EAST HANOVER, N.J.,
Sept. 23, 2016 /PRNewswire/ --
Novartis announced today that the US Food and Drug Administration
(FDA) has granted three simultaneous approvals for the expanded use
of Ilaris® (canakinumab) to treat three rare and
distinct types of Periodic Fever Syndromes.
Ilaris is the first and only FDA-approved biologic treatment for
patients with Tumor Necrosis Factor Receptor-Associated Periodic
Syndrome (TRAPS), Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate
Kinase Deficiency (MKD) and Familial Mediterranean Fever
(FMF).1,2 All three conditions are part of a group of
rare autoinflammatory diseases called Periodic Fever Syndromes,
which are also referred to as Hereditary Periodic Fevers (HPF). The
most common syndrome is FMF, which mainly affects people of Eastern
Mediterranean ancestry. It affects 1 in 250 to 1 in 1,000
individuals in these populations, many of whom are
children.3
"We are grateful to the scientists, clinical trial investigators
and all associates who worked tirelessly in support of patients to
gain three simultaneous FDA approvals of ILARIS," said Fabrice Chouraqui, President of Novartis
Pharmaceuticals Corporation, "It is through their hard work that
Novartis can offer a desperately needed new treatment option to the
adults and children who suffer from these debilitating conditions
and continue to serve the rare disease community."
The FDA approvals are based on results from the pivotal Phase
III CLUSTER study which showed rapid (at Day 15) and sustained
disease control with Ilaris compared to placebo through 16 weeks,
in patients with either TRAPS, HIDS/MKD or FMF.2 As a
result of the positive data, the FDA granted Ilaris Breakthrough
Therapy status and priority reviews for each of the three Periodic
Fever Syndrome conditions.
Periodic Fever Syndromes are a group of rare autoinflammatory
diseases that cause disabling and persistent fevers which may be
accompanied by joint pain, swelling, muscle pain and skin rashes
with complications that can be life-threatening.1
"Adults and children living with TRAPS, HIDS/MKD or FMF often
experience extensive delays in diagnosis because the disorders are
so rare that many physicians are unaware of them," said
Hal M. Hoffman, M.D., chief of
Pediatric Allergy, Immunology, and Rheumatology at Rady Children's
Hospital-San Diego and University of
California San Diego. "Following diagnosis, our goal is to
get patients treated as soon as possible and that has been
challenging due to the lack of available treatment options. That's
what makes these three approvals for ILARIS so important for
patients."
Ilaris is already approved and marketed in the US as an
effective and well-tolerated treatment for another Periodic Fever
Syndrome condition – Cryopyrin-Associated Periodic Syndromes
(CAPS), and another autoinflammatory condition – Systemic Juvenile
Idiopathic Arthritis (SJIA).
About Periodic Fever Syndromes
Periodic Fever
Syndromes are a group of diseases that cause serious recurrent
fever and inflammation through non-infectious activation of the
immune system. Most patients present with symptoms in infancy or
childhood, but in some patients the condition only becomes apparent
or diagnosed in adulthood.1
Previous treatments for these rare conditions consisted of oral
anti-inflammatory drugs, such as corticosteroids, which were used
only to help manage the symptoms. While other medicines, such as
non-steroidal anti-inflammatory drugs, have also been used to help
reduce symptoms, they do not prevent or change the overall course
of a flare.1
About Ilaris
Ilaris is a selective, high-affinity,
human monoclonal antibody that inhibits Interleukin-1 (IL-1) beta,
which is an important part of the body's immune system
defenses.4 Excessive production of IL-1 beta plays a
prominent role in certain inflammatory
diseases.5,6 Ilaris works by blocking
the action of IL-1 beta for a sustained period of time, therefore
inhibiting inflammation that is caused by its
over-production.4
Ilaris is currently approved and marketed for the treatment of
SJIA in the US and EU and for the treatment of Adult-Onset Still's
Disease (AOSD) and the symptomatic treatment of refractory acute
gouty arthritis in the EU. Ilaris is also approved in more than 70
countries, including in the EU, Switzerland, Canada, and Japan for the treatment of the Periodic Fever
Syndrome CAPS: rare, lifelong, genetic disorders with debilitating
symptoms. In the US, Ilaris is approved for two subtypes of CAPS:
Muckle-Wells Syndrome (MWS) and Familial Cold Autoinflammatory
Syndrome (FCAS). The approved indications may vary depending upon
the individual country.
Important Safety Information
ILARIS can cause
serious side effects, including increased risk of serious side
infections. ILARIS can lower the ability of your immune
system to fight infections. Your healthcare provider should: test
you for tuberculosis (TB) before you receive ILARIS; monitor
you closely for symptoms of TB during treatment with
ILARIS; check you for symptoms of any type of infection
before, during, and after treatment with ILARIS. Tell your
healthcare provider right away if you have any symptoms of an
infection such as fever, sweats or chills, cough, flu-like
symptoms, weight loss, shortness of breath, blood in your phlegm,
sores on your body, warm or painful areas on your body, diarrhea or
stomach pain, or feeling very tired.
You should not receive ILARIS if you are allergic to canakinumab
or any of the ingredients in ILARIS.
Before you receive ILARIS, tell your healthcare provider
about all your medical conditions, including if you: think you
have or are being treated for an active infection; have symptoms of
an infection; have a history of infections that keep coming
back; have a history of low white blood cells; have
or have had HIV, Hepatitis B, or Hepatitis C; are scheduled
to receive any immunizations (vaccines) as you should not get 'live
vaccines' if you are receiving ILARIS; are pregnant or planning to
become pregnant since it is not known if ILARIS will harm an unborn
baby (patients who become pregnant while receiving ILARIS should
tell their healthcare provider right away); are breastfeeding
or planning to breastfeed as it is not known if ILARIS passes into
your breast milk. You and your healthcare provider should
decide if you will receive ILARIS or breastfeed. You should
not do both.
