IlarisÂ® recommended for European approval as new biologic drug to
treat a rare but serious group of auto-inflammatory diseases
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* Set to become first medicine in EU to treat patients aged four
and older suffering from life-long cryopyrin-associated periodic
* Ilaris targets interleukin-1 beta (IL-1ÃŸ), a key driver of
inflammation,, - studies ongoing in other diseases
involving IL-1ÃŸ such as gout, COPD and type 2 diabetes
* EU opinion follows US and Swiss approvals based on data showing
Ilaris produced rapid and sustained remission in CAPS patients
after one dose
* CAPS comprises three disorders of increasing severity with
potentially fatal complications, - most patients suffer
from severe and disabling symptoms,
Basel, July 24, 2009 - The biotechnology medicine IlarisÂ®
(canakinumab) has passed another major milestone with a
recommendation for approval in the European Union to treat patients
with a life-long and potentially fatal auto-inflammatory disease
called cryopyrin-associated periodic syndrome (CAPS). When approved,
Ilaris will be the only treatment in the EU indicated for CAPS
patients aged four years and older.
Ilaris represents an important advance in the development of
personalized medicines because it targets a condition that is
triggered by a specific genetic mutation. In CAPS patients, this
mutation drives the overproduction of interleukin 1-beta (IL-1ÃŸ)
which causes the widespread sustained inflammation and tissue damage
associated with the disease,,.
Because Ilaris normalizes the production of IL-1ÃŸ,,, it is
also being studied in other diseases in which IL-1ÃŸ plays a pivotal
role such as systemic juvenile idiopathic arthritis (SJIA), gout,
chronic obstructive pulmonary disorder (COPD), and type 2 diabetes.
"By concentrating initially on a rare syndrome with a well-defined
disease process such as CAPS, we have been able to demonstrate a
clear therapeutic advantage with Ilaris," said Trevor Mundel, MD,
Head of Global Development at Novartis Pharma AG. "Our focus now is
to establish whether this could also provide a new approach to the
treatment of other diseases involving a similar underlying process."
A positive opinion recommending the approval of Ilaris for CAPS was
issued by the Committee for Medicinal Products for Human Use (CHMP),
which reviews medicines for the European Commission. The
recommendation comes shortly after approvals in the US and
Switzerland where Ilaris was granted priority review based on its
potential to fulfil an important unmet need for CAPS patients.
The EU submission was supported by data showing that Ilaris, a
monoclonal antibody formerly known as ACZ885, produced rapid and
sustained remission of symptoms in up to 97% of CAPS patients, with
most responding from the first injection.
Ilaris is given by subcutaneous injection only once every two months
making it a convenient treatment, especially for younger patients.
More than 90% of patients studied were free from painful
CAPS includes three distinct auto-inflammatory disorders. These are
familial cold auto-inflammatory syndrome (FCAS) which is the mildest
form of CAPS, Muckle-Wells syndrome (MWS), and neonatal-onset
multisystem inflammatory disease (NOMID, also known as chronic
infantile neurological cutaneous articular syndrome or CINCA) - the
most severe form of the disease,.
"CAPS is a life-long and potentially fatal condition for which there
are currently no approved medications in the European Union," said
Helen J. Lachmann, MD of the UK National Amyloidosis Centre at UCL
Medical School in London, UK. "In clinical trials, canakinumab has
been shown to switch off disease activity in as little as 24 hours
following a single dose. It has the potential to transform patients'
lives, not only providing relief from their debilitating daily
symptoms but also offering the possibility of long-term control of
The symptoms of CAPS, such as debilitating fatigue, rash, fever,
headaches, joint pain and conjunctivitis, can be present from birth
or infancy, and can occur daily throughout patients' lives,.
Serious long-term consequences may include deafness, bone
deformities, erosive joint destruction, and central nervous system
damage leading to loss of vision,,. Around 25% of CAPS
patients develop amyloidosis, a condition in which the build-up of
proteins can cause vital organs to fail, resulting in renal failure
and death within five to 10 years.
