Regeneron's Rilonacept Granted European Marketing Authorization for Treatment
of Cryopyrin-Associated Periodic Syndromes (CAPS)
TARRYTOWN, N.Y., Oct. 27 /PRNewswire-FirstCall/ -- Regeneron Pharmaceuticals,
Inc. (Nasdaq: REGN) today announced it has received marketing authorization in
the European Union for rilonacept, an interleukin-1 blocker, for the treatment
of Cryopyrin-Associated Periodic Syndromes (CAPS) with severe symptoms,
including Familial Cold Auto-inflammatory Syndrome (FCAS) and Muckle-Wells
Syndrome (MWS), in adults and children aged 12 years and older. Marketing
authorization for rilonacept was granted by the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency (EMEA) under
exceptional circumstances.
"Rilonacept, which is known as ARCALYST® in the United States, has been
prescribed to treat the majority of patients documented to have been treated
for CAPS symptoms to date in the U.S. ARCALYST is the only treatment for CAPS
in the U.S. that has demonstrated a significant improvement in CAPS symptoms
as reported by patients in a pivotal clinical program and is approved for
self-administration at home," said Robert Terifay, Senior Vice President,
Commercial at Regeneron. "Currently, rilonacept is available to patients in
the European Union through their prescribers on a named-patient or
individualized case review basis. We are evaluating our broader
commercialization options for this very small, ultra-orphan patient
population, which is dispersed across Europe."
CAPS include a group of rare, inherited, auto-inflammatory conditions
characterized by life-long, recurrent symptoms of rash, fever/chills, joint
pain, eye redness/pain, and fatigue. Intermittent, disruptive exacerbations
or flares can be triggered at any time by exposure to cooling temperatures,
stress, exercise, or other unknown stimuli. Rilonacept is a targeted
inhibitor of interleukin-1 (IL-1), the key driver of inflammation in CAPS. In
the pivotal clinical development program, patients treated with rilonacept
reported a greater improvement in overall symptom scores than patients treated
with placebo. These improvements were sustained over time with continued
rilonacept treatment. Patients reported their symptoms using a validated
daily diary instrument. These assessments represent a critical measure of
effectiveness in a disease characterized by frequent, unpredictable symptom
flares of variable severity and duration. Unlike other agents used in the
treatment of CAPS, rilonacept is supported by patient-reported symptoms data
using a validated assessment instrument.
Rilonacept has been developed as a once-weekly injection which can be
administered at home by the patient or their care giver following appropriate
training. The most commonly reported adverse reactions with rilonacept were
injection-site reaction and upper respiratory tract infection. IL-1 blockade
may interfere with immune response to infections and treatment should not be
initiated in patients with active or chronic infections. There have been
reports of serious, life-threatening infections in patients taking rilonacept.
Rilonacept should be discontinued if a patient develops a serious infection.
The authorization for approval of rilonacept in the E.U. under exceptional
circumstances is permissible for products for which a company can demonstrate
that comprehensive data cannot be provided, for example because of the rarity
of the condition. Each year, Regeneron will need to provide the EMEA with any
new information that may become available for review.
About Cryopyrin-Associated Periodic Syndromes (CAPS)
Recently, medical researchers have identified and described a group of rare,
inherited, auto-inflammatory disorders, known as Cryopyrin-Associated Periodic
Syndromes or CAPS. Three related conditions make up the broader disease known
as CAPS: Familial Cold Auto-inflammatory Syndrome (FCAS), Muckle-Wells
Syndrome (MWS), and Neonatal-Onset Multisystem Inflammatory Disease (NOMID).
Rilonacept has not been studied in patients with NOMID.
CAPS are characterized by life-long, recurrent symptoms of rash, fever/chills,
joint pain, eye redness/pain, and fatigue. Intermittent, disruptive
exacerbations or flares can be triggered at any time by exposure to cooling
temperatures, stress, exercise, or other unknown stimuli.
CAPS are generally caused by autosomal-dominant mutations (changes) in the
NLRP-3 (previously known as CIAS1) gene and resultant alterations in the
protein, cryopyrin, which it encodes. Cryopyrin, active in circulating,
infection-fighting, white blood cells, controls the production of a protein
called interleukin-1 (IL-1). As part of the body's infection-fighting defense
system, IL-1 circulates throughout the body and can trigger inflammatory
reactions when it binds to inflammatory cells. Researchers have found that
alterations in the cryopyrin protein lead to over-production of IL-1,
resulting in an inflammatory response and the symptoms of CAPS. Most, but not
all, patients with CAPS have the NLRP-3 gene mutation.