ILARIS can cause serious side effects, including: serious
infections; decreased ability of your body to fight infections
(immunosuppression; for people treated with medicines that cause
immunosuppression like ILARIS, the chances of getting cancer may
increase); allergic reactions (call your healthcare provider
right away if you have any of these symptoms of an allergic
reaction: rash, itching and hives, difficulty breathing or
swallowing, dizziness or feeling faint); risk of infection with
live vaccines (you should not get live vaccines if you are
receiving ILARIS; tell your healthcare provider if you are
scheduled to receive any vaccines).
The most common side effects of ILARIS include:
When ILARIS is used for the treatment of CAPS: cold
symptoms; diarrhea, flu (influenza), runny nose, headache, cough,
body aches; nausea, vomiting, and diarrhea (gastroenteritis),
feeling like you are spinning (vertigo), weight gain, injection
site reactions (such as redness, swelling, warmth, or itching) and
nausea.
When ILARIS is used for the treatment of TRAPS, HIDS/MKD, and
FMF: cold symptoms, upper respiratory tract infection, runny
nose, sore throat, nausea, vomiting, and diarrhea
(gastroenteritis), and injection site reactions (such as redness,
swelling, warmth, or itching).
When ILARIS is used for the treatment of SJIA: cold
symptoms, upper respiratory tract infection, pneumonia, runny nose,
sore throat, urinary tract infection, nausea, vomiting and diarrhea
(gastroenteritis), stomach pain, and injection site reactions (such
as redness, swelling, warmth, or itching).
What is Macrophage Activation Syndrome (MAS)?
MAS is a syndrome associated with SJIA and some other
autoinflammatory diseases like HIDS/MKD that can lead to death.
Tell your healthcare provider right away if your SJIA symptoms get
worse or if you have any of these symptoms of an infection: a fever
lasting longer than 3 days; a cough that does not go away; redness
in one part of your body; warm feeling or swelling of your
skin.
You are encouraged to report negative side effects of
prescription drugs to FDA. Visit www.fda.gov/medwatch or call
1-800-FDA-1088
Please see full Prescribing Information, including Medication
Guide at
https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/ilaris.pdf
for additional Important Safety Information.
Disclaimer
The foregoing release contains
forward-looking statements that can be identified by words such as
"Breakthrough Therapy Designations," "Breakthrough Therapy,"
"goal," or similar terms, or by express or implied discussions
regarding potential new indications or labeling for Ilaris, or
regarding potential future revenues from Ilaris. You should not
place undue reliance on these statements. Such forward-looking
statements are based on the current beliefs and expectations of
management regarding future events, and are subject to significant
known and unknown risks and uncertainties. Should one or more of
these risks or uncertainties materialize, or should underlying
assumptions prove incorrect, actual results may vary materially
from those set forth in the forward-looking statements. There can
be no guarantee that Ilaris will be submitted or approved for any
additional indications or labeling in any market, or at any
particular time. Nor can there be any guarantee that Ilaris will be
commercially successful in the future. In particular, management's
expectations regarding Ilaris could be affected by, among other
things, the uncertainties inherent in research and development,
including unexpected clinical trial results and additional analysis
of existing clinical data; unexpected regulatory actions or delays
or government regulation generally; the company's ability to obtain
or maintain proprietary intellectual property protection; general
economic and industry conditions; global trends toward health care
cost containment, including ongoing pricing pressures; unexpected
safety, quality or manufacturing issues, and other risks and
factors referred to in Novartis AG's current Form 20-F on file
with the US Securities and Exchange Commission. 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 Pharmaceuticals Corporation
offers a broad range of medicines for cancer, cardiovascular
disease, endocrine disease, inflammatory disease, infectious
disease, neurological disease, organ transplantation, psychiatric
disease, respiratory disease and skin conditions.
Located in East Hanover, NJ
Novartis Pharmaceuticals Corporation is an affiliate of Novartis
AG, which 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 and cost-saving generic pharmaceuticals.
Novartis is the only global company with leading positions in these
areas. In 2015, the Group achieved net sales of USD 49.4 billion, while R&D throughout the
Group amounted to approximately USD 8.9
billion (USD 8.7 billion
excluding impairment and amortization charges). Novartis Group
companies employ approximately 118,000 full-time-equivalent
associates. Novartis products are available in more than 180
countries around the world. For more information, please visit
http://www.novartis.com.
References
- Cleveland Clinic. Periodic Fever Syndrome. Available at:
https://my.clevelandclinic.org/services/orthopaedics-rheumatology/diseases-conditions/periodic-fever-syndrome.
Accessed September
2016.https://my.clevelandclinic.org/services/orthopaedics-rheumatology/diseases-conditions/periodic-fever-syndrome.
Accessed September 2016.
- Novartis. Ilaris Prescribing Information. September 2016. Available at:
www.pharma.us.novartis.com/product/pi/pdf/ilaris.pdf. Accessed
September 2016.
- National Amyloidosis Centre. Amyloidosis Patient Information
Site: The inherited periodic fever syndromes – general information.
Available at:
www.amyloidosis.org.uk/fever-syndromes/inherited-fever-syndromes/.
Accessed September 2016.
- Dhimolea E. Canakinumab, MAbs. 2010
Jan-Feb;2(1):3-13.
- Jesus AA, Goldbach-Mansky R. IL-1 blockade in autoinflammatory
syndromes. Annu Rev Med.
2014;65:223-244.
- Toker O, Hashkes PJ. Critical appraisal of canakinumab in the
treatment of adults and children with cryopyrin-associated periodic
syndrome (CAPS). Biologics. 2010;4:131-138.
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