CAPS is believed to occur in around 6,500 patients worldwide and
2,500 in the EU,. However due to lack of diagnosis or
misdiagnosis, fewer than 1,000 cases have been officially reported
The Ilaris filing was based on a clinical trial program involving
more than 100 CAPS patients. The pivotal study is a three-part,
one-year Phase III study involving 35 patients aged nine to 74 years
old with varying degrees of disease severity. Data published in
The New England Journal of Medicine in June 2009 show that Ilaris
produced a rapid, complete and sustained response in the majority of
Results for the primary endpoint showed that none of the patients
treated with Ilaris (0 out of 15) experienced a disease outbreak or
'flare' compared to 13 of the 16 patients who received placebo (0%
vs. 81% respectively, p<0.001).
In general, Ilaris was well tolerated with no consistent pattern of
adverse events apart from a slight increase in infections. Two
patients experienced serious adverse events, namely a lower urinary
tract infection and vertigo. The most common adverse events
reported in Ilaris-treated patients were nasopharyngitis, diarrhea,
influenza, headache and nausea.
No impact on the type or frequency of adverse events was seen with
longer-term treatment. Ilaris was not associated with any severe
reactions at the injection site, and those that did occur were
mild-to-moderate in nature.
The CHMP recommended approval under exceptional circumstances,
granted when comprehensive data are not yet available due to the
rarity of the disease or limited scientific knowledge. The approval
is subject to certain obligations for the company and is re-assessed
each year until normal approval can be given.
Ilaris was approved in Switzerland in July 2009 to treat all three
forms of CAPS in adults and children over four years old, and in the
US in June 2009 to treat two forms of CAPS, namely FCAS and MWS. A
study in NOMID patients is under way in the US and priority reviews
are being conducted in other countries, including Australia and
In addition to orphan drug status for CAPS, Ilaris has also been
designated as an orphan drug for treating systemic juvenile
idiopathic arthritis (SJIA) in the US, EU and Switzerland, and has
fast-track status for SJIA in the US. Orphan drugs are those
developed to treat diseases affecting fewer than 200,000 people (in
the US) or fewer than five out of 10,000 people (in the EU).
The foregoing release contains forward-looking statements that can be
identified by terminology such as "potentially," "will," "could,"
"potential," "can," "may," or similar expressions, or by express or
implied discussions regarding potential future regulatory filings or
marketing approvals for Ilaris, or the timing of any such potential
filings or approvals, or regarding potential future revenues from
Ilaris. You should not place undue reliance on these statements. Such
forward-looking statements reflect the current views of the Company
regarding future events, and involve known and unknown risks,
uncertainties and other factors that may cause actual results with
Ilaris to be materially different from any future results,
performance or achievements expressed or implied by such statements.
There can be no guarantee that Ilaris will be approved for sale in
any additional market, or for any additional indication, or that any
such approvals will occur at any particular time. Nor can there be
any guarantee that Ilaris will achieve any levels of revenue in the
future. In particular, management's expectations regarding Ilaris
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; the company's
ability to obtain or maintain patent or other proprietary
intellectual property protection; competition in general; government,
industry and general public pricing pressures; the impact that the
foregoing factors could have on the values attributed to the 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.
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Novartis offers a diversified portfolio to best meet these needs:
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1. National Horizon Scanning Centre. Canakinumab for cryopyrin
associated periodic syndrome. November 2008. Available at:
Last accessed April 21, 2009.
2. Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB, et al. Use of
Canakinumab in the Cryopyrin-Associated Periodic Syndrome. N Engl J
Med, 360;23. June 4, 2009.
3. Durrant KLW, Goldbach-Mansky R, Hoffman H, Leslie K, Rubin B.
CAPS Cryopyrin-Associated Periodic Syndromes 2008. Available at:
http://www.nomidalliance.net/Download1.html Last accessed April 19,
4. Joost PH, Drenth MD, Jos W.M. van der Meer. The Inflammasome -
A Linebacker of Innate Defense. N Engl J Med 2006. Vol 355:730-732.
5. Lachmann HJ, Lowe P, Felix SD, et al. In vivo regulation of
interleukin 1 in patients with cryopyrin-associated periodic
syndromes. J Exp Med 2009. Published online April 13, 2009. Available
6. European Medicines Agency (EMEA). Pre-authorisation evaluation
of medicines for human use. Committee for orphan medicinal products.
accessed 14 July 2009.
7. Orphan Drug Act. US Food and Drug Administration. Section 526
8. The orphan drug strategy. Europa: Gateway to the European
Union. Paragraph 1, Line 1.
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