of genetic disorders that affect red
blood cell production, causing anemia. Unlike patients with other types of
thalassemia, those with NTDT syndromes don't require regular transfusions, a
significant cause of chronic iron overload. However, even without
transfusions, NTDT patients still accumulate excess iron through intestinal
absorption, leading to debilitating health complications like liver fibrosis
and cirrhosis, blood clots, bone disease, pulmonary hypertension, and vascular
and endocrine diseases^2,7.

"For years, Exjade has effectively treated chronic iron overload in transfused
thalassemia patients," said Alessandro Riva, Global Head, Oncology Development
and Medical Affairs, Novartis Oncology. "Now, for the first time, thalassemia
patients who do not receive transfusions but suffer the same debilitating
effects from chronic iron overload, have an approved treatment option."

According to published studies, at least three quarters of a million people
worldwide have NTDT syndromes, although as understanding of the disease
increases it is probable the number will grow^3-5. Because NTDT patients are
not symptomatic at birth, when most thalassemias are diagnosed, they are often
underdiagnosed and undertreated^6. Many complications associated with chronic
iron overload begin to appear as early as age 10 and become increasingly
common as patients reach their 20s or 30s^8. Most NTDT patients are of South
and Southeast Asian, Mediterranean or Middle Eastern origin, with immigration
broadening the global prevalence^6,9.

About the THALASSA Study
The THALASSA trial showed that Exjade at a 10 mg/kg per day starting dose
significantly reduced LIC from baseline by 3.8 mg of iron per gram of liver
dry weight (Fe/g dw) compared to an increase of 0.38 mg Fe/g dw in patients
receiving placebo after 52 weeks of treatment (p<0.001)^1. The study also
determined that a 10 mg/kg per day dose was superior to a 5 mg/kg per day dose
(p=0.009)^1. Additional research has also demonstrated Exjade continues to
provide benefit over the longer term, with LIC levels reduced by 7.14 mg Fe/g
dw from baseline after 24 months of treatment^10. The most common reported
adverse events (at least 5% in any Exjade or placebo group) were nausea, skin
rash, diarrhea and headache^1.

About Exjade
In the US, Exjade is now indicated for the treatment of chronic iron overload
in patients 10 years of age and older with non-transfusion-dependent
thalassemia (NTDT) syndromes and with a liver iron concentration (LIC) of at
least 5 mg of iron per gram dry weight (mg Fe/g dw) and a serum ferritin
measurement greater than 300 micrograms per liter. The basis of this
indication is data showing achievement of an LIC less than 5 mg Fe/g dw after
treatment with Exjade. An improvement in survival or disease-related symptoms
has not been established.

Since 2005, Exjade has been approved in the US for the treatment of chronic
iron overload due to blood transfusions in adult and pediatric patients (aged
2 years and over). Exjade is approved in over 100 countries including the US,
Switzerland, Japan and the countries comprising the European Union. The
approved indication may vary depending upon the individual country.

Exjade Important Safety Information
Exjade is contraindicated in patients with creatinine clearance <40 mL/min or
serum creatinine >2 times the age-appropriate upper limit of normal; poor
performance status and high-risk myelodysplastic syndromes or advanced
malignancies: platelet counts <50 x 10^9/L; known hypersensitivity to
deferasirox or any component of Exjade.

There have been postmarketing reports of acute renal failure, hepatic failure
and cytopenias. Renal failure requiring temporary or permanent dialysis, renal
tubulopathy and interstitial nephritis have been reported. Upper
gastrointestinal ulceration and hemorrhage, sometimes fatal, have been
reported. Caution should be used in elderly patients due to a higher frequency
of adverse reactions. Exjade is not recommended in patients with a short life
expectancy (e.g., high-risk myelodysplastic syndromes), especially when
co-morbidities could increase the risk of adverse events.

Skin rashes, serious hypersensitivity reactions, decreased hearing and lens
opacities have been reported. The most common adverse reactions are nausea,
vomiting, diarrhea, abdominal pain, rash, non‐progressive increases in serum
creatinine, increased transaminases, abdominal distension, constipation,
dyspepsia, proteinuria and headache.

Please visit www.exjade.com. The full prescribing information including the
Boxed Warning for Exjade is available at
http://www.pharma.us.novartis.com/product/pi/pdf/exjade.pdf.

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About Novartis
Novartis Pharmaceuticals Corporation researches, develops, manufactures and
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References

1.Taher A, Porter J, Viprakasit V,  et al. Deferasirox significantly reduces
    iron overload in non-transfusion-dependent thalassemia: 1-year results
    from a prospective, randomized, double-blind, placebo-controlled study.
    Blood. 2012. Published online before print May 15, 2012.
2.Musallam KM, Cappellini MD, Wood JC, et al. Iron overload in
    non-transfusion-dependent thalassemia: a clinical perspective. Blood
    Reviews. 2012:26S:S16-S19.
3.Vichinsky E. Hemoglobin E syndromes. Hematology Am Soc Hematol Educ
    Program. 2007;79-83.
4.Weatherall DJ. The definition and epidemiology of
    non-transfusion-dependent thalassemia. Blood Reviews. 2012:26S:S3-S6.
5.Vichinsky EP. Changing patterns of thalassemia worldwide. Ann NY Acad Sci.
    2005;1054:18-24.
6.Thalassaemia International Federation. The Thalassaemia International
    Federation's (TIF) New Focus: Addressing the Management of
    Non-Transfusion-Dependent Thalassaemias (NTDT). Position Paper 5.2. March
    20, 2012. Accessed at:
    http://www.thalassaemia.org.cy/pdf/NTDT_Position_Paper_Final.pdf.
7.Musallam KM, Cappellini MD, Wood JC, Motta I, Graziadei G, Tamin H, Taher
    AT. Elevated liver iron concentration is a marker of increased morbidity
    in patients with β thalassemia intermedia. Haematologica. 2011
    Nov;96(11):1605-12.
8.Taher AT. Age-related complications in treatment-naïve patients with
    thalassemia intermedia. Brit J Haematol. 2010;150:486-489.
9.Taher A, Cappellini MD, Musallam KM. Recent advances and treatment
    challenges in patients with non-transfusion-dependent thalassemia. Blood.
    2012;26S:S1-2.
10.Taher AT, Porter JB, Viprakasit V et al. Deferasirox continues to reduce
    iron overload in non-transfusion-dependent thalassemia: a one-year,
    open-label extension to a one-year, randomized double-blind,
    placebo-controlled study (THALASSA). Poster presented at the 54^th
    American Society of Hematology Annual Meeting and Exposition in Atlanta,
    GA (8-11 December 2012). Abstract #3258.

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