Novartis International AG : Novartis drug Exjade® first treatment approved by FDA for chronic iron overload in patients with

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

Novartis International AG / Novartis drug Exjade® first treatment approved by
FDA for chronic iron overload in patients with non-transfusion-dependent
thalassemia . Processed and transmitted by Thomson Reuters ONE. The issuer is
solely responsible for the content of this announcement.

  *Pivotal placebo-controlled study data show Exjade significantly decreases
    iron burden in NTDT patients versus placebo, with similar overall adverse
    event rate[1]

  *Patients with NTDT accumulate excess iron increasing their risk of
    complications, including liver fibrosis, cirrhosis, blood clots, and bone
    and vascular disease[2]

  *At least three quarters of a million people worldwide have NTDT[3]-[5] and
    many patients are undiagnosed until serious symptoms arise[6]

Basel, January 23, 2013-  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  regular  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).

In the European Union (EU), Exjade is an oral iron chelation therapy indicated
for the treatment of chronic iron overload due to frequent blood  transfusions 
(>=7 ml/kg/month of packed red blood cells) in patients with  beta-thalassemia 
aged 6 years and older. It is also indicated for the treatment of chronic iron
overload  due   to   blood   transfusions   when   deferoxamine   therapy   is 
contraindicated or inadequate in the  following patient groups: patients  with 
beta-thalassemia major with iron overload  due to frequent blood  transfusions 
(>=7 ml/kg/month of packed red blood cells)  aged 2 to 5 years; patients  with 
beta-thalassemia major with iron overload due to infrequent blood transfusions
(<7 ml/kg/month  of  packed red  blood  cells) aged  2  years and  older;  and 
patients with other anemias aged 2  years and older. Exjade is also  indicated 
for the treatment of  chronic iron overload  requiring chelation therapy  when 
deferoxamine  therapy  is  contraindicated  or  inadequate  in  patients  with 
non-transfusion-dependent thalassemia syndromes aged 10 years and older.

Exjade is approved in over 100 countries including the US, Switzerland,  Japan 
and countries comprising the  EU. The approved  indication may vary  depending 
upon the individual country.

Exjade Important Safety Information
Exjade is contraindicated in patients  with an estimated creatinine  clearance 
<60 mL/min,  with hypersensitivity  to  the active  substance  or any  of  the 
excipients, or in combination  with other iron  chelator therapies. Exjade  is 
not recommended in patients with severe hepatic impairment.

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 for  more 
information.

Disclaimer
The  foregoing  release  contains  forward-looking  statements  that  can   be 
identified by  terminology  such as  "will,"  or similar  expressions,  or  by 
express or  implied  discussions  regarding  potential  future  revenues  from 
Exjade. You  should  not  place  undue  reliance  on  these  statements.  Such 
forward-looking statements reflect the  current views of management  regarding 
future events, and 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  achieve  any 
particular levels  of  revenue  in the  future.  In  particular,  management's 
expectations regarding  Exjade  could  be affected  by,  among  other  things, 
unexpected clinical trial results, including unexpected new clinical data  and 
unexpected  additional  analysis   of  existing   clinical  data;   unexpected 
regulatory actions or delays  or government regulation generally;  competition 
in  general;  government,  industry  and  general  public  pricing  pressures; 
unexpected manufacturing issues; the company's  ability to obtain or  maintain 
patent or other proprietary intellectual property protection; the impact  that 
the foregoing factors  could have  on the  values attributed  to the  Novartis 
Group's assets and liabilities as recorded in the Group's consolidated balance
sheet, and other risks and factors  referred to in Novartis AG's current  Form 
20-F on file  with the US  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.

About Novartis
Novartis provides innovative  healthcare solutions that  address the  evolving 
needs of patients and societies. Headquartered in Basel, Switzerland, Novartis
offers a diversified portfolio to best meet these needs: innovative medicines,
eye  care,  cost-saving  generic  pharmaceuticals,  preventive  vaccines   and 
diagnostic tools, over-the-counter and animal health products. Novartis is the
only global company with leading positions in these areas. In 2011, the  Group 
achieved net sales of  USD 58.6 billion, while  approximately USD 9.6  billion 
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around the world. For more information, please visit http://www.novartis.com.

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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 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 B 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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