The incidence of CAPS has been estimated to be approximately 1 in 1,000,000
people in the European Union.
About Rilonacept
Rilonacept is a targeted inhibitor of interleukin-1 (IL-1), the key driver of
inflammation in Cryopyrin-Associated Periodic Syndromes (CAPS). In the
pivotal clinical development program for rilonacept, change in disease
activity was measured using a composite patient-reported symptom score
composed of a daily evaluation of rash, feelings of fever/chills, joint pain,
eye redness/pain, and fatigue. Patients treated with rilonacept experienced
an improvement in overall symptom scores as compared with patients treated
with placebo. These improvements were sustained over time with continued
treatment with rilonacept. The most commonly reported adverse reactions with
rilonacept were injection-site reaction and upper respiratory tract infection.
In the United States, rilonacept (ARCALYST®) is indicated for the treatment of
Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold
Auto-inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults
and children 12 and older. ARCALYST was approved by the U.S. Food and Drug
Administration in February 2008 and has been available in the United States
since March 2008.
IL-1 blockade may interfere with immune response to infections. Serious,
life-threatening infections have been reported in patients taking rilonacept.
Rilonacept should be discontinued if a patient develops a serious infection.
Treatment with rilonacept should not be initiated in patients with active or
chronic infections. Taking rilonacept with tumor necrosis factor inhibitors
is not recommended because this may increase the risk of serious infections.
Patients should not receive a live vaccine while taking rilonacept. It is
recommended that patients receive all recommended vaccinations prior to
initiation of treatment with rilonacept. Patients should be monitored for
changes in their lipid profiles and provided with medical treatment if
warranted. Hypersensitivity reactions associated with rilonacept
administration have been rare. Please see the full U.S. Prescribing
Information for ARCALYST® (rilonacept), available online at
www.regeneron.com/ARCALYST-fpi.pdf. Please see the European Summary of
Product Characteristics at www.regeneron.com/RILONACEPT-EU.pdf.
About Regeneron Pharmaceuticals
Regeneron is a fully integrated biopharmaceutical company that discovers,
develops, and commercializes medicines for the treatment of serious medical
conditions. In addition to ARCALYST® (rilonacept) Injection for Subcutaneous
Use, its first commercialized product in the United States, Regeneron has
therapeutic candidates in clinical trials for the potential treatment of
cancer, eye diseases, inflammatory diseases, and pain and has preclinical
programs in other diseases and disorders. Additional information about
Regeneron and recent news releases are available on Regeneron's web site at
www.regeneron.com.
Forward Looking Statement
This news release discusses historical information and includes
forward-looking statements about Regeneron and its products, development
programs, finances, and business, all of which involve a number of risks and
uncertainties, such as risks associated with preclinical and clinical
development of Regeneron's drug candidates, determinations by regulatory and
administrative governmental authorities which may delay or restrict
Regeneron's ability to continue to develop or commercialize its product and
drug candidates, competing drugs that are superior to Regeneron's product and
drug candidates, uncertainty of market acceptance of Regeneron's product and
drug candidates, unanticipated expenses, the availability and cost of capital,
the costs of developing, producing, and selling products, the potential for
any collaboration agreement, including Regeneron's agreements with the
sanofi-aventis Group and Bayer HealthCare, to be canceled or to terminate
without any product success, risks associated with third party intellectual
property, and other material risks. A more complete description of these and
other material risks can be found in Regeneron's filings with the United
States Securities and Exchange Commission (SEC), including its Form 10-K for
the year ended December 31, 2008 and Form 10-Q for the quarter ended June 30,
2009. Regeneron does not undertake any obligation to update publicly any
forward-looking statement, whether as a result of new information, future
events, or otherwise unless required by law.
Contact Information:
Peter Dworkin Laura Lindsay
Investor Relations Media Relations
914.345.7640 914.345.7800
peter.dworkin@regeneron.com laura.lindsay@regeneron.com
SOURCE Regeneron Pharmaceuticals, Inc.
Peter Dworkin, Investor Relations, +1-914-345-7640,
peter.dworkin@regeneron.com, or Laura Lindsay, Media Relations,
+1-914-345-7800, laura.lindsay@regeneron.com
