Novartis drug Exjade® recommended by CHMP for EU approval to treat patients with non-transfusion-dependent thalassemia syndromes
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Novartis drug Exjade® recommended by CHMP for EU approval to treat patients with
non-transfusion-dependent thalassemia syndromes
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* Exjade would be the first oral treatment approved specifically for patients
with non-transfusion-dependent thalassemia (NTDT) syndromes
* Clinical data show Exjade significantly decreases iron burden in NTDT
patients compared to placebo, with similar overall adverse event rate[1]
* NTDT patients accumulate excess iron, increasing their risk of
complications, including liver fibrosis, cirrhosis, blood clots and bone and
vascular disease[2]
Basel, November 16, 2012 - Novartis announced today that the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)
adopted a positive opinion for Exjade(®) (deferasirox) for the treatment of
chronic iron overload requiring chelation therapy when deferoxamine therapy is
contraindicated or inadequate in patients aged 10 years and older with non-
transfusion-dependent thalassemia (NTDT) syndromes. Exjade would be the first
oral treatment in the European Union (EU) specifically indicated for the
treatment of iron overload in patients with these types of thalassemia.
Results from the first prospective placebo-controlled study of iron chelation in
NTDT patients, THALASSA, showed a significant dose-dependent decrease in iron
burden compared to placebo[1]. In this pivotal study, Exjade was well tolerated,
with an overall adverse event rate similar to the placebo arm[1].
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 can live without regular transfusions, a
significant cause of 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],[3].
"Patients with NTDT have suffered the effects of iron overload without accurate
diagnosis, clear treatment guidelines or specifically approved oral therapies,"
said Hervé Hoppenot, President, Novartis Oncology. "The CHMP recommendation is
an important step toward improving the outcomes of patients with this type of
thalassemia."
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[4],[5],[6]. Because NTDT patients
are not symptomatic at birth, when most thalassemias are diagnosed, they are
often underdiagnosed and undertreated[7]. Many complications associated with
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[7],[9].
The European Commission generally follows the recommendations of the CHMP and
usually delivers its final decision within three months of the CHMP
recommendation. The decision will be applicable to all 27 EU member states plus
Iceland and Norway. Exjade has been approved to treat chronic iron overload in
patients with NTDT in Canada and several other countries; further regulatory
submissions are ongoing.
About Exjade
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[10].
It is approved in more than 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 "recommended," "would," "positive opinion,"
"recommendation," "will," "generally follows," or similar expressions, or by
express or implied discussions regarding potential new indications or labeling
for Exjade or 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 be approved for any additional indications or
labeling in any market, or at any particular time. Nor can there be any
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 regulatory actions or delays or
government regulation generally; unexpected clinical trial results, including
unexpected new clinical data and unexpected additional analysis of existing
clinical data; 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's
continuing operations achieved net sales of USD 58.6 billion, while
approximately USD 9.6 billion (USD 9.2 billion excluding impairment and
amortization charges) was invested in R&D throughout the Group. Novartis Group
companies employ approximately 127,000 full-time-equivalent associates and
operate in more than 140 countries around the world. For more information,
please visit http://www.novartis.com.
Novartis is on Twitter. Sign up to follow (at)Novartis at
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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] 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 ß thalassemia intermedia. Haematologica. 2011 Nov;96(11):1605-12.
[4] Vichinsky E. Hemoglobin E syndromes. Hematology Am Soc Hematol Educ Program.
2007;79-83.
[5] Weatherall DJ. The definition and epidemiology of non-transfusion-dependent
thalassemia. Blood Reviews. 2012:26S:S3-S6.
[6] Vichinsky EP. Changing patterns of thalassemia worldwide. Ann NY Acad Sci.
2005;1054:18-24.
[7] 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.
[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] EMC. Summary of product characteristics: EXJADE 125 mg, 250mg, 500mg
dispersible tablets. Last updated January 13, 2012. Accessed at:
http://www.medicines.org.uk/emc/medicine/18805.
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Datum: 16.11.2012 - 13:46 Uhr